Use of ACTIMMUNE in Patients With ADO2

NCT02584608 | Completed Phase 2 Results DMC

• 1 other identifier: 1510340687
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Autosomal Dominant Osteopetrosis Type 2

Outcome Measures

Primary Outcomes (1)

• Changes in Bone Resorption Markers From Baseline to 14 Weeks.

  Evaluate for changes in bone resorption markers including CTX, NTX/creatinine ratio between baseline and 14 weeks

  baseline, 14 weeks

Secondary Outcomes (1)

• Change in Bone Turnover Markers Between After Completion of 6-12 Weeks of Treatment

  6-12 weeks

Study Arms (1)

Treatment

EXPERIMENTAL

ACTIMMUNE 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks

Drug: ACTIMMUNE

Interventions

ACTIMMUNE DRUG

Treatment

Eligibility Criteria

• Age: 3 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 \< 35 ml/min/1.73m2.
• Nephrocalcinosis on screening ultrasound Grade 3 or higher \[18\]. Subjects with grade 3 or higher nephrocalcinosis will be excluded because we anticipate that use of study drug will increase bone resorption, resulting in increased urinary calcium excretion, which could, potentially, lead to worsening nephrocalcinosis. The grading scale is listed below:
• = Normal
• = Faint hyperechogenic rim around the sides and tip of the medullary pyramids
• = More intense echogenic rim with echoes faintly filling the entire medullary pyramid
• = Intense echoes throughout the medullary pyramid
• = Solitary focus of echoes at the tip of the medullary pyramid/nephrolithiasis
• \. Use of any investigational product (drug or device) within 30 days prior to randomization.
• \. Subject reported history of hepatitis C.
• \. A recent (past 5 years) history of alcoholism or intravenous drug abuse.
• \. History of hypersensitivity to IFN-ɣ or E. coli-derived products.
• \. History of liver disease as evidenced by laboratory results at Screening (aspartate aminotransferase \[AST\] or alanine aminotransferase \[ALT\] \>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.

Sponsors & Collaborators

• Indiana University: lead
• Horizon Pharma Ireland, Ltd., Dublin Ireland: collaborator

Study Sites (1)

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

Related Publications (1)

• Imel EA, Liu Z, Acton D, Coffman M, Gebregziabher N, Tong Y, Econs MJ. Interferon Gamma-1b Does Not Increase Markers of Bone Resorption in Autosomal Dominant Osteopetrosis. J Bone Miner Res. 2019 Aug;34(8):1436-1445. doi: 10.1002/jbmr.3715. Epub 2019 May 13.

  PMID: 30889272 RESULT

MeSH Terms

Conditions

Osteopetrosis

Interventions

interferon gamma-1b

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Micheal Econs
• Organization: Indiana Univerity School of Medicine

mecons@iu.edu

317-274-1339

Study Officials

• Michael J Econs, MD

  Indiana University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

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: MD, Professor of Endocrinlogy and Metabolism

Study Record Dates

• First Submitted: October 20, 2015
• First Posted: October 22, 2015
• Study Start: January 1, 2016
• Primary Completion: November 12, 2019
• Study Completion: November 12, 2019
• Last Updated: January 8, 2021
• Results First Posted: January 8, 2021

Record last verified: 2020-12

Locations

US (1)