How Research Turns into Treatments for HD
You can become involved in finding treatments for Huntington’s disease (HD) that may lead to improvements and potentially a cure by volunteering to help doctors and researchers learn more about HD
Types of Studies
|Clinical Trials (Interventional studies)||studies the effectiveness and safety of potential treatments on people|
|Observational Studies||Observes data or collects information from participants|
Stages of Research
|Pre-clinical||researchers investigate a new idea in their lab usually using animals or cell models. If promising, they apply to the FDA for an Investigational New Drug Application.|
|Clinical||3 Phases of trials to understand the effectiveness and safety of treatments on volunteers.|
|FDA New Drug Application||reviewing and approval leads to the next stage.|
|After FDA Approval||researchers continue to monitor research volunteers for side effects and they submit their safety reports to the FDA.|
Phases of Clinical Trials
|Phase 1||involves healthy volunteers finding out if a drug or therapy is safe and exploring different ways to administer treatments such as pills or injections.|
|Phase 2||involves volunteers with HD to test if the experimental treatment is safe and effective. Some volunteers receive a placebo.|
|Phase 3||given to a larger group of volunteers to learn about safety, effectiveness, and test for different dosage amounts.|
|Informed Consent||explains the purpose of the study, how long it’s expected to last, tests or procedures being done, risks and benefits, and whom to contact for further information.|
|Inclusion/Exclusion Criteria||are the reasons a person can/cannot participate in a trial. Each trial uses different criteria.|
|Open-Label Extension (OLE)||allows eligible participants to take the active drug instead of a placebo if a drug is found to have the potential for benefit.|
|Compassionate Use (Expanded Access)||allows the use of the treatment without FDA approval and outside of the clinical trial if an individual has a life-threatening condition.|
Key Questions to Ask
- What expenses do you cover?
- Do you provide a stipend for travel/food/lodging?
- Do you provide a stipend for my partner?
- Can I bring my children? What accommodations do you have for them? Do you provide a stipend for childcare?
- Do you have a stipend for pet care expenses?
- Do you offer a concierge service?
- What is your policy on open-label extension (OLE)?
- What is your policy on compassionate use?
- If I participate, will I be able to participate in other studies or future studies?
- Are you flexible on dates and times of visits?
- How many visits should I anticipate each month (or year)?
- When will the results from the study be available?
- How long will it take to administer the drug/therapy?
- Can I work/do other activities while receiving the drug/therapy?
- Who can I call between visits if I have questions or concerns?
- Do you provide any professional support such as a social worker or therapist?
- What other questions have other participants asked?