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Use of ACTIMMUNE in Patients With ADO2

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02584608
Recruitment Status : Completed
First Posted : October 22, 2015
Last Update Posted : November 14, 2019
Sponsor:
Collaborator:
Horizon Pharma Ireland, Ltd., Dublin Ireland
Information provided by (Responsible Party):
Michael Econs, Indiana University

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.

Condition or disease Intervention/treatment Phase
Autosomal Dominant Osteopetrosis Type 2 Drug: ACTIMMUNE Phase 2

Detailed Description:

This is a single center, open-label, dose-escalation study evaluating the efficacy, as defined by biochemical endpoints, and safety profiles of ACTIMMUNE in ADO2 subject.

The investigators will treat 12 ADO2 subjects (children or adults age 3-65) with Actimmune® via a dose escalation protocol to a dose of 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks. If serum CTX does not increase by more than 25% by week 8, the dose will be increased to 100 µg/m2 subcutaneously TIW.

Individual subjects in whom ACTIMMUNE administration increases bone resorption markers during the 14 weeks of this trial will be eligible for a 1 year extension trial.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 Osteopetrosis
Actual Study Start Date : January 1, 2016
Actual Primary Completion Date : November 12, 2019
Actual Study Completion Date : November 12, 2019


Arm Intervention/treatment
Experimental: Treatment
ACTIMMUNE 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks
Drug: ACTIMMUNE



Primary Outcome Measures :
  1. Bone resorption markers [ Time Frame: 14 weeks ]
    CTX, NTX/creatinine ratio


Secondary Outcome Measures :
  1. Bone turnover markers [ Time Frame: 6-12 weeks ]
    TRAP5b, NTX, CTX/TRAP5b ratio



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   3 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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.

  2. 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
  3. Ages 3 to 65 years inclusive.
  4. 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:

  1. Any unstable illness that in the investigator's opinion precludes participation in the study.
  2. Serum calcium >10.6 mg/dl at screening.
  3. eGFR using the MDRD equation in adults (or the modified Schwartz equation for children) of < 35 ml/min/1.73m2.
  4. 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:

0 = Normal

  1. = Faint hyperechogenic rim around the sides and tip of the medullary pyramids
  2. = More intense echogenic rim with echoes faintly filling the entire medullary pyramid
  3. = Intense echoes throughout the medullary pyramid
  4. = Solitary focus of echoes at the tip of the medullary pyramid/nephrolithiasis

5. Use of any investigational product (drug or device) within 30 days prior to randomization.

6. Subject reported history of hepatitis C.

7. A recent (past 5 years) history of alcoholism or intravenous drug abuse.

8. History of hypersensitivity to IFN-ɣ or E. coli-derived products.

9. 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.

10. Pregnant or nursing women or those who plan on becoming pregnant during the study.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02584608


Locations
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United States, Indiana
Indiana University School of Medicine
Indianapolis, Indiana, United States, 46202
Sponsors and Collaborators
Indiana University
Horizon Pharma Ireland, Ltd., Dublin Ireland
Investigators
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Principal Investigator: Michael J Econs, MD Indiana University

Publications of Results:
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Responsible Party: Michael Econs, MD, Professor of Endocrinlogy and Metabolism, Indiana University
ClinicalTrials.gov Identifier: NCT02584608    
Other Study ID Numbers: 1510340687
First Posted: October 22, 2015    Key Record Dates
Last Update Posted: November 14, 2019
Last Verified: November 2019
Additional relevant MeSH terms:
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Osteopetrosis
Osteosclerosis
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Interferon-gamma
Antiviral Agents
Anti-Infective Agents