Health Trials

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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

  • SRP-1005

    Drug

    Subcutaneous injection

  • Placebo

    Drug

    Subcutaneous injection

Treatment groups

32 Participants
are divided into 4 treatment groups
Group A: Cohort 1: SRP-1005 Dose 1Experimental treatment 2 interventions
Group B: Cohort 2: SRP-1005 Dose 2Experimental treatment 2 interventions
Group C: Cohort 3: SRP-1005 Dose 3Experimental treatment 2 interventions
Group D: Cohort 4: SRP-1005 Dose 4Experimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)

Time frame
Baseline through Week 24

Secondary outcomes

1

Plasma Concentration of SRP-1005

Time frame
Baseline through Day 17
2

Urine Concentration of SRP-1005

Time frame
Baseline through Day 17
3

Cerebrospinal Fluid Concentration of SRP-1005

Time frame
Baseline through Week 24

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Sarepta Therapeutics, Inc.

Lead sponsor

Source ClinicalTrials.gov
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