Conditions: Anemia Liver Disease Hypoxemia
Keywords: anemia HHT Liver disease Hypoxemia Iron deficiency anemia Liver disease with high cardiac output Diffuse pulmonary AVMs with hypoxemia
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 10
Healthy volunteers: no

Brief summary:
The purpose of the study is to evaluate the safety and tolerability of pegylated interferon alpha-2b (PEG-Intron) in patients with severe complications related to Hereditary hemorrhagic telangiectasia (HHT).

Funding Source - FDA Office of Orphan Products Development (OOPD)

Detailed description:
The objective of this study is to evaluate the safety and tolerability of pegylated interferon alpha-2b (PEG-Intron) in patients with severe complications related to Hereditary Hemorrhagic Telangiectasia (HHT). Participants will be randomized to the treatment arm or control arm and then crossed over to the alternate arm at 6 months for the remainder of the 12-month study. Study treatment will consist of weekly subcutaneous injections of pegylated interferon alpha-2b (PEG-Intron), 1 microgram/kilogram/week. Adverse events as well as monitoring and treatment of toxicities will be followed as stated in the protocol. Adverse events will be graded according to the Modified NCI Common Toxicity Criteria. After every five participants have completed one month of treatment, an independent data safety monitoring board will review any adverse events.

Arms:
arm 1: Weekly subcutaneous injection of pegylated interferon alpha2b 1 microgram/kg/week for 6 months, then standard care for 6 months. arm 2: Standard care for 6 months, then weekly subcutaneous injection of pegylated interferon alpha2b 1 microgram/kg/week for 6 months.

Interventions: intervention 1: Pegylated Interferon Alpha2b intervention 2: Standard care
Intervention descriptions: intervention 1: Weekly subcutaneous injection of 1 microgram/kg/week intervention 2: Standard care

Protocol document: