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InsigHHT: A Phase 1/2, Randomized, Double-blind, Placebo-controlled Study of Single and Multiple Doses of ALN-6400 in Healthy Adults and Patients With Hereditary Hemorrhagic Telangiectasia
Led by Alnylam Pharmaceuticals · Updated on 2026-05-14
120
Participants Needed
16
Research Sites
24 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating ALN-6400 in a combined Phase 1/2 clinical trial involving healthy adult volunteers and adult patients with Hereditary Hemorrhagic Telangiectasia (HHT). The study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of single ascending doses of ALN-6400 in healthy volunteers and multiple doses in patients with HHT. This investigation also explores the efficacy of ALN-6400 in managing symptoms related to HHT. The study is divided into two parts: Part A includes healthy volunteers receiving a single dose of ALN-6400 or a placebo, and Part B involves patients with HHT receiving multiple doses of ALN-6400 or placebo. Both treatments are administered subcutaneously. Participants are randomly assigned to either the experimental or placebo groups, and the study is triple-blinded to ensure unbiased results. Participants will be monitored for safety through the frequency of adverse events up to 36 weeks in healthy volunteers and up to 96 weeks in patients with HHT. Researchers will measure drug concentrations in plasma, changes in plasminogen protein and activity levels, and various clinical outcomes related to epistaxis severity and frequency, hematologic support, and quality of life using patient-reported outcomes. The study includes long-term follow-up assessments to track these parameters throughout the study period.
CONDITIONS
Brief Title
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Part A: Healthy adult volunteers
- Part B: Adult patients with a clinical diagnosis of Hereditary Hemorrhagic Telangiectasia (HHT)
- Willingness to comply with contraceptive requirements during the study period
You will not qualify if you...
- Part A: Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) above the upper limit of normal
- Part A: Known infection with HIV, hepatitis B, or hepatitis C
- Part A: Estimated glomerular filtration rate (eGFR) below 90 mL/min/1.73m² at screening
- Part B: ALT or AST greater than 2 times the upper limit of normal
- Part B: Total bilirubin above 1.5 times the upper limit of normal
- Part B: eGFR below 30 mL/min/1.73m² at screening
- Not willing to comply with contraceptive requirements during the study period in both Parts A and B
- Other protocol-defined criteria apply as per study guidelines
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.
1 screening visit (in-person)
Duration - Up to 36 weeks for Part A and up to 96 weeks for Part B
Participants receive either single or multiple subcutaneous doses of ALN-6400 or placebo depending on their study part.
1 dosing visit for single-dose participants and multiple dosing visits for multiple-dose participants
Duration - Up to 96 weeks postdose
Participants are monitored for safety and changes in clinical measures up to 96 weeks after dosing.
Regular follow-up visits during the monitoring period
Trial Site Locations
Total: 16 locations
1
Clinical Trial Site
Birmingham, Alabama, United States, 35233
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2
Clinical Trial Site
Cypress, California, United States, 90630
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3
Clinical Trial Site
Gainesville, Florida, United States, 32608
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4
Clinical Trial Site
Indianapolis, Indiana, United States, 46260
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5
Clinical Trial Site
Boston, Massachusetts, United States, 02114
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6
Clinical Trial Site
Rochester, Minnesota, United States, 55905
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7
Clinical Trial Site
St Louis, Missouri, United States, 63110
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8
Clinical Trial Site
Chapel Hill, North Carolina, United States, 27514
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9
Clinical Trial Site
Camperdown, Australia, 2050
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10
Clinical Trial Site
Parkville, Australia, 3050
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11
Clinical Trial Site
Mount Royal, Canada, H3P 3P1
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12
Clinical Trial Site
Toronto, Canada, M5B 1W8
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13
Clinical Trial Site
Bordeaux, France, 33000
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14
Clinical Trial Site
Bron, France, 69500
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15
Clinical Trial Site
Homburg, Germany, 66421
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16
Clinical Trial Site
L'Hospitalet de Llobregat, Spain, 8907
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Research Team
A
Alnylam Clinical Trial Information Line
A
Alnylam Clinical Trial Information Line
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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