A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
- Conditions
- Hereditary Hemorrhagic Telangiectasia
- Interventions
- Drug: Placebo
- Registration Number
- NCT06659640
- Lead Sponsor
- Alnylam Pharmaceuticals
- 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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 96
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Part A: ALN-6400 ALN-6400 Participants will be administered a single dose of ALN-6400. Part B: ALN-6400 ALN-6400 Participants will be administered multiple doses of ALN-6400. Part A: Placebo Placebo Participants will be administered a single dose of placebo. Part B: Placebo Placebo Participants will be administered multiple doses of placebo.
- Primary Outcome Measures
Name Time Method Part A: Frequency of Adverse Events Up to Week 36 Part B: Frequency of Adverse Events Up to Week 96
- Secondary Outcome Measures
Name Time Method 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 Epistaxis frequency will be assessed using a daily patient epistaxis diary.
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 Intensity-adjusted epistaxis duration will be assessed using a daily patient epistaxis diary.
Part B: Change from Baseline in Epistaxis Severity Score (ESS) Scale Baseline up to Week 96 Validated bleeding scale in HHT scored between 0-10, higher scores indicate worse bleeding.
Part B: Change from Baseline in Epistaxis Duration Baseline up to Week 96 Epistaxis duration will be assessed using a daily patient epistaxis diary.
Part B: Change from Baseline in Epistaxis Intensity Baseline up to Week 96 Epistaxis intensity will be assessed using a daily patient epistaxis diary.
Part B: Change from Baseline in Epistaxis-free Days per Month Baseline up to Week 96 Epistaxis-free days per month will be assessed using a daily patient epistaxis diary.
Part B: Change from Baseline in Hematologic Support Score (HSS) Baseline up to Week 96 The HSS is a quantitative tool designed to longitudinally assess the red blood cells (RBC) and iron supplementation needs of patients with HHT and other chronic bleeding disorders.
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 HHT-specific QoL/PRO will be assessed using the NOSE HHT score. The NOSE HHT is a 29-item patient-reported, clinically validated outcome measure, with total scores ranging continuously from 0 to 4 with higher scores indicating worse scores.
Part B: Change from Baseline in QoL/PRO assessed by Modified Patient Global Impression of Severity (mPGI-S) Score Baseline up to Week 84 HHT-specific QoL/PRO will be assessed using the mPGI-S. The patient will respond to a single question, providing their global impression of change in their overall status and epistaxis experience.
Trial Locations
- Locations (1)
Clinical Trial Site
🇨🇦Montreal, Canada
Clinical Trial Site🇨🇦Montreal, Canada