Multicenter Transplant Study for Fanconi Anemia

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2013 by Children's Hospital Medical Center, Cincinnati
Sponsor:
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT01082133
First received: March 5, 2010
Last updated: June 20, 2013
Last verified: June 2013
  Purpose

The trial proposed is a multicenter treatment protocol designed to examine transplant related events in patients with Fanconi anemia who lack matched sib donors have severe aplastic anemia (SAA), or myelodysplastic syndrome(MDS) or acute myelogenous leukemia (AML).


Condition Intervention Phase
Fanconi Anemia
Drug: Chemotherapy Preparative Regimen
Biological: Miltenyi CliniMACS
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Franconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

Resource links provided by NLM:


Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome Measures:
  • The primary objectives are: The incidence and quality of engraftment and hematopoietic reconstitution. The incidence of early transplant related mortality The incidence and severity of acute GvHD (graft versus host disease) and chronic GvHD. [ Time Frame: 1-5 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • The incidence of overall survival and disease free survival over time. [ Time Frame: 5 Years ] [ Designated as safety issue: No ]

Estimated Enrollment: 40
Study Start Date: October 2009
Estimated Study Completion Date: October 2020
Estimated Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Chemotherapy Preparative Regimen
Busulfan 0.6-1.0 mg/Kg/dose IV Q 12 hours X4 Doses Fludarabine 35 mg/m2/dose IV once daily X4 Doses Cyclophosphamide 10 mg/Kg/dose IV once daily X4 Doses Anti-thymocyte globulin - Thymoglobulin 2.5 mg/Kg/dose IV daily X4 Doses
Drug: Chemotherapy Preparative Regimen
Busulfan 0.6-1.0 mg/Kg/dose IV Q 12 hours X4 Doses Fludarabine 35 mg/m2/dose IV once daily X4 Doses Cyclophosphamide 10 mg/Kg/dose IV once daily X4 Doses Anti-thymocyte globulin - Thymoglobulin 2.5 mg/Kg/dose IV daily X4 Doses
Biological: Miltenyi CliniMACS
The CliniMACs device (Miltenyi Biotec, Auburn, CA) will be employed for the CD34 selection procedure. It consists of tubing, bags, and a pair of columns, placed at appropriate locations in the Tubing Set to facilitate the cell selection process.

Detailed Description:

The trial proposed is a single arm phase II multicenter treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine and ATG (Anti-thymocyte globulin)for the treatment of patients with Fanconi anemia who have severe aplastic anemia (SAA), or myelodysplastic syndrome(MDS) or acute myelogenous leukemia (AML), lacking HLA-genotypically (HLA = human leukocyte antigen) identical donors using stem cell transplants derived from HLA-compatible unrelated donors or HLA haplotype-mismatched related donors using Miltenyi's CliniMACS.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Confirmed Diagnosis of Fanconi Anemia
  • One of the following:Severe Aplastic Anemia or Severe Single Lineage Cytopenia; Myelodysplastic Syndrome; Acute Myelogenous Leukemia
  • Unrelated and Related Donors
  • Adequate Physical Function (Cardiac, Hepatic, Renal, Pulmonary)
  • Available for Long-Term Follow Up
  • Performance status >= 70%

Exclusion Criteria:

  • Co-existing medical problems that increase the risk of transplant
  • Active CNS (central nervous system) leukemic involvement
  • Pregnant or Breastfeeding (Females)
  • Active, Uncontrolled Infection
  • HIV/HTLV (Human T-lymphotropic virus)Positive
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01082133

Contacts
Contact: Jamie Wilhelm 513-803-1102 jamie.wilhelm@cchmc.org
Contact: Stephanie Edwards, RN 513-636-9292 stephaniel.edwards@cchmc.org

Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Investigators
Principal Investigator: Stella Davies, MD CCHMC
  More Information

No publications provided

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01082133     History of Changes
Other Study ID Numbers: Multicenter FA SCT
Study First Received: March 5, 2010
Last Updated: June 20, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital Medical Center, Cincinnati:
Fanconi Anemia

Additional relevant MeSH terms:
Anemia
Fanconi Anemia
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Fanconi Syndrome
Hematologic Diseases
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Kidney Diseases
Urologic Diseases
Renal Tubular Transport, Inborn Errors
Metabolism, Inborn Errors
Cyclophosphamide
Fludarabine
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists

ClinicalTrials.gov processed this record on September 18, 2014