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Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases

NCT01019876 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 38

Last updated 2024-10-31

No results posted yet for this study

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.

Conditions

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

Interventions

DRUG

Fludarabine

DRUG

Cyclophosphamide

DRUG

Busulfan

DRUG

Alemtuzumab

DRUG

Rabbit Anti-thymocyte Globulin

DRUG

Horse Anti-thymocyte Globulin

Sponsors & Collaborators

Principal Investigators

  • James Garvin, MD, PhD · Columbia University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2002-09-30
Primary Completion
2021-09-07
Completion
2021-09-07

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01019876 on ClinicalTrials.gov