Phase 2
N=12
Use of ACTIMMUNE in Patients With ADO2
Autosomal Dominant Osteopetrosis Type 2
Bottom Line
View on ClinicalTrials.gov: NCT02584608 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Jan 2021
Primary outcome: Primary: Changes in Bone Resorption Markers From Baseline to 14 Weeks. — 2.2; -2.1 percentage of change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- ACTIMMUNE (Drug)
- Age
- Pediatric, Adult, Older Adult · 3+ yrs
- Sex
- All
- Sponsor
- Indiana University
- Primary completion
- Nov 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Changes in Bone Resorption Markers From Baseline to 14 Weeks. |
2.2; -2.1 | — |
| SECONDARY Change in Bone Turnover Markers Between After Completion of 6-12 Weeks of Treatment |
-15.34; -2.09; 26.31 | — |
Summary
This study is an open label use of ACTIMMUNE for patients with Autosomal Dominant Osteopetrosis Type 2(ADO2). Effects of treatment will be evaluated after 14 weeks on ACTIMMUNE by bone resorption markers. This study will treat 12 patients with ADO2 recruited from Indiana University and Riley Hospital for Children at Indiana University Health.
Eligibility Criteria
Inclusion Criteria
- Subject is diagnosed with clinically significant ADO2 as determined by the investigator.
Individuals will be screened who have either been diagnosed with osteopetrosis and have a clinical phenotype and/or family history that is consistent with ADO2, have been told that they have an abnormally high bone density (>3SD above mean for age and sex), or a clinical presentation consistent with ADO2. Initial contact will be with members of ADO2 kindreds who have known disease.
- Provide written informed consent for competent adults and for minors provide written assent (if appropriate) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
- Ages 3 to 65 years inclusive.
- Willing to use reliable method of contraception [i.e. oral or patch hormonal contraceptives, intrauterine device, physical barrier methods, tubal ligation or hysterectomy, vasectomy (partner) or abstinence] throughout the study and for 30 days after the last dose of study drug.
Exclusion Criteria
- Any unstable illness that in the investigator's opinion precludes participation in the study.
- Serum calcium >10.6 mg/dl at screening.
- eGFR using the MDRD equation in adults (or the modified Schwartz equation for children) of 2x the upper limit of normal), except when in the opinion of the investigator the liver disease is caused by extra medullary hematopoiesis.
- Pregnant or nursing women or those who plan on becoming pregnant during the study.
Data sourced from ClinicalTrials.gov (NCT02584608). 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.