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Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
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 up to 2 years
Awards & highlights
All Individual Drugs Already Approved
Approved for 20 Other Conditions
No Placebo-Only Group
Summary
This study proposes the use of a reduced intensity chemotherapy/radiation therapy regimen followed by stem cell transplantation, as compared to standard ablative chemotherapy regimens associated with stem cell transplantation, in a population of patients with non-malignant diseases (non-cancer). Eligible patients will have a non-malignant disease in one of the following four strata: bone marrow failure syndromes, immunodeficiencies, inborn errors of metabolism, or histiocytoses. Patients will be assigned to therapy according to diagnosis. Patients will be stratified by disease into one of four strata and treatment regimens will be based on specific disease criteria and conditions. Although these diseases are non-malignant in name, they are often malignant by nature of the disease progression, treatment and associated complications.
Eligible Conditions
- Fanconi Anemia
- Severe Combined Immune Deficiency
- Malignant Osteopetrosis
- Osteopetrosis
- Severe Combined Immunodeficiency
- Bone Marrow Failure
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 2 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Number of participants with Adverse Events
Secondary study objectives
Immune reconstitution
Incidence of graft versus host disease (GVHD)
Metabolic/Immune reconstitution
+1 moreAwards & Highlights
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 20 Other Conditions
This treatment demonstrated efficacy for 20 other conditions.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
4Treatment groups
Experimental Treatment
Active Control
Group I: Regimen DExperimental Treatment3 Interventions
Includes patients with the following specific diagnosis of: Severe Combined Immune Deficiency Syndrome with no evidence of host NK function (will receive Regimen A) and matched related donor
* Cyclophosphamide (30 mg/kg total dose)
* Fludarabine (90 mg/m2 total dose)
* Rabbit anti-thymocyte globulin (Thymoglobulin)(TMG)\*\*(8 mg/kg total dose)
TMG only in family haploidentical and unrelated donors
Group II: Regimen CExperimental Treatment3 Interventions
Includes patients with any one of the following specific diagnosis: Fanconi Anemia, Dyskeratosis Congenita or Schwachman Diamond Syndrome
* Cyclophosphamide (40 mg/kg total dose)
* Fludarabine (140 mg/m2 total dose)
* Anti-Thymocyte Globulin (horse) (150mg/kg total dose)/TBI 450 cGy
Group III: Regimen BExperimental Treatment3 Interventions
Includes patients with a diagnosis of: Osteopetrosis and Severe Aplastic Anemia with a history of \>10 blood transfusions, or Blackfan-Diamond's anemia, or Chronic Granulomatous Disease, or Wiskott Aldrich Syndrome:
* Cyclophosphamide (200 mg/kg total dose)
* Fludabarine (180 mg/m2 total dose)
* Rabbit Anti-thymocyte Globulin (8mg/kg total dose)
Group IV: Regimen A - Standard Conditioning RegimenActive Control3 Interventions
Standard conditioning regimen for all diseases except those diseases or conditions noted in Regimen B, C and D:
* Fludarabine (180 mg/m2 total dose)
* Busulfan (16 mg/kg less than or equal to 4 yrs and 12.8 mg/ kg \>4 yrs total dose)
* Alemtuzumab (2mg/m2 x 1day, 6mg/m2 x 2 days, 20mg/m2 x 2days)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Rabbit Anti-thymocyte Globulin
2002
Completed Phase 3
~50
Horse Anti-thymocyte Globulin
2002
Completed Phase 3
~40
Fludarabine
FDA approved
Cyclophosphamide
FDA approved
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Who is running the clinical trial?
Columbia UniversityLead Sponsor
1,492 Previous Clinical Trials
2,664,820 Total Patients Enrolled
James Garvin, MD, PhDPrincipal InvestigatorColumbia University
James Garvin, MD. PhDPrincipal InvestigatorColumbia University