Phase 2 N=5 Treatment

ACTIMMUNE in Intermediate Osteopetrosis

Osteopetrosis

Bottom Line

View on ClinicalTrials.gov: NCT02666768 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Oct 2023

Primary outcome: Primary: Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher — 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Interferon gamma-1b (Drug)
Age: Pediatric, Adult, Older Adult · 1+ yrs
Sex: All
Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Primary completion: Mar 2019

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher	—	—
SECONDARY Percent Change From Baseline in Bone Mineral Density (BMD)	—	—
SECONDARY Change From Baseline in White Blood Cell Count (WBC)	-0.5	—
SECONDARY Change From Baseline in Pain	-0.8	—

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.

Eligibility Criteria

Inclusion Criteria

Diagnosis of osteopetrosis; and
Anemia (Hemoglobin 1 year; and
Ability to travel to a study center for every 3-6 month study visits; and
Patient or parent/legal guardian is able and willing to provide informed consent. For patients 7 to 17 years of age, assent must also be provided.

Exclusion Criteria

12 months or fewer following HCT;
Pregnancy or breastfeeding;
Known or suspected allergy to interferon gamma-1b or related products;
Participation in simultaneous therapeutic study that involves an investigational study drug or agent within 4 weeks of study enrollment;
ALT greater than 3 fold higher than normal; or
Any other social or medical condition that the Investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated or be detrimental to the study.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02666768). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.