alnylam/studies/attachments/ALN-6400-001_Protocol_Plain_Language_Summary_English.pdf

English (United States)

alnylam/studies/attachments/ALN-6400-001_Protocol_Plain_Language_Summary_French_FRA.pdf

French (France)

alnylam/studies/attachments/ALN-6400-001_Protocol_Plain_Language_Summary_German_DEU.pdf

German (Germany)

alnylam/studies/attachments/ALN-6400-001_Protocol_Plain_Language_Summary_Spanish_ESP.pdf

Spanish (Spain)

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

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

Hereditary Hemorrhagic Telangiectasia

HEMATOLOGY

Placebo

ALN-6400

Clinical Trial Site

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)

Intensity-adjusted epistaxis duration will be assessed using a daily patient epistaxis diary.

Validated bleeding scale in HHT scored between 0-10, higher scores indicate worse bleeding.

Epistaxis duration will be assessed using a daily patient epistaxis diary.

Epistaxis frequency will be assessed using a daily patient epistaxis diary.

Epistaxis intensity will be assessed using a daily patient epistaxis diary.

Epistaxis-free days per month will be assessed using a daily patient epistaxis diary.

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.

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.

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.

Participants will be administered a single dose of ALN-6400.

Participants will be administered a single dose of placebo.

Participants will be administered multiple doses of ALN-6400.

Participants will be administered multiple doses of placebo.