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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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23 October 2023 |
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Main ID: |
NCT02666768 |
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Date of registration:
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23/06/2015 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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ACTIMMUNE in Intermediate Osteopetrosis
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Scientific title:
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Open-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of Osteopetrosis |
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Date of first enrolment:
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February 22, 2016 |
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Target sample size:
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5 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT02666768 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Lynda E Polgreen, MD, MS |
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Address:
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Telephone:
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Email:
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Affiliation:
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Los Angeles BioMedical Research Center at Harbor-UCLA |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- 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.
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.
Age minimum:
1 Year
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Osteopetrosis
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Intervention(s)
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Drug: Interferon gamma-1b
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Primary Outcome(s)
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Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher
[Time Frame: 12 months]
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Secondary Outcome(s)
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Change From Baseline in White Blood Cell Count (WBC)
[Time Frame: 6 months]
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Percent Change From Baseline in Bone Mineral Density (BMD)
[Time Frame: 6 months]
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Change From Baseline in Pain
[Time Frame: 6 months]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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