Clinical Trial: Use of ACTIMMUNE in Patients With ADO2

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 Osteopetrosis

Brief Summary: This study is an open label use of ACTIMMUNE for patients with Autosomal Dominant Osteopetrosis Type 2(ADO2). Effects of treatment will be evaluated after 14 weeks on ACTIMMUNE by bone resorption markers. This study will treat 12 patients with ADO2 recruited from Indiana University and Riley Hospital for Children at Indiana University Health.

Detailed Summary:

This is a single center, open-label, dose-escalation study evaluating the efficacy, as defined by biochemical endpoints, and safety profiles of ACTIMMUNE in ADO2 subject.

The investigators will treat 12 ADO2 subjects (children or adults age 3-65) with Actimmune® via a dose escalation protocol to a dose of 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks. If serum CTX does not increase by more than 25% by week 8, the dose will be increased to 100 µg/m2 subcutaneously TIW.

Individual subjects in whom ACTIMMUNE administration increases bone resorption markers during the 14 weeks of this trial will be eligible for a 1 year extension trial.

Sponsor: Indiana University

Current Primary Outcome: Bone resorption markers [ Time Frame: 14 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Bone turnover markers [ Time Frame: 6-12 weeks ]

Original Secondary Outcome: Same as current

Information By: Indiana University

Dates:
Date Received: October 20, 2015
Date Started: January 2016
Date Completion: December 2020
Last Updated: July 12, 2016
Last Verified: July 2016