Use of ACTIMMUNE in Patients With ADO2

Phase 2 Completed 12 enrolled Indiana University Horizon Pharma Ireland, Ltd., Dublin Ireland · collab

NaSingle-groupOpen-labelTreatment

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.

Timeline

Start: 2016-01-01
Primary completion: 2019-11-12
Completion: 2019-11-12

Outcome

Missed primary endpoint

paper Over 14 weeks, interferon gamma-1b failed to significantly increase bone turnover markers in ADO2 and was poorly tolerated. PMID 30889272 ↗

paper Only 3 of 11 (27.3%) completing subjects achieved the primary outcome of increasing CTX ≥25% above baseline at week 14. PMID 30889272 ↗

Publications

Results Imel EA, Liu Z, Acton D, Coffman M, Gebregziabher N, Tong Y, Econs MJ. Interferon Gamma-1b Does Not Increase Markers of Bone Resorption in Autosomal Dominant Osteopetrosis. J Bone Miner Res. 2019 Aug;34(8):1436-1445. doi: 10.1002/jbmr.3715. Epub 2019 May 13.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Interferon gamma-1b	Protein / enzyme biologic	50 ug/m2	Subcutaneous
Subject	Interferon gamma-1b	Protein / enzyme biologic	100 ug/m2	Subcutaneous