Multicenter Transplant Study for Fanconi Anemia

Fanconi Anemia Clinical Trial

Official title:

A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01082133
• Other study ID #: Multicenter FA SCT
• Status: Completed
• Phase: Phase 2
• First received: March 5, 2010
• Last updated: June 28, 2016
• Start date: October 2009
• Est. completion date: July 2015

Study information

• Verified date: June 2016
• Source: Children's Hospital Medical Center, Cincinnati
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 45
• Est. completion date: July 2015
• Est. primary completion date: July 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Fanconi Anemia
• Fanconi Syndrome

Intervention

Drug:
• Chemotherapy
Busulfan 0.4-1.0 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.

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital Medical Center, Cincinnati

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.		1-5 years	Yes
Secondary	The incidence of overall survival and disease free survival over time.		5 Years	No