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Procedure

Fludarabine for Osteopetrosis

Phase 2 & 3
Waitlist Available
Led By James Garvin, MD. PhD
Research Sponsored by Columbia University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 30, day 60, day 100, 1 year, 2 years
Awards & highlights

Study Summary

Reduced intensity conditioning followed by allogeneic stem cell transplantation will result in mixed/complete donor chimerism and potentially alter the natural history and outcome of patients with non-malignant diseases.

Eligible Conditions
  • Osteopetrosis
  • Fanconi Anemia
  • Severe Combined Immunodeficiency
  • Bone Marrow Failure

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 30, day 60, day 100, 1 year, 2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 30, day 60, day 100, 1 year, 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
This study is to determine the toxicity of administering a fludarabine/cyclophosphamide (Flu/CY) or busulfan (Bu)/Flu based conditioning regimen followed by allogeneic stem cell transplant.
Secondary outcome measures
To determine metabolic/immune (gene/protein) reconstitution by standard biochemical/PCR assays in patients
To determine the risk of disease progression (including neuropsychological deterioration in patients with metabolic non-malignant diseases) following a Flu/CY or Bu/Flu based conditioning regimen.
To estimate the incidence and severity of GVHD following a Flu/Cy or Bu/Flu based conditioning regimen
+1 more

Trial Design

4Treatment groups
Experimental Treatment
Group I: FludarabineExperimental Treatment1 Intervention
Group II: Cyclophosphamide 40Experimental Treatment1 Intervention
Group III: Cyclophosphamide 30Experimental Treatment1 Intervention
Group IV: Cyclohosphamide 200Experimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fludarabine
FDA approved
Cyclophosphamide
FDA approved

Find a Location

Who is running the clinical trial?

Columbia UniversityLead Sponsor
1,433 Previous Clinical Trials
2,447,759 Total Patients Enrolled
James Garvin, MD. PhDPrincipal InvestigatorColumbia University

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
2002. "Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01019876.
All > Stem Cell Transplant
~2 spots leftby May 2025
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