InsigHHT: A Phase 1/2 Study to Evaluate ALN-6400 in Adult Healthy Volunteers and Adult Patients with HHT

You may be able to join if

• Age and Sex 1. Age ≥18 years, at the time of initial informed consent.
• Patient and Disease Characteristics 2. Clinical diagnosis of HHT, either per criterion 2a or 2b: a. Definite diagnosis of HHT as per Curaçao criteria[Shovlin 2000], ie, at least 3 of the 4 criteria: • Recurrent and spontaneous epistaxis. • Multiple telangiectasias on the skin of the hands, lips, face, or inside of the nose or mouth. • Arteriovenous malformations (AVMs) or telangiectasias in 1 or more internal organs, including the lungs, brain, liver, intestines, stomach, and spinal cord. • Family history of HHT (ie, first-degree relative [brother, sister, parent, or child] meeting these same criteria for “definite HHT”). b. Genetic confirmation of HHT (with recurrent and spontaneous epistaxis)
• 3. Epistaxis severity score (ESS) ≥4 (ie, moderate or severe) measured at screening to assess symptoms and bleeding over the prior 4 weeks.
• 4. Iron deficiency anemia (ie, hemoglobin <12.0 g/dL in women and <13.0 g/dL in men) at screening; and/or treatment with ≥250 mg of intravenous iron and/or ≥1 red blood cell (RBC) transfusion within 24 weeks of screening.
• 5. Patient agrees not to undergo procedural or surgical management of nasal telangiectasias/epistaxis (except for emergent situations in which it is required) and agrees not to start new therapies for bleeding in HHT during the DB Treatment Period; during the OLE Period, such treatments and therapies are discouraged but not prohibited. Routine nasal packing is not considered procedural management of epistaxis.
• Informed Consent 6. Patient is able to understand and is willing and able to comply with the study requirements, including completing the daily patient epistaxis diary (including ≥1 week during screening) and to provide written informed consent.