ACTIMMUNE in Intermediate Osteopetrosis (NCT02666768) | Clinical Trial Compass
CompletedPhase 2
ACTIMMUNE in Intermediate Osteopetrosis
United States5 participantsStarted 2016-02-22
Plain-language 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.
Who can participate
Age range
1 Year
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher
Timeframe: 12 months
Trial details
NCT IDNCT02666768
SponsorLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center