ACTIMMUNE in Intermediate Osteopetrosis
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|ClinicalTrials.gov Identifier: NCT02666768|
Recruitment Status : Completed
First Posted : January 28, 2016
Last Update Posted : July 2, 2019
|Condition or disease||Intervention/treatment||Phase|
|Osteopetrosis||Drug: Interferon gamma-1b||Phase 2|
Osteopetrosis is a rare inherited metabolic bone disease characterized by impaired osteoclast function resulting in defective bone resorption and generalized high bone mass and mineral density (BMD). In patients with severe disease, this high bone mass compromises bone marrow space leading to marrow failure and frequent infections, along with hepatosplenomegaly from extramedullary hematopoiesis. Currently, the only treatment for individuals with severe forms of osteopetrosis is hematopoietic cell transplantation (HCT), however survival in patients with osteopetrosis treated with HCT is only around 55%. Therefore, this treatment is only indicated in select individuals with life-threatening complications of their disease. Thus additional treatments for osteopetrosis are needed both for individuals who are not candidates for HCT and to prolong the time until HCT is needed.
Interferon gamma (IFN-γ) is a naturally occurring cytokine that has been shown to have anti-microbial and anti-viral immunomodulatory effects, and is a potent stimulator of superoxide anion production which in turn promotes the formation and activation of osteoclasts. Two previous studies of IFN-γ1b in a small group of individuals with osteopetrosis found a decrease in trabecular bone area, an increase in marrow space, a decrease in the number of severe infections requiring antibiotic therapy, and an increase in superoxide generation by granulocyte-macrophage colonies.
Therefore, the investigators will conduct an early phase 2, multi-center, open-label, 12-month clinical trial of ACTIMMUNE (IFN-γ1b) treatment of patients with intermediate osteopetrosis to determine the following:
- The feasibility and tolerability of interferon gamma-1b treatment for 1 year in patients with intermediate osteopetrosis. Specifically, i) the ability to enroll patients, and ii) continued treatment throughout the 1-year observational period.
- Change in immunologic and hematologic function, bone mineral density and osteoclast function, physical function and quality of life.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Open-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of Osteopetrosis|
|Actual Study Start Date :||February 22, 2016|
|Actual Primary Completion Date :||March 11, 2019|
|Actual Study Completion Date :||April 11, 2019|
Experimental: gamma interferon-1b
Gamma interferon-1b 100 mcg SC 3 times weekly for 12 months
Drug: Interferon gamma-1b
gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Other Name: ACTIMMUNE
- Number of participants with treatment related adverse events CTCAE v4.0 Grade 3 or higher [ Time Frame: 12 months ]
- Change in Bone Mineral Density (BMD) [ Time Frame: 12 months ]BMD measured by peripheral quantitative computed tomography (pQCT)
- Change in white blood cell count (WBC) [ Time Frame: 12 months ]
- Change in pain score [ Time Frame: 4 weeks ]Self reported pain intensity over the last 4 weeks
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): NCT02666768
|United States, California|
|Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center|
|Torrance, California, United States, 90502|
|United States, Minnesota|
|University of Minnesota|
|Minneapolis, Minnesota, United States, 55454|
|Principal Investigator:||Lynda E Polgreen, MD, MS||Los Angeles BioMedical Research Center at Harbor-UCLA|