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A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)

ConditionHereditary Hemorrhagic Telangiectasia
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorAlnylam Pharmaceuticals

About this trial

The purpose of this study is to:

* evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers * evaluate the efficacy, safety, tolerability and PD of multiple doses of ALN-6400 in adult patients with HHT

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Is a healthy adult volunteer

Is an adult patient with a clinical diagnosis of HHT

Disqualifiers

Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > upper limit of normal (ULN)

Has known human immunodeficiency virus (HIV) infection; or known current or chronic hepatitis C virus or hepatitis B virus infection

Has an estimated glomerular filtration (eGFR) of <90 mL/min/1.73m² at screening

Has ALT or AST >2×ULN

Trial design

Design model

Parallel

Treatments tested in this trial

  • ALN-6400

    Drug

    ALN-6400 will be administered subcutaneously (SC)

  • Placebo

    Drug

    Placebo will be administered subcutaneously (SC)

Treatment groups

120 Participants
are divided into 4 treatment groups
Group A: Part A: ALN-6400Experimental treatment 1 intervention
Group B: Part A: PlaceboPlacebo comparator 1 intervention
Group C: Part B: ALN-6400Experimental treatment 1 intervention
Group D: Part B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Part A: Frequency of Adverse Events

Time frame
Up to Week 36
2

Part B: Frequency of Adverse Events

Time frame
Up to Week 96

Secondary outcomes

1

Part A: Concentrations of ALN-6400 in Plasma

Time frame
Predose and up to 2 days postdose
2

Part A: Change from Baseline in Plasminogen (PLG) in Plasma Protein Levels

Time frame
Predose and up to Week 36 postdose
3

Part B: Change from Baseline in Plasminogen (PLG) in Plasma Protein Levels

Time frame
Screening and up to Week 96 postdose
4

Part A: Change from Baseline in Plasminogen (PLG) in Plasma Activity Levels

Time frame
Predose and up to Week 36 postdose

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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