Hematopoietic Stem Cell Transplant for Fanconi Anemia

Phase 2

Completed

• Conditions: Fanconi Anemia
• Interventions: Device: CliniMACs device; Drug: Busulfan; Drug: Fludarabine; Drug: Cyclophosphamide; Drug: ATG

• Registration Number: NCT01071239
• Lead Sponsor: Medical College of Wisconsin

Brief 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.

Detailed Description

We are currently recruiting patients.

Study Timeline

• Study Start: 2009-04
• Study First Submitted: 2009-05-20
• Study First Submitted QC: 2010-02-18
• Study First Posted: 2010-02-19
• Primary Completion: 2016-08-30
• Study Completion: 2016-08-30
• Disposition First Submitted: 2018-03-20
• Disposition First Submitted QC: 2018-03-21
• Disposition First Posted: 2018-03-23
• Status Verified: 2019-09
• Last Update Submitted: 2019-09-03
• Last Update Posted: 2019-09-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Bone marrow processing	CliniMACs device	Bone Marrow processing using the CliniMACs device
Bone marrow processing	Busulfan	Bone Marrow processing using the CliniMACs device
Bone marrow processing	Fludarabine	Bone Marrow processing using the CliniMACs device
Bone marrow processing	Cyclophosphamide	Bone Marrow processing using the CliniMACs device
Bone marrow processing	ATG	Bone Marrow processing using the CliniMACs device

Primary Outcome Measures

Name	Time	Method
To measure the incidence and quality of engraftment and hematopoietic reconstitution.	1, 3, 6 and 12 months post transplant date	To measure the incidence and quality of engraftment and hematopoietic reconstitution.

Secondary Outcome Measures

Name	Time	Method
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	The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD

Trial Locations

Locations (1)

Medical College of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States