Cell Therapy for Multiple Myeloma: Vaccination with Dendritic Cell/Myeloma Fusion Post-Transplant for Multiple Myeloma
The BMT CTN 1401 study was designed as a Phase II, multicenter trial of vaccination with Dendritic Cell (DC)/myeloma fusions with granulocyte-macrophage colony-stimulating factor (GM-CSF) adjuvant plus lenalidomide maintenance therapy versus maintenance therapy alone or with GM-CSF following autologous transplant as part of upfront treatment of multiple myeloma. In this whitepaper, we compare the proportion of patients alive and in complete response at one-year post-transplant between patients receiving DC/myeloma vaccine/GM-CSF with lenalidomide maintenance therapy to those receiving lenalidomide maintenance therapy with or without GM-CSF.
What’s Inside:
- Outline of challenges and how they were overcome
- Trial outcome
- Highlights and achievements
- Success factors
- Key Data
Challenges of Conducting Clinical Trials in Rare Hematologic Disorders: A focus on hematopoietic cell transplantation.
Hematopoietic cell transplant (HCT) is a life-saving treatment option for patients with rare blood and marrow disorders. Using blood or marrow cells from a related or unrelated donor is a therapeutic approach that has been undergoing research predating the first HCT performed in 1956. As the HCT field has evolved with further understanding of the biological mechanisms, there remain numerous questions that need to be addressed due to the heterogeneous group of disorders, variety of treatment modalities, and complex relationship of competing outcomes in the post-HCT period.
Research is always improving medical practices, and in a new Whitepaper, we go into detail surrounding one treatment we have been working on since 1995.
What’s Inside:
- Impact of HCT on Patients and Families
- Site Selection and Recruitment
- Flexible Eligibility Criteria
- Trial Monitoring
- Statistical Analysis
- Conclusion
Conducting clinical trials in rare neurological disorders
It is widely known that approximately 300 million people around the world are living with rare conditions. Approximately one third of rare conditions are acknowledged to include neurological components and symptoms due to the condition affecting the number and/or function of nerves. So, even if they are not categorised as a neurological rare condition, patients’ may still cause a wide range of nervous-system derived experiences, including (but not limited to) pain, headaches, seizure-risk, mobility issues, and/or reduced mental capacity.
Rare disease experts with experience in clinical trial design and delivery use their knowledge to build with these conditions in mind. Every child’s experience of their condition is likely to be different, and the breadth of symptoms that rare disease trial participants may experience needs to be acknowledged from the start. In this white paper we will be exploring some of the modifications and approaches needed to ensure that trial participants who experience neuronal effects of their condition have positive experiences in research.What’s Inside:
- Trial practicalities
- Consent vs assent
- Legal representation
- Who makes the decision?
- Remote vs in-clinic visits
- Delivering a trial that lasts
2021: A year in review
2021 has been another year of confusion and contradiction … but it has also been a year of united action by communities, a year when new legislation has been brought forth, and a year when healthcare started to change.As 2021 comes to a close, we’d like to take the time to think back on the year it has been. Not dwell on the ongoing scientific details of Covid-19 itself, but to appreciate some of the many things we have achieved together because of, or despite, the pressures of the pandemic. We truly believe that this year is one we can all be proud of for the way we have supported each other during hardship, built on successes, and made changes needed to achieve even more in 2022.
Please click the link to download the review.
Rare trials you can trust: Biostatistics
Although difficult, it is possible to run high quality trials involving small numbers of people scattered around the globe. For the challenges experienced when running rare disease studies you need an expert team who will work seamlessly together to design and deliver something that fits. There is a group of people who quietly form part of this expert team. These people sit behind trials and provide a pivotal foundation. The term ‘biostatistics’ may not be at the top of news articles, but biostatistics is at the core of every single piece of research. It is the critical element that is needed so that we know how much we can trust the results. Biostatisticians working in the field of rare disease are, themselves, uniquely qualified for the challenges the area brings – people with years of experience who are driven to use their skills every day to help others. This white paper aims to shed light on the difference that biostatisticians specialising in rare conditions bring to this united trials-team.What’s Inside:
- The role of biostatistics.
- The 3 key areas for designing a successful trial to test the benefit-risk of a drug in rare disease:
- Natural history of the condition in question – understanding what you’re seeing and knowing what to measure.
- Constraints of study delivery e.g. small population and study design optimisation, simulations of the trial to optimise trial performance.
- Power to make a difference – address the aspect of disease that matter – be it in terms of quality of life, treatment of a disease, or symptom control.
Key Considerations when Conducting Rare Disease Paediatric Trials
When conducting rare disease paediatric trials, understanding, and integrating the multitude of varied regulations and ethical considerations into the design of the study continues to be essential. However, the experience and protection of children within trials, as well as the quality of data gathered to facilitate approval is dependent upon the operational delivery of the study itself. Within this whitepaper we would like to share with you some of the learning, considerations, and insights that we have gained through many years of experience delivering paediatric trials in rare disease, with the hope that this sparks healthy debate and further roll-out of optimised trials.What’s Inside:
- Discussion around the most efficient way to create a successful protocol.
- Recruitment and retention considerations to take into account when enrolling patients.
- Informed consent processes.
- Example trial communication activities.
- Child-first approach to trial delivery.
Finding the Right Contract Research Organisation in Rare Disease
For sponsors, selection of the right CRO to develop their orphan drug is crucial to the delivery of a successful clinical trial. Plus, the future of the potential medicine for rare disease patients is entirely dependent upon the quality of the trials that are run. Choosing the right CRO is therefore a fundamentally important step towards success and one that should be undertaken carefully. In our latest whitepaper “Finding the Right CRO in Rare Disease”, we discuss what might impact upon this decision-making process...What’s Inside:
- Running good clinical trials within rare disease communities requires a unique skillset. There are many groups who need to collaborate to successfully run trials. In this whitepaper we discuss the role of the CRO in relation to the main groups of people who come together to deliver clinical trials.
- The standard approach to building a clinical trial programme.
- Key considerations to bear in mind when selecting the right CRO partner for your project.
The impact of COVID-19 on clinical research in rare diseases
In our latest whitepaper, The impact of COVID-19 on clinical research in rare diseases, stakeholders can gain insight into the impact that Covid-19 has had across the rare disease sector and the ways in which Orphan Reach have adapted in order to deliver projects successfully throughout a challenging year.What’s Inside:
- Looking back with you and sharing the challenges faced this year, so that we can come together to deliver on the early indicators of benefits that have also been realised during this toughest of years.
- How new ideas and funding have been impacted during a year of virtual interaction.
- The impact on trial initiations due to reallocation of resources to focus on COVID-19 research across the clinical research sector.
- The effect of the pandemic on trials that were ongoing at the start of 2020 and also the impact on the reporting and approvals for trials that were closed or completed this year.
Designing and executing a robust feasibility in rare disease clinical trials
In our new whitepaper, Designing and executing a robust feasibility in rare disease clinical trials, we explore critical factors that should be taken into consideration when conducting feasibilities.What’s Inside:
- Data review, epidemiology and patient identification alongside other pointers that will support the decision-making process with respect to narrowing the country selection and tailoring the feasibility according to protocol requirements.
- Site and investigator selection, including site infrastructure, patient recruitment and retention and data mining and undiagnosed patients.
Planning a rare disease registry
Our new white paper, Planning a Rare Disease Registry, will aid sponsors in evaluating how a registry can impact the success of bringing new orphan treatments to market as well as how to initiate and plan for a rare disease registry.What’s Inside:
- Defining the rare disease registry.
- Initiating and planning using collaboration across stakeholders.
- Protocol structure and planning insights.
- Ethical requirements and standards of practices.
- Cost management strategies.
- Details about scheduling, communication, and both quality and risk management.
How to rescue a clinical trial
In our new whitepaper, How to Rescue a Clinical Trial, sponsors can learn how to evaluate whether their current CRO is delivering within expectations, and if not, how to initiate and manage a successful transition.What’s Inside:
- Evaluation tools to determine whether a failing study should be transitioned to another CRO – either completely or partially.
- Insights for a systematic approach to a timely, cost-effective transition to drive the best outcome.
- Analysis of key functional service areas and associated transition elements in a rescue trial.
Enhancing Recruitment and Retention in Rare Disease Trials
In our new whitepaper, Enhancing Recruitment and Retention in Rare Disease Trials, sponsors gain valuable insights and guidance to improve patient recruitment and retention in rare disease clinical trials.What’s Inside:
- Utilizing patient advocacy groups and forums to effectively reach the “right” patients.
- Data analysis and mining; using real world evidence for specific inclusion/exclusion criteria, patient journeys, site locations and more.
- Leveraging internet, Websites and social media to inform potential patients.
- Development of customized study protocols; including travel and home visit considerations.