Health TrialsbyHealthPanel

A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease

ConditionHuntington's Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age25-70
SponsorAlnylam Pharmaceuticals

About this trial

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Has stage 2 or early Stage 3 Huntington's disease (HD), per the Huntington's Disease Integrated Staging System (HD-ISS)

Disqualifiers

Has significant structural or degenerative neurologic disease other than Huntington's Disease (HD) at screening

Has primary or secondary immune compromise at screening due to infections, medical conditions, or chronic therapies

Has alanine aminotransferase or aspartate aminotransferase >2× upper limit of normal (ULN)

Has an estimated glomerular filtration rate (eGFR) of <60 mL/min/1.73m² at screening

Trial design

Design model

Parallel

Treatments tested in this trial

  • ALN-HTT02

    Drug

    ALN-HTT02 will be administered intrathecally

  • Placebo

    Drug

    Placebo will be administered intrathecally

Treatment groups

66 Participants
are divided into 2 treatment groups
Group A: ALN-HTT02Experimental treatment 1 intervention
Group B: Placebo + ALN-HTT02Placebo comparator 2 interventions

Trial outcomes

Primary outcomes

1

Frequency of Adverse Events (AEs) in the Double-blind Part of the Study

Time frame
Up to 12 months
2

Frequency of AEs in the Open-label Part of the Study

Time frame
Up to 12 months
3

Frequency of AEs in the OLE Part of the Study

Time frame
Up to 36 months

Secondary outcomes

1

Change from Baseline in Levels of Mutant Huntingtin (mHTT) in Cerebrospinal Fluid (CSF)

Time frame
Baseline up to Month 12 in the Double-blind Part of the study; Baseline up to Month 12 in the Open-label Part of the study ; Baseline up to Month 36 in the OLE Part of the study
2

Concentrations of ALN-HTT02 in Plasma

Area Under the Plasma Concentration-time Curve (AUC) Maximum Observed Plasma Concentration (Cmax) Time to Maximum Plasma Concentration (Tmax)

Time frame
Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study; Up to Month 36 in the OLE Part of the study
3

Concentrations of ALN-HTT02 in Cerebrospinal Fluid (CSF)

Time frame
Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study ; Up to Month 36 in the OLE Part of the study
4

Concentrations of ALN-HTT02 in Urine

Fractional Excretion of ALN-HTT02

Time frame
Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study; Up to Month 36 in the OLE Part of the study

Other outcomes

Sponsors and contacts

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

Alnylam Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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