Actively Recruiting
A Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study to Evaluate the Effect of SRP-1005 in Subjects With Huntington's Disease (INSIGHTT)
Led by Sarepta Therapeutics, Inc. · Updated on 2026-06-04
32
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a first-in-human, multi-center clinical trial to study the effects of SRP-1005 in people with Huntington's disease (HD). The trial is designed to evaluate the safety and impact of SRP-1005, a drug given by subcutaneous injection, compared to a placebo. The study includes adult participants aged 21 to 70 years who have a genetically confirmed diagnosis of HD and are at specific disease stages. Participants will be randomly assigned to one of four groups receiving different doses of SRP-1005 or a placebo. The study is double-blind, meaning neither the participants nor the researchers know who receives the drug or placebo. The treatment period includes dose escalation and monitoring to assess the drug's presence in plasma, urine, and cerebrospinal fluid over several weeks. During the study, participants will be monitored for side effects and adverse events from baseline through 24 weeks. Researchers will collect blood, urine, and cerebrospinal fluid samples to measure drug levels and safety. Participants must agree to use effective contraception if applicable, and the study excludes those with certain infections, other neurological illnesses, or conditions that prevent safe lumbar puncture or MRI. The total participation lasts at least 24 weeks with ongoing safety assessments.
CONDITIONS
Brief Title
A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Genetically confirmed diagnosis of huntingtin (HTT) cytosine-adenine-guanine repeat length ≥40
- Classified as Huntington's disease Integrated Staging System (HD-ISS) Stage 2 or Mild Stage 3 at screening
- Age between 21 and 70 years
- If of childbearing potential or with partners of childbearing potential and sexually active, agrees to use a highly effective contraception method during the study and for at least 90 days after
You will not qualify if you...
- Any condition that makes lumbar puncture or MRI unsafe or not feasible
- Presence of other significant neurological or systemic illnesses
- Current, chronic, or active HIV, hepatitis B, or hepatitis C infection
- Recent use of investigational agents or huntingtin-lowering therapies
- Uncontrolled psychiatric illness or substance use disorders
- Pregnancy or breastfeeding
- Other unspecified exclusion criteria as noted by the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 24 weeks
Participants receive SRP-1005 or placebo through subcutaneous injections as part of the study intervention.
Trial Site Locations
Total: 2 locations
1
Pacific Clinical Research Network - Auckland (PCRN Auckland)
Auckland, New Zealand, 622
Not Yet Recruiting
2
New Zealand Brain Research Institute
Christchurch, New Zealand, 8014
Actively Recruiting
Research Team
S
Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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