OsteopetrosisPhase 3NCT00004402
Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
A Phase 3 study testing calcitriol and interferon gamma in people with Osteopetrosis, run by FDA Office of Orphan Products Development.
Trial summary
OBJECTIVES: I. Compare the rate of treatment failure in osteopetrosis patients receiving interferon gamma in combination with calcitriol to the rate of treatment failure in patients receiving calcitriol alone. II. Read more about this study
- Who is it for?
- Ages Up to 10 years
- Conditions the study is for
- Osteopetrosis
- What is being tested?
- calcitriol and interferon gamma
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- Nov 1999to Jun 2000
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 10 years
- All sexes
- No healthy volunteers
- Child
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Biopsy and x-ray confirmed primary osteopetrosis
- Presence of anemia and/or cranial nerve compression
--Prior/Concurrent Therapy--
- Biologic therapy: No prior/concurrent bone marrow transplantation No prior interferon gamma No other investigational biologic agents
- Chemotherapy: No prior/concurrent chemotherapeutic agents for bone marrow transplantation
- Endocrine therapy: Prior/concurrent corticosteroid as supportive therapy allowed
- Radiotherapy: Not specified
- Surgery: At least 5 days since major surgery
- Other: Prior/concurrent calcitriol as supportive therapy allowed Prior/concurrent transfusion as supportive therapy allowed Prior/concurrent dietary therapy allowed
--Patient Characteristics--
- Age: 2 months to 10 years
- Performance status: Not specified
- Life expectancy: At least 6 months
- Hematopoietic: Not specified
- Hepatic: Bilirubin less than 2 mg/dL
- Renal: Creatinine less than 1.5 mg/dL OR Creatinine clearance greater than 50 mL/min
- Pulmonary: No uncorrected airway obstruction
- Other: No active infection requiring intravenous antibiotics No known seizure disorder not related to hypocalcemia No uncorrected hydrocephalus No MRI evidence of cerebral atrophy Must maintain or gain body weight No sleep apnea No thrombocytopenia No massive splenomegaly
About this study
OBJECTIVES: I. Compare the rate of treatment failure in osteopetrosis patients receiving interferon gamma in combination with calcitriol to the rate of treatment failure in patients receiving calcitriol alone.
II. Compare the number of adverse events or clinical manifestations of disease progression occurring in these patients.
III. Assess the effects of interferon gamma on hematopoiesis, cranial nerve function, and rate of infection in these patients.
Read the detailed description
PROTOCOL OUTLINE: This is a randomized, placebo controlled, open label study. Patients are randomized to one of two arms (interferon gamma in combination with calcitriol or calcitriol alone).
Arm I: Patients receive calcitriol once daily. Interferon gamma is administered by subcutaneous injection three times a week.
Arm II: Patients receive calcitriol once daily. Patients may continue treatment in the absence of toxicity and disease progression. If disease progression is diagnosed in the control group, patients will then receive interferon gamma in combination with calcitriol.
Patients are followed every 4 weeks.
- Conditions
- Osteopetrosis
- Being tested
- calcitriol, interferon gamma
- Keywords
- genetic diseases and dysmorphic syndromes, osteopetrosis, rare disease
- Medical subject headings
- Osteopetrosis, Genetic Diseases, Inborn, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
calcitriolDrug
interferon gammaDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study startNov 1999
- Study completionJun 2000
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for OsteopetrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Enrolment
- 30 participants
Sponsor and investigators
- Lead sponsor
- FDA Office of Orphan Products Development
- Sponsor type
- US federal agency
- Collaborators
- Medical University of South Carolina
- Investigators
- L. Lyndon Key, Jr., Study chair, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004402
- Sponsor's protocol ID
- 199/13284
- Other IDs
- MUSC-FDR000768
- Status last verified
- Jan 2001
- First submitted
- 18 Oct 1999
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
- ClinicalTrials.gov ID
- NCT00004402
- Acronym / sponsor ID
- 199/13284
- Conditions
- Osteopetrosis
- Keywords
- genetic diseases and dysmorphic syndromes, osteopetrosis, rare disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants
- Lead sponsor
- FDA Office of Orphan Products DevelopmentUS federal agency
- Collaborators
- Medical University of South Carolina
- Study start
- Nov 1999
- Study completion
- Jun 2000
- First posted
- 19 Oct 1999 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
OBJECTIVES: I. Compare the rate of treatment failure in osteopetrosis patients receiving interferon gamma in combination with calcitriol to the rate of treatment failure in patients receiving calcitriol alone.
II. Compare the number of adverse events or clinical manifestations of disease progression occurring in these patients.
III. Assess the effects of interferon gamma on hematopoiesis, cranial nerve function, and rate of infection in these patients.
Detailed description
PROTOCOL OUTLINE: This is a randomized, placebo controlled, open label study. Patients are randomized to one of two arms (interferon gamma in combination with calcitriol or calcitriol alone).
Arm I: Patients receive calcitriol once daily. Interferon gamma is administered by subcutaneous injection three times a week.
Arm II: Patients receive calcitriol once daily. Patients may continue treatment in the absence of toxicity and disease progression. If disease progression is diagnosed in the control group, patients will then receive interferon gamma in combination with calcitriol.
Patients are followed every 4 weeks.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- Up to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| calcitriol | Drug | – |
| interferon gamma | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
Related trials for Osteopetrosis
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome
RecruitingPhase 1/2
Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO)
Osteopetrosis
RecruitingObservational study
Longitudinal Study of Neurodegenerative Disorders
MLD, Krabbe Disease
RecruitingObservational study
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
RecruitingPhase 2
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
L. Lyndon Key, Jr. study chair
Medical University of South Carolina
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing calcitriol and interferon gamma in people with Osteopetrosis, run by FDA Office of Orphan Products Development.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2015.