As the CMTA’s $23.5 million investment in research begins to return dividends, more and more potential treatments are advancing to the clinical trial stage. Here’s a quick guide to clinical trials and how to be the first to hear about opportunities to participate.

A clinical trial is a scientific study to investigate how safe and effective a new medical treatment is. In a clinical trial, participants are assigned to either receive the new treatment or a placebo treatment for comparison. In the United States, trials are conducted under strict guidelines set out by the Food and Drug Administration and monitored closely by doctors and researchers to ensure safety and accuracy. Clinical trials are an essential step in the development and approval of new treatments for CMT.

Stages of a Clinical Trial

Preclinical Testing:

Preclinical testing involves studies using human or animal cells and CMT animal models to evaluate the safety and effectiveness of a treatment. The CMTA continues to invest in the development of high-quality animal models and patient-derived cell lines—our Toolbox—to provide the resources needed for preclinical studies. We also provide comprehensive expert support through our preclinical testing program and network of established contract research and service partners. If preclinical testing shows promising results and a good safety profile, the next step is a clinical trial in human volunteers.

Phase 1:

The new treatment is tested on a small group of healthy volunteers to evaluate its safety, dosage, and potential side effects. If a potential treatment is unsuitable for healthy volunteers, some trials may use CMT patients.

Phase 2:

The treatment is tested on a larger group of people with CMT to determine its effectiveness and optimal dosage.

Phase 3:

In the final stage before approval, the treatment is tested on a large group of people to confirm its effectiveness, monitor side effects, and compare it to existing treatments.

Phase 4:

After a treatment has been approved and is on the market, further monitoring and research are conducted to evaluate its long-term safety and effectiveness.

It’s worth noting that not all clinical trials follow this exact sequence. Some may have additional phases or different structures depending on the treatment being tested. How long a clinical trial lasts depends on how quickly a measurable difference in symptoms can be detected between those taking the treatment and those taking the placebo.

At every stage, volunteers are closely monitored for effectiveness and potential side effects. Sensitive biomarkers and clinical outcome measures supported by comprehensive natural history study data are key to knowing whether a treatment is working. The CMTA has invested in the development of all these and works closely with CMTA Clinical Center of Excellence sites to support use of these markers, measures and data in current and future clinical trials.

Want to be the first to hear about clinical trials? See our Patients as Partners in Research webpage and create your own profile today.

Supercharge the CMTA’s research efforts and advancement to clinical trials by making a donation to STAR today.

Clinical Trials Jargon Buster

Informed Consent: A process where participants are provided with information about a clinical trial to help them decide whether to participate. This information includes the potential benefits and risks of participating, as well as their rights as a participant.

Placebo: A substance that looks like the treatment being tested but does not contain any active ingredients. Placebos are used as a control group in clinical trials to determine the effectiveness of the treatment being tested.

Double-Blind: A type of clinical trial where neither the participants nor the researchers know who is receiving the treatment being tested or the placebo. This is done to eliminate bias in the study.

Randomized: A type of clinical trial where participants are randomly assigned to either receive the treatment being tested or the placebo. This is done to ensure that the results of the trial are not affected by any pre-existing conditions or biases.

Adverse Event: Any unwanted or unexpected medical event that occurs during a clinical trial, including side effects from the treatment being tested or other health issues that coincidentally occur during the trial.

Protocol: A detailed plan for conducting a clinical trial that outlines the study’s objectives, design, methodology, statistical analysis, and other key aspects.

Endpoint: The specific outcome that the clinical trial is designed to measure, such as reduced disease progression over time, or reduction of symptoms.

Sponsor: The organization or individual who initiates and funds a clinical trial.

Investigator: The person responsible for conducting a clinical trial according to the protocol and regulatory requirements.

IRB (Institutional Review Board): A group of experts who review and approve the protocol for a clinical trial to ensure it is ethical and protects the safety and rights of the participants.