ACTIMMUNE in Intermediate Osteopetrosis

Status Completed

Clinical Trial Summary

This study evaluates the effects of ACTIMMUNE (IFN-γ1b) in children and adults with intermediate osteoporosis. All participants will receive treatment with ACTIMMUNE for 12 months. The investigators hypothesize that ACTIMMUNE will be tolerated by participants for the full 12 months and result in decreased disease severity.

Clinical Trial Description

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: 1. 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. 2. Change in immunologic and hematologic function, bone mineral density and osteoclast function, physical function and quality of life. ;

Study Design

Related Conditions & MeSH terms

Osteopetrosis

Clinical Trial Details

NCT number	NCT02666768
Study type	Interventional
Source	Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Contact
Status	Completed
Phase	Phase 2
Start date	February 22, 2016
Completion date	April 11, 2019

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT01087398` - Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis	Phase 2/Phase 3
Terminated	`NCT00638820` - Reduced Intensity AlloTransplant For Osteopetrosis	Phase 2
Completed	`NCT00004402` - Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis	Phase 3
Recruiting	`NCT01019876` - Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases	Phase 2/Phase 3
Terminated	`NCT00145587` - Stem Cell Transplantation for Children Affected With Osteopetrosis	N/A
Completed	`NCT01199094` - Clinical Assessment of Patients With High Bone Mass Due to Mutation in Lrp5	N/A
Completed	`NCT00043329` - Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis	Phase 4
Recruiting	`NCT03333200` - Longitudinal Study of Neurodegenerative Disorders
Terminated	`NCT00145886` - rhPTH Therapy for Low Turnover Bone Fragility	Phase 1
Terminated	`NCT00968864` - T-cell Depleted Alternative Donor Transplantation	Phase 2
Completed	`NCT00730314` - Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells	Phase 1/Phase 2
Active, not recruiting	`NCT03301168` - Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant	Phase 1/Phase 2
Terminated	`NCT02065869` - Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant	Phase 1/Phase 2
No longer available	`NCT01200017` - Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.