A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease
ConditionHuntington's Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age21-70
SponsorSarepta Therapeutics, Inc.
About this trial
This is a first-in-human, multi-center trial studying the effects of SRP-1005 in participants with Huntington's disease (HD).
Eligibility criteria
Qualifiers
Genetically confirmed diagnosis of huntingtin (HTT) cytosine-adenine-guanine repeat length ≥40.
Participant has HD Integrated Staging System (HD-ISS) Stage 2 or Mild Stage 3. At screening, the participant must be classified with the Enroll-HD HD-ISS Modified Calculator as either Stage 2 or Mild Stage 3.
Participants who are of childbearing potential, or with partners of childbearing potential, who are sexually active must agree to use a highly effective method of contraception throughout study participation, and for at least 90 days following the end of study.
Disqualifiers
Any condition that would compromise the safety or feasibility of lumbar puncture or magnetic resonance imaging (MRI).
Presence of other significant neurological or systemic illnesses.
Current, chronic or active human immunodeficiency virus, hepatitis B/C.
Recent use of investigational agents or HTT-lowering therapies.
Trial design
Treatments tested in this trial
- SRP-1005
- Placebo
Treatment groups
32 Participants
are divided into 4 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov