Hematopoietic Stem Cell Transplant for Fanconi Anemia

Fanconi Anemia Clinical Trial

— FA  

Official title:

A 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: NCT01071239
• Other study ID #: FA 08/89
• Status: Completed
• Phase: Phase 2
• Start date: April 2009
• Est. completion date: August 30, 2016

Study information

• Verified date: September 2019
• Source: Medical College of Wisconsin
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The trial proposed is a single arm phase II 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 anti-thymocyte globulin (ATG- a non-chemotherapy drug whose role is to kill your immune system) 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 identical donors using stem cell transplants derived from (1) HLA-compatible unrelated donors or (2) HLA haplotype-mismatched related donors.

Description:

We are currently recruiting patients.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1
• Est. completion date: August 30, 2016
• Est. primary completion date: August 30, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Fanconi Anemia (confirmed by mitomycin C or DEB chromosomal breakage testing and one of the following hematological diagnoses: Severe Aplastic Anemia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia

• Karnofsky or Lansy performance scale > or = to 70%.

• Must have adequate cardiac, hepatic, renal and pulmonary function.

• Must have 7/8 or 8/8 available unrelated donor.

Exclusion Criteria:

• Pregnant or breastfeeding.

• Active CNS leukemic involvement

• Active uncontrolled viral, bacterial or fungal infection

• Positive for HIV.

Related Conditions & MeSH terms

• Anemia
• Fanconi Anemia
• Fanconi Syndrome

Intervention

Device:
• CliniMACs device
Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device

Drug:
• Busulfan
Chemotherapy administered as a part of the HSCT conditioning regimen.
• Fludarabine
Chemotherapy administered as a part of the HSCT conditioning regimen.
• Cyclophosphamide
Chemotherapy administered as a part of the HSCT conditioning regimen.
• ATG
Chemotherapy administered as a part of the HSCT conditioning regimen.

Locations

Country	Name	City	State
United States	Medical College of Wisconsin	Milwaukee	Wisconsin

Sponsors (2)

Lead Sponsor	Collaborator
Medical College of Wisconsin	Memorial Sloan Kettering Cancer Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To measure the incidence and quality of engraftment and hematopoietic reconstitution.	To measure the incidence and quality of engraftment and hematopoietic reconstitution.	1, 3, 6 and 12 months post transplant date
Secondary	The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD	The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD	weekly for the first 30 days and then 3, 6, and 12 months post transplant date