ACTIMMUNE in Intermediate Osteopetrosis

NCT02666768 | Completed Phase 2 Results

• 1 other identifier: 21549-01
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Osteopetrosis

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher

  Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 defines a Grade 4 event as having life-threatening consequences, and/or urgent intervention indicated.

  12 months

Secondary Outcomes (3)

• Percent Change From Baseline in Bone Mineral Density (BMD)

  6 months

• Change From Baseline in White Blood Cell Count (WBC)

  6 months

• Change From Baseline in Pain

  6 months

Study Arms (1)

gamma interferon-1b

EXPERIMENTAL

Gamma interferon-1b 100 mcg subcutaneous (SC) 3 times weekly for 12 months

Drug: Interferon gamma-1b

Interventions

Interferon gamma-1b DRUG

gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly

Also known as: ACTIMMUNE

gamma interferon-1b

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of osteopetrosis; and
• Anemia (Hemoglobin \<12 g/dL) not related to iron deficiency, or
• Neutropenia (Neutrophil count \<1000 neutrophils/ul unsupported with cytokines), or
• Thrombocytopenia (Platelet count \<50,000 cells x 109/L), or
• History of impaired bone healing, or
• ≥ 1 serious infection over prior year defined as requiring hospitalization and/or IV antibiotics, and
• Age \> 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center: lead
• University of Minnesota: collaborator
• Horizon Pharma Ireland, Ltd., Dublin Ireland: collaborator

Study Sites (2)

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Torrance, California, 90502, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55454, United States

Location

Related Publications (1)

• Lertwilaiwittaya P, Suktitipat B, Khongthon P, Pongsapich W, Limwongse C, Pithukpakorn M. Identification of novel mutation in RANKL by whole-exome sequencing in a Thai family with osteopetrosis; a case report and review of RANKL osteopetrosis. Mol Genet Genomic Med. 2021 Jul;9(7):e1727. doi: 10.1002/mgg3.1727. Epub 2021 May 30.

  PMID: 34056870 DERIVED

MeSH Terms

Conditions

Osteopetrosis

Interventions

interferon gamma-1b

Condition Hierarchy (Ancestors)

Osteosclerosis; Osteochondrodysplasias; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases

Limitations and Caveats

Limitations of this study include the small sample size, heterogeneity of the population, and difficulty obtaining endpoint measurements due to high withdrawal rate, all resulting in limited data for interpretation. Despite these limitations, this study provides information on the longest follow-up of treatment with interferon gamma-1b in this population and identified an approach to make the treatment tolerable.

Results Point of Contact

• Title: Dr. Lynda Polgreen
• Organization: The Lundquist Institute at Harbor-UCLA Medical Center

lpolgreen@lundquist.org

310-222-1961

Study Officials

• Lynda E Polgreen, MD, MS

  Los Angeles BioMedical Research Center at Harbor-UCLA

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 23, 2015
• First Posted: January 28, 2016
• Study Start: February 22, 2016
• Primary Completion: March 11, 2019
• Study Completion: April 11, 2019
• Last Updated: October 22, 2024
• Results First Posted: October 11, 2023

Record last verified: 2024-10

Locations

US (2)