ValiSeek – ValiRx unveils positive results from VAL401 Phase II lung cancer trial

Below, we are re-publishing the news issued by ValiSeek on the 12th of December 2017

ValiSeek – Clinical Development Update on VAL401

Dr Suzy Dilly, CEO of ValiSeek provides a positive update on the clinical progress of VAL401, the anti-cancer compound. The Company has released positive formal data pertaining to disease impact. The results demonstrate that the VAL401 treatment has a statistically significant improvement in Overall Survival for patients with non-small cell lung cancer compared to those receiving no treatment.

Further information is available at: http://www.valirx.com/video/valirx-unveils-positive-results-val401-phase-ii-lung-cancer-trial/ 

* The study is conducted by Clinical Accelerator

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Clinical Accelerator announces collaboration with VisCardia for investigating a novel device therapy for chronic heart failure

Kiev, Ukraine – December 11, 2017: Clinical Accelerator, a full-service clinical contract research organization, is pleased to announce it has entered into a collaboration with VisCardia Inc. for conducting clinical studies for its VisONE medical device in the Ukraine.

The purpose of the study is to demonstrate the VisONE system improves hemodynamic parameters associated with improved cardiac structure, symptoms and outcomes in patients with chronic heart failure.

VisCardia’s VisOne system is a fully implantable, electronic sensor and electrode system for delivering chronic asymptomatic diaphragmatic stimulation for modulating thoracic pressure augmentation. The system is implanted through a minimally invasive procedure, and programmed for individualized patient use using an external programmer.

According to VisCardia the study intends to demonstrate improvements in accepted “gold standard” cardio physiologic parameters, indicative of patient well-being, while observing clinical heart failure endpoints.

About Clinical Accelerator

Clinical Accelerator is an independent clinical trial management organisation operating principally in Central and Eastern Europe, Russia, Ukraine and CIS countries. The organisation offers a broad range of clinical trial services together with dedicated patient enrolment support to worldwide clients in the pharmaceutical, biotechnological, nutraceutical and medical device industries. Clinical Accelerator’s model of operation is designed to achieve significant cost savings for its clients and to guarantee compact timelines for patient enrolment with a firm focus on the quality of clinical trial data.

About VisCardia

VisCardia is a privately held company located in Portland Oregon, USA, developing a novel fully implantable medical device therapy to improve hemodynamic function for managing medical refractory heart failure. Their proprietary approach uses the patient’s own thoracic musculature to improve cardiac filling and output, and relieve symptoms resulting from hemodynamic insuffciency.

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Promising Interim Clinical Data for NOX66

Below, we are re-publishing with permission the press-release issued by Noxopharm Limited on the 20th of November 2017

PROMISING INTERIM CLINICAL DATA FOR NOX66

  • Patients with metastatic, late-stage cancers
  • 10 of 11 patients showing no disease progression following 3-months treatment with NOX66 in combination with low-dose carboplatin
  • Well-tolerated drug combination

20 November 2017, Sydney: Noxopharm is pleased to release interim data from its first-in-human study of NOX66 being conducted in Georgia. The data was presented to the European Society of Medical Oncology Asia conference in Singapore on 18 November 2017.

The data shows promising outcomes both in terms of safety and disease сontrol in patients receiving NOX66 in combination with a low dose of carboplatin. The Company notes that this is interim data, with the majority of patients yet to reach the end of the study, and in particular yet to include additional data from the higher dose of NOX66 considered by the Company to be the final clinical dose.

The headline data read-out is:
• 19 patients have been enrolled with late-stage (Stage 4) metastatic solid cancers (breast, ovarian, lung, prostate, head & neck)
• patients must have failed to respond to standard treatment options and be eligible for experimental drug therapy
• 11 of the 19 patients have been treated for 3 months with a combination of NOX66 and low-dose carboplatin
• only 1 of the 11 patients showed disease progression over this time; of the remaining 10 patients, 9 showed stable disease and 1 showed a partial response
• NOX66 alone and in combination with low-dose carboplatin was well tolerated and with no reported adverse events.

Study rationale
The majority of patients with metastatic disease involving solid cancers eventually run out of treatment options and experience unchecked disease progression.

The hypothesis behind the current study is that a combination of NOX66 and carboplatin will block disease progression and provide a meaningful survival benefit in patients whose cancers have stopped responding to standard treatment options, including carboplatin.

Importantly, the Company is looking to see if this anti-cancer effect with NOX66 can be achieved in combination with a dose of carboplatin low enough to be well tolerated.

Carboplatin is one of the most commonly used chemotherapy drugs in the treatment of solid cancers. Its major adverse side-effect is suppression of bone marrow function, leading to low levels of white blood cells and greater susceptibility to infections. Avoiding this side-effect, which is common to many chemotherapies apart from carboplatin, has been identified as a major need in cancer care, particularly with the growing emergence of antibiotic-resistant superbugs.

Combination therapy
The study is testing NOX66 in combination with 2 dosages of carboplatin. Carboplatin typically is used within a dosage range designated AUC4 (lower end) and AUC6 (higher end). Dosages of AUC5 and AUC6 are more usual when carboplatin is used on its own. A dosage of AUC4, while generally well tolerated in terms of bone marrow function, typically is only marginally effective in most patients when used on its own, usually being reserved for when the drug is used in combination with other chemotherapy drugs.

The combination therapy typically is administered over 6 months in monthly cycles. Each cycle comprises a single intravenous injection of carboplatin each month; NOX66 is administered daily for 14 consecutive days, starting 1 day before each monthly carboplatin injection.

Study design summary
Patients must have late-stage, metastatic solid cancers of the following types: breast, ovarian, lung, prostate, head & neck. Patients must have progressed on standard treatments and have a minimum life-expectancy of 3 months.

Each patient undergoes 3 separate sequential treatment steps:
• Step 1 is a 3-week Run-In arm of NOX66 alone;
• Step 2 is NOX66 in combination with low-dose carboplatin for 3 months;
• Step 3 is NOX66 in combination with high-dose carboplatin for 3 months.

Nineteen patients have been divided into 2 cohorts depending on NOX66 dosage:
Cohort 1 (8 patients); receiving 400 mg NOX66 daily throughout the 3 steps.
Cohort 2 (11 patients); receiving 800 mg NOX66 daily throughout the 3 steps.

Tumour response is being assessed by radiological measurement (RECIST) at the conclusion of Steps 2 and 3 and compared to CT scans done immediately pre-Study.

Study status
The study is fully recruited (19 patients). Two patients dropped out voluntarily during Step 1; one patient was withdrawn due to disease progression after Step 2; one patient was withdrawn due to a severe adverse event in Step 3; one patient has completed all 3 steps and is off-study. All remaining 14 patients continue on-study, with the last patient due to have the 6-month scan in April 2018.

Results: Safety data
No adverse events were observed in Step 1 (NOX66 alone) at either dosage.
No adverse events have been observed in 13 patients who have completed Step 2 (NOX66 + low-dose carboplatin).
A serious adverse event (infusion reaction) occurred in one patient following the first intravenous injection of high-dose carboplatin in Step 3, leading to withdrawal of that patient from the study.

Results: Tumour response data
Of 11 patients with measurable disease (able to be measured by scans) who have been treated with NOX66 and low-dose carboplatin, only 1 of the 11 patients showed disease progressed after 3 months. The results are:
• 9 patients = stable disease (no disease progression)
• 1 patient = partial response
• 1 patient = progressive disease.

Of these 11 patients, 5 received 400 mg NOX66 daily (Cohort 1) and 6 received 800 mg NOX66 daily (Cohort 2). The one case of progressive disease (lung cancer) was in the 400 mg dose cohort, and the one case of partial response (prostate cancer) was in the 800 mg dose cohort. Five Cohort 2 patients are yet to complete Step 2.

Only 1 patient to date has completed Step 3 and continues to show stable disease.

Comment
Noxopharm CEO, Dr Graham Kelly, said, “The clinical team is highly encouraged by this data. NOX66 has proven to be well tolerated both on its own and in combination with low-dose carboplatin. Being able to deliver a meaningful clinical benefit to patients with late-stage cancer in a way that doesn’t add to their burden by exposing them to debilitating or life-threatening side-effects, is a major aim of this Company.”

“But it is the disease status of the patients that is worth noting. The patients coming into this study are all late-stage cancer patients with metastatic disease and with no remaining standard treatment options. So, to see only 1 of 11 patients show disease progression after 3 months of treatment on combined NOX66 and low-dose carboplatin is something that we regard as highly encouraging. We obviously cannot categorically rule out this effect being due to carboplatin alone, but that seems highly unlikely given both the treatment history of these patients and the considerable clinical experience of monthly carboplatin (AUC4) on its own delivering very marginal clinical benefit across most forms of cancer.”

“It also is noteworthy that this effect has been achieved across a range of common cancers. And having achieved this without any serious toxicity essentially means that we appear to be on our way of achieving one of two key aims that this study set out to achieve.” Kelly added.

There are another 5 patients (Cohort 2) yet to complete their 3-months of low-dose carboplatin treatment, and all but 1 patient to complete their 3-months of treatment with high-dose carboplatin early next year.

Kelly added, “The anti-cancer drug scene in recent years has been dominated by the arrival of drugs that target the immune system and which have come to market to considerable acclaim. What is less well understood is that their benefits generally are limited to specific tumour types, with generally relatively modest response rates, and where the response where it does occur generally is of limited duration. All of this with side-effects that can be serious.”

“If the interim outcome we are seeing in this study is confirmed once all data is in, and then confirmed in a larger, controlled study, then we are confident that NOX66 has the potential to become an important new anti-cancer drug in combination with carboplatin, offering hope for patients where little currently exists.”

About the study
Study NOX66-001 is an open-label Phase 1a/Phase 1b study being conducted in Georgia in four FDA-audited clinical sites.

Assessment of tumour response
Tumour response is being assessed by standard RECIST parameters involving CT scans. RECIST (Response Evaluation Criteria in Solid Tumors) identifies up to 5 lesions in total whose longest diameter can be measured. These are termed target lesions; all other lesions are counted but not measured (non-target lesions).
Complete Response: Disappearance of all target and non-target lesions.
Partial Response: At least a 30% decrease in the sum of the longest diameters of target lesions; no new lesions.
Progressive Disease: At least a 20% increase in the sum of the longest diameters of target lesions; or the appearance of 1 or more new lesions.
Stable Disease: Neither sufficient shrinkage to quality for partial response, nor sufficient increase to qualify for progressive disease.

About carboplatin
Carboplatin is a cytotoxic chemotherapy approved for the treatment of breast, ovarian, lung, head & neck, and brain cancer, and neuroblastoma. The dosage normally is expressed as Area Under the Curve (AUC). Side-effects are common, with bone marrow suppression the major one, and nausea, peripheral neuropathy and allergic reactions generally less common.

About NOX66
NOX66 is an innovative dosage formulation of the experimental anti-cancer drug, idronoxil, developed specifically to preserve the anti-cancer activity of idronoxil in the body and to enhance its drug-like behaviour.

Idronoxil is a kinase inhibitor that works by inhibiting a range of enzymes including sphingosine kinase and PI3 kinase that regulate cell pro-survival mechanisms and which are over-expressed in cancer cells, as well as inhibiting external NADH oxidase Type 2 (ENOX 2) which is responsible for maintaining the transmembrane electron potential (TMEP) in the plasma membrane of cancer cells and whose expression is limited to cancer cells. Inhibition of these enzymes results in disruption of key downstream prosurvival mechanisms including resistance mechanisms, sensitizing the cancer cell to the cytotoxic effects of chemotherapy drugs and radiotherapy.

About Noxopharm
Noxopharm is an Australian drug development company with offices in Sydney and Hong Kong. The Company has a primary focus on the development of drugs to address the problem of drug-resistance in cancer cells, the major hurdle facing improved survival prospects for cancer patients. NOX66 is the first pipeline product, with later generation drug candidates under development. The Company also has initiated a pipeline of non-oncology drugs that are held by subsidiary company, Nyrada, Inc.

* The study is conducted by Clinical Accelerator

 

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First Verified Data Release from VAL401 Phase II Clinical Trial

Below, we are re-publishing with permission the press-release issued by ValiSeek on the 28th of September 2017

 ValiRx Plc

(“ValiRx” or the “Company”)

VALISEEK CLINICAL DEVELOPMENT UPDATE

“First Verified Data Release from VAL401 Phase II Clinical Trial”

London, UK., 28 September 2017: ValiRx Plc (AIM: VAL), the clinical stage biotechnology companyis pleased to provide a positive development update on the clinical development of the joint venture between ValiRx and Tangent Reprofiling Limited.

ValiSeek was formed to progress the novel cancer treatment drug, VAL401, into Clinical Efficacy trials for the treatment of lung cancer and other oncology indications.  First dosing in the Phase II clinical trial commenced in October 2016.

This update provides the first formal release of verified clinical data emerging from our trial in Tbilisi, Georgia.  As reported in June 2017, we closed entry to the trial to new patients, and have since collated, verified and analysed the data collected over the first 2 weeks of all patients.

ValiSeek announces that the dosing portion of the trial is complete, with the process of final data collection, regulatory submissions for trial completion and finally for data analysis, is now underway.

First results

The major results from this period include the pharmacokinetic measurements recorded for each patient after a single 2 mg dose of VAL401.  This is carried out by a blood measurement of the active pharmaceutical ingredient in VAL401, Risperidone, and the blood measurements of the known by-product, or metabolite, of Risperidone, 9-hydroxy-Risperidone. 

This analysis has revealed some significant and interesting differences between the absorption and subsequent metabolism of the conventionally formulated Risperidone, which is in line with expectations for our unique formulation.  Importantly, we have demonstrated that this patient population, biologically interacts with the Risperidone in VAL401 to produce 9-hydroxy-Risperidone as expected, demonstrating suitability of our proposed treatment paradigm, as well as demonstrating that our formulation is compatible with drug absorption and behaviour.

The absolute values in this pharmacokinetic analysis allow us great confidence in our future studies, as the blood levels, even at the lowest 2 mg dosage are sufficient to mirror the doses used in the pre-clinical testing models.  As 2 mg doses have been shown to be broadly safe and tolerated in this patient population, this has been nominated as the preferred dose going forwards.

Commercial

ValiSeek CEO, Dr Suzanne Dilly will be attending the Bio-Europe bio-partnering conference in Berlin in November 2017 in order to partake in one to one meeting with other delegates to discuss these emerging results.

Dr Suzy Dilly, CEO of ValiSeek, commented: “It is gratifying to be able to share this early trial data release, and in particular to be able to confirm anticipated dosing levels in preparation for the next trial.  The analytical databases are now being continuously populated, and I anticipate analysis from this final database to be available, on schedule, before year end.”

 

About ValiSeek

ValiSeek Limited (“ValiSeek”) is a joint venture (“JV”) company between ValiRx Plc and Tangent Reprofiling Limited, part of the SEEK Group.  ValiSeek was formed to progress the novel cancer treatment drug, VAL401, through its remaining pre-clinical development and towards Phase II trials for the treatment of lung cancer and other oncology indications.

About ValiRx

ValiRx is a biotechnology oncology focused company specialising in developing novel treatments for cancer and associated biomarkers. It aims to make a significant contribution in “precision” medicine and science, namely to engineer a breakthrough into human health and well-being, through the early detection of cancer and its therapeutic intervention.

* The study is conducted by Clinical Accelerator

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Replicor announces publication of its REP 301 study in HBV / HDV co-infection in The Lancet Gastroenterology & Hepatology

Below, we are re-publishing with permission the press-release issued by Replicor on the 28th of September 2017

MONTREAL, September 28, 2017 – Replicor Inc., a privately held biopharmaceutical company targeting a cure for patients with chronic hepatitis B virus (HBV) and chronic HBV and hepatitis delta virus (HDV) co-infection, announced today the publication of its REP 301 study on the activity of REP 2139 and pegylated interferon α-2a (pegIFN) in The Lancet Gastroenterology & Hepatology (www.replicor.com/science/publications).

The REP 301 study assessed the safety and efficacy of REP 2139 combined with pegIFN in patients with chronic HBV / HDV co-infection.  This study demonstrated several key breakthroughs in this patient population including: REP 2139-mediated HBsAg reduction to below 1IU/mL dramatically potentiates the activity of pegIFN, that profound functional control of both HBV and HDV can be established in a high proportion of patients, that this function control persists for 1 year after therapy was withdrawn and is accompanied by normalization of liver function. The critical importance of HBsAg reduction below 1IU/mL was emphasized by the lack of potentiation of pegIFN in three patients who achieved HBsAg levels as low as 16.4, 5.74 and 1.88 IU/mL during exposure to pegIFN.

Dr. Vaillant, CSO of Replicor commented, “We are pleased to see the results of the REP 301 study published in The Lancet family of journals.  This publication demonstrates a real therapeutic advance for patients with the most aggressive form of viral hepatitis, for which there is currently no effective treatment option”, commented Dr. Andrew Vaillant, CSO of Replicor who added, “we are confident that the breakthrough results we have achieved in the REP 301 study will be significantly improved upon using the more mature REP 2139-based combination regimen currently being validated in the REP 401 protocol.”

About Replicor

Replicor is a privately held biopharmaceutical company with the most advanced animal and human clinical data in the development of the cure for HBV and HDV. The company is dedicated to accelerating the development of an effective treatment for patients with HBV and HBV/HDV co-infection. For further information about Replicor please visit our website at www.replicor.com.

* The study is conducted by Clinical Accelerator

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Engage with your patients and win greater patient engagement

Part of what makes clinical trials efficient and successful are the smart decisions that are made about protocol design, site selection, staff training, technologies used, information provided and so on. Patients’ motivation for enrolment and perception of clinical trials play a key role in this decision making process, and therefore it is imperative that the pharma, biotech and medtech industry professionals have a clear understanding and grasp of it.

That is why Avoca Quality Consortium (AQC) carried out a patient survey which encompassed topics such as:

  • Levels of confidence in the pharmaceutical companies and study teams
  • Reasons for recommending or not recommending clinical trials to a loved one
  • Motivation for participation
  • Patient attitude towards healthcare providers
  • How demographic characteristics and types of illnesses affect patient outlook

A clear understanding of these topics would greatly help pharmaceutical companies, CROs and study teams determine the right actions to surely influence clinical trial participation and improve the impressions given off by clinical trials. In this day and age, many impressive innovations in healthcare have occurred, resulting in patients developing increasingly high expectations of their healthcare and treatment choices. To meet these increasing expectations, smart study decisions and understanding become ever so vital.

The misunderstood

The survey uncovered that on average, patients did not feel that their healthcare providers understood their situation and what it was like to have their condition. Mean ‘perceived understanding’ ratings ranged from 2.9 to 3.4 on a scale of 1 (no understanding) to 5 (very good understanding). The strongest factor influencing the difference of attitude was Age and Marital Status. Those 76 years + and single and married individuals felt most understood, whilst those aged 31-45 or divorced felt the least. Interestingly enough, disease characteristics and history of clinical trial participation had little impact on patients’ thoughts. Many of those who didn’t feel understood stated that they don’t believe medical providers have time to understand them and that they disperse patient care amongst several people resulting in providers only understanding a ‘piece’ of the patient.

Motivation for Participation

In regards to motivation and reasoning for consecutive participation, surprisingly again, type and impact of medical condition had little significance. The top 3 reasons were: to contribute to science; learn more about their condition; and financial considerations. Medical condition as well as demographic characteristics did however impact motivation for initial participation. It was found that those with chronic and degenerative conditions were motivated by additional contact and access to superior medical professionals. Those mildly impacted by their disease were motivated by access to free healthcare and those limitedly impacted by their disease were motivated by payment.

To recommend or not to recommend

The survey showed that 67% of people would recommend clinical trials to a loved one. 29% said they were unsure and 4% said no. Those who would recommend said that it is because trial operations are in more sophisticated institutions and the benefits outweigh the risks. Those who said no exhibited concern about receiving ineffective treatment and placebo. They also declared mistrust and suspicion of the clinical research industry. Clearly then, an area which demands major work is patient confidence.

Confidence

The type of information that patients stated they were or were not confident in came as no surprise. The survey showed that the highest level of confidence was in information regarding the right to withdraw, procedures and possible benefits. The least level of confidence was in treatment alternatives, risks and side effects and doctors’ motivation. A more intriguing discovery was that patients generally had a good level of confidence in compliance, care and ethics of the site study teams. Scepticism came regarding the morality of pharmaceutical companies and the degree to which they cared about the patients. However, they also stated that their confidence increased when their opinions were accounted for during protocol and operational design. Their confidence also increased when there was clear transparency at every level during study implementation.

Although patient interest in treatment options has increased over the years, consideration of clinical trials as a favourable option is still not a popular enough notion. The survey carried out by AQC has shown that a major part of the problem lays in the fact that patients do not feel understood and have little confidence in the integrity of pharmaceutical companies. Clearly the healthcare industry needs to do more to incorporate patient voice in study design and more time must be devoted to developing patient physician relationships and communication.

Anna Nikitina

 

References:

Denise Calaprice-Whitty, Jennifer Byrne, Jeremy Gilbert. Bridging the Gap for Better Patient Engagement. 31 May 2017. 18th August 2017 <http://www.appliedclinicaltrialsonline.com/bridging-gap-better-patient-engagement?pageID=1&gt;.

 

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Referring physicians: the overlooked resource for faster patient enrolment

It is widely known and understood that the no. 1 cause of delays in clinical trials is inefficient patient enrolment. The problem has become widespread and does not show any signs of abating. According to a TUFTS analysis, 48% of investigative sites either under-enroll or fail to enroll subjects. This leads to prolongation of study timelines, delayed marketing authorizations for important drugs and very significant financial losses for pharmaceutical and biopharmaceutical companies.

To rectify the patient enrollment gap, much time and effort is spent on advertisement for participation in clinical trials. The use of traditional and social media in direct-to-patient advertising of clinical trials is well established and popular in areas such as the United States, but the problems regarding delays in enrollment are not ceasing. This clearly indicates that new strategies should be sought out and implemented.

Many sponsors and CROs have tried to increase patient enrolment rates by engaging external physicians. EC/IRB-approved principal investigator (PI) letters to colleagues and other physicians is a well-known example of such attempts. However, in reality, PIs typically lack both time and motivation to fully engage in building and managing a referral network. Even when external physicians respond to a PI’s letter and try to refer patients, without proper organization and support, many of these patients simply never reach the investigative sites.

Some useful data illustrating the current state of engagement of referring healthcare professionals comes from a 2017 Tufts CSDD survey of 2,000 nurses and physicians. It was revealed that 70% and 90% of them are comfortable with discussing clinical trials with their patients but less than 0.2% actually do so. The main reasons for this, according to the Tufts impact report, are that: Physicians and nurses have limited information about the relevant clinical trials, they do not have enough time, and poor communication and co-ordination between the parties involved in the referral process makes it inefficient.

This firmly suggests that supportive strategies should be developed, which will create a system of support for external physicians’ referrals and improve communication between referring physicians, sponsors and CROs, and study investigators.

A new, innovative strategy which targets these problems, is ePatientFinder’s Clinical Trial Exchange™ platform. On the platform, patients’ electronic health records (EHR) and information regarding ongoing trials (filtered by geography and specialty) are matched and brought to the attention of physicians who have suitable patient resources. The patients identified through the system, are invited by physicians for a discussion and personal pre-evaluation which makes sure that the patients who are eventually referred are highly qualified and willing to participate in clinical studies.

We here at Clinical Accelerator and ClinAccel.Net have always valued the role of external physicians in effective patient enrolment campaigns. Having worked in the patient enrolment support field for many years, we have developed an enhanced patient enrolment model involving a large, ever-growing network of referring physicians working in all major therapeutic areas and our PEMs (patient enrolment managers) who identify relevant external physicians, help them with patient identification and manage the whole referral process. They ensure streamlined and effective communication between the referring physicians and study investigators and take care of simple but important tasks such as patients’ transportation to clinical sites and remuneration for their spent time.

Rather than focusing on direct-to-patient advertising campaigns, our focus is on patient information campaigns delivered by our partner physicians who care medically for the patients we seek for enrolment into clinical trials. Our PEMs assist doctors in designing and delivering these patient-oriented campaigns and providing them with IRB-approved supporting materials which are typically used within healthcare institution environments.

The end result of taking full advantage of referring physicians’ untapped potential, is that qualified patients are identified, screened and referred in high numbers and at speed, leading to faster patient recruitment, more compact study timelines and reduced trial costs.

Anna Nikitina.

References:

Poor physician and nurse engagement driving low patient recruitment. Tufts Center for the Study of Drug Development Impact Report.  Jan/Feb, 2017, Vol. 19 No. 1.

 

Posted in ClinAccel.Net, Clinical Accelerator, Cost of Clinical Trials, Patient Enrolment | Leave a comment

Why do rare disease clinical trials face greater challenges and what are some solutions?

Over 6,800 rare diseases such as Haemophilia, Tay Sach’s disease and Gaucher disease are affecting more than 25 million Americans. Still yet, there is limited understanding or knowledge of these debilitating, life threatening and progressive diseases. The Orphan Drug Act defines rare diseases as disorders affecting less than 200,000 people in the United States. Such a small patient population is the exact cause of problems and challenges in patient enrolment.

One requirement for the study of human diseases is an appropriate trial design. Randomization and control are considered essential if efficacy is to be attained and bias to be reduced, but for this a large sample must be acquired. However, due to scattered patient populations, variations among disease sub types and simply by necessity, clinical trials in rare diseases enrol small samples and still yet with difficulty.

This is partly due to the fact that the majority of rare diseases affect children and reduce lifespan. This leads to families being less likely to enrol their child into a study in which they may receive a placebo. Ethical issues may arise too as children are considered a ‘vulnerable’ population. In addition, problems occur if several studies are being carried out simultaneously as enrolment of a patient in one study may cause illegibility in another. As a result of enrolment challenges, it’s calculated that approximately 30% of phase III trials in rare diseases fail.

Clearly, rare diseases require an approach that is tailored to the study goal and specific indications. Two solution that are arguably vital are: in-home Clinical Trial Support and Patient Registries.

In-home Clinical Trial Support

Traditionally, patients are faced with the inconveniency of traveling to sites numerous times during the study. This puts strain on the patients’ budget and induces drop-outs. In-home Clinical Trial Support however takes into account the patients preferences and difficulties. The nurses travel to the patients’ home instead to carry out the clinical tests, PK sampling, drug infusion administration etc. This saves the patients major costs in regards to accommodation and reduces the number of visits to the sites making the concept of enrolment more appealing.

Patient Registries

With rare diseases it is often the case that sufficient data or information is unavailable. Patient registries help improve understanding and expand the knowledge base for companies developing treatments. Patient registries are global online data-repositories encompassing patients’ treatment-related health information which are created by several nations such as the U.S and Europe. They allow the development of communities which are able to share information regarding clinical trials which in turn increases the rate of patient recruitment, reduces enrolment delays, and allows better site planning as wider knowledge on the subject is made available.

The current status in regards to rare diseases is that we have a significant knowledge gap. In order to reduce this, continued collective efforts must be made to improve the clinical trial protocol design which will ultimately reduce time delays, hasten patient recruitment and most importantly, benefit those suffering from rare diseases.

References:

Augustine, Erika F., Heather R. Adams, and Jonathan W. Mink. “Clinical Trials in Rare Disease: Challenges and Opportunities.” Journal of child neurology28.9 (2013): 1142–1150. PMC. Web. 9 June 2017.

Khaleel, Samiya. “Rare Disease Patient Recruitment And Retention.” Clinical Leader. N.p., n.d. Web. 8 June 2017.

Leavy, Michelle, and Richard Gliklich. “Patient Registries and Rare Diseases.” Applied Clinical Trials. N.p., 2011. Web. 8 June 2017.

Stevenson, Danielle. “Clinical Trials for Rare Diseases – Finding and Keeping Patients.” BHD Foundation. N.p., n.d. Web. 8 June 2017.

Tirunagari, Sreedhar. “Rare Disease Clinical Trials‘Patient Recruitment Challenges.’” Global Health Trials. N.p., n.d. Web. 8 June 2017.

Anna Nikitina

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Clinical Accelerator forms ClinAccel.Net

ca-net-logo

Clinical Accelerator forms ClinAccel.Net, a dedicated division focusing on expanding a network of partnering clinical research sites, and fast and efficient patient enrolment into clinical studies

Douglas, February 28, 2017 – Clinical Accelerator, a clinical contract research organization with operations in Central and Eastern Europe announced today the separation of its operations into two divisions. The clinical operations division will continue functioning as Clinical Accelerator, while a new dedicated division operating under the name ClinAccel.Net will be devoted to expanding a regional network of high enrolling investigators and supporting sites, and to designing and implementing proactive, regionally validated strategies of patient enrolment and retention.

Dr Nikolai Nikitin, CEO of Clinical Accelerator, stated, “Through this evolution, we will enable a deeper focus on the hugely important tasks of identifying and enrolling patients into clinical studies. The environment for patient enrolment is becoming ever more competitive throughout the world. It also affects the so-called emerging regions, such as Central and Eastern Europe where competition for patients and investigators has significantly intensified in the recent years.

Patient enrolment and retention support is now an important and well established service in the clinical trial industry and is offered by many international organizations. However, practical strategies often originate in locations with the highest competition for patients, such as the United States, and these strategies are not necessarily as effective in other parts of the world. We have been involved in patient enrolment and retention activities in Central and Eastern Europe for years and have developed and improved our customized, regionally validated models which work in our countries of operation. Through ClinAccel.Net, we plan to continue and further expand these services which we offer to international pharmaceutical and biotech companies, contract research organizations, and to regional clinical research sites.”

About Clinical Accelerator

Clinical Accelerator is an independent clinical trial management organization operating principally in Central and Eastern Europe, Russia, Ukraine and CIS countries. The organization offers a broad range of clinical trial services together with dedicated patient enrolment support to worldwide clients in the pharmaceutical, biotechnology, nutraceutical and medical device industries. Clinical Accelerator’s model of operation is designed to achieve significant cost savings for its clients and to guarantee compact timelines for patient enrolment, with a firm focus on the quality of clinical trial data.

www.clinicalaccelerator.com

About ClinAccel.Net

CliniAccel.Net is a network of investigative and supporting clinical trial sites and a patient enrolment organization with a special focus on the region of Central and Eastern Europe. ClinAccel.Net helps pharmaceutical, biotech and medical technology companies as well as CROs to accelerate implementation of clinical trials through shorter start-up times and through faster patient enrolment. ClinAccel.Net improves efficiency and patient enrolling capacity of clinical research sites by optimizing their systems and procedures, and by employing a dedicated staff of patient enrolment managers who design and implement effective region-specific patient enrolment and retention strategies.

www.clinaccel.net

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Find FDA Documents by Name, Text and Other Keywords

searchOpenTrialsFDA works on making clinical trial data from the FDA (the US Food and Drug Administration) more easily accessible and searchable. Until now, this information has been hidden in the user-unfriendly Drug Approval Packages that the FDA publishes via its dataportal Drugs@FDA. These are often just images of pages, so you cannot even search for a text phrase in them. OpenTrialsFDA scrapes all the relevant data and documents from the FDA documents, runs Optical Character Recognition across all documents and links this information to other clinical trial data.

Explore the public beta version through a new user-friendly web interface at https://fda.opentrials.net.

László Szakács

Posted in FDA | Leave a comment