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)

Condition studied

Hereditary Hemorrhagic Telangiectasia (HHT)

Investigational medicinal product(s)

Eligibility

Primary endpoint

Frequency of AEs. Safety will also be evaluated through vital signs, ECGs, and clinical laboratory assessments.

Endpoint detail

Change from baseline in PLG o Plasma activity levels o Plasma protein levels, Change from baseline in epistaxis o Intensity-adjusted epistaxis duration o ESS o Epistaxis duration o Epistaxis frequency o Epistaxis intensity o Epistaxis-free days per month, • Change from baseline in hematologic parameters o HSS o Iron infusions o RBC transfusions o Hemoglobin, • Change from baseline in QoL/PRO scores o NOSE HHT o mPGI-S