Use of ACTIMMUNE in Patients With ADO2
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|ClinicalTrials.gov Identifier: NCT02584608|
Recruitment Status : Completed
First Posted : October 22, 2015
Results First Posted : January 8, 2021
Last Update Posted : January 8, 2021
|Condition or disease||Intervention/treatment||Phase|
|Autosomal Dominant Osteopetrosis Type 2||Drug: ACTIMMUNE||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 Osteopetrosis|
|Actual Study Start Date :||January 1, 2016|
|Actual Primary Completion Date :||November 12, 2019|
|Actual Study Completion Date :||November 12, 2019|
ACTIMMUNE 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks
- Changes in Bone Resorption Markers From Baseline to 14 Weeks. [ Time Frame: baseline, 14 weeks ]Evaluate for changes in bone resorption markers including CTX, NTX/creatinine ratio between baseline and 14 weeks
- Change in Bone Turnover Markers Between After Completion of 6-12 Weeks of Treatment [ Time Frame: 6-12 weeks ]Evaluate for changes in bone turnover markers including TRAP5b, NTX, CTX/TRAP5b ratio after 6-12 weeks of treatment.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02584608
|United States, Indiana|
|Indiana University School of Medicine|
|Indianapolis, Indiana, United States, 46202|
|Principal Investigator:||Michael J Econs, MD||Indiana University|