NCT06659640
Recruiting
Phase 1
InsigHHT: A Phase 1/2, Randomized, Double-blind, Placebo-controlled, 2-part Study of the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Single Dose ALN-6400 in Adult Healthy Volunteers and Multiple Dose ALN-6400 in Adult Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
Overview
- Phase
- Phase 1
- Intervention
- ALN-6400
- Conditions
- Not specified
- Sponsor
- Alnylam Pharmaceuticals
- Enrollment
- 120
- Locations
- 16
- Primary Endpoint
- Part A: Frequency of Adverse Events
- Status
- Recruiting
- Last Updated
- yesterday
Overview
Brief Summary
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
Investigators
Eligibility Criteria
Inclusion Criteria
- •Is a healthy adult volunteer
- •Is an adult patient with a clinical diagnosis of HHT
Exclusion Criteria
- •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/1.73m\^2 at screening
- •Has ALT or AST \>2×ULN
- •Has total bilirubin \>1.5×ULN
- •Has eGFR of \<30 mL/min/1.73m\^2 at screening
- •Parts A and B:
- •Is not willing to comply with the contraceptive requirements during the study period
- •Note: other protocol defined inclusion / exclusion criteria apply
Arms & Interventions
Part A: ALN-6400
Participants will be administered a single dose of ALN-6400.
Intervention: ALN-6400
Part A: Placebo
Participants will be administered a single dose of placebo.
Intervention: Placebo
Part B: ALN-6400
Participants will be administered multiple doses of ALN-6400.
Intervention: ALN-6400
Part B: Placebo
Participants will be administered multiple doses of placebo.
Intervention: Placebo
Outcomes
Primary Outcomes
Part A: Frequency of Adverse Events
Time Frame: Up to Week 36
Part B: Frequency of Adverse Events
Time Frame: Up to Week 96
Secondary Outcomes
- Part A: Concentrations of ALN-6400 in Plasma(Predose and up to 2 days postdose)
- Part B: Change from Baseline in Plasminogen (PLG) in Plasma Protein Levels(Screening and up to Week 96 postdose)
- Part A: Change from Baseline in Plasminogen (PLG) in Plasma Activity Levels(Predose and up to Week 36 postdose)
- Part B: Change from Baseline in Plasminogen (PLG) in Plasma Activity Levels(Part B: Screening and up to Week 96 postdose)
- Part B: Change from Baseline in Epistaxis Frequency(Baseline up to Week 96)
- Part B: Change from Baseline in Iron Infusions(Baseline up to Week 96)
- Part A: Change from Baseline in Plasminogen (PLG) in Plasma Protein Levels(Predose and up to Week 36 postdose)
- Part B: Change from Baseline in Intensity-adjusted Epistaxis Duration(Baseline up to Week 96)
- Part B: Change from Baseline in Epistaxis Severity Score (ESS) Scale(Baseline up to Week 96)
- Part B: Change from Baseline in Epistaxis Duration(Baseline up to Week 96)
- Part B: Change from Baseline in Epistaxis Intensity(Baseline up to Week 96)
- Part B: Change from Baseline in Epistaxis-free Days per Month(Baseline up to Week 96)
- Part B: Change from Baseline in Hematologic Support Score (HSS)(Baseline up to Week 96)
- Part B: Change from Baseline in Red Blood Cell (RBC) Infusions(Baseline up to Week 96)
- Part B: Change from Baseline in Hemoglobin(Baseline up to Week 96)
- Part B: Change from Baseline in Quality of Life Patient-reported Outcomes (QoL/PRO) assessed by Nasal Outcome Score for Epistaxis in Hereditary Hemorrhagic Telangiectasia (NOSE HHT) Score(Baseline up to Week 84)
- Part B: Change from Baseline in QoL/PRO assessed by Modified Patient Global Impression of Severity (mPGI-S) Score(Baseline up to Week 84)
Study Sites (16)
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