NCT01071239

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Apr 2009

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2009

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

May 20, 2009

Completed
9 months until next milestone

First Posted

Study publicly available on registry

February 19, 2010

Completed
6.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 30, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 30, 2016

Completed
Last Updated

September 11, 2019

Status Verified

September 1, 2019

Enrollment Period

7.4 years

First QC Date

May 20, 2009

Last Update Submit

September 3, 2019

Conditions

Fanconi Anemia

Keywords

Fanconi AnemiaHematopoietic stem cell transplant

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (1)

  • 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

Study Arms (1)

Bone marrow processing

EXPERIMENTAL

Bone Marrow processing using the CliniMACs device

Device: CliniMACs deviceDrug: BusulfanDrug: FludarabineDrug: CyclophosphamideDrug: ATG

Interventions

CliniMACs deviceDEVICE

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

Also known as: Milteny Biotec CliniMACS device
Bone marrow processing
BusulfanDRUG

Chemotherapy administered as a part of the HSCT conditioning regimen.

Bone marrow processing
FludarabineDRUG

Chemotherapy administered as a part of the HSCT conditioning regimen.

Bone marrow processing
CyclophosphamideDRUG

Chemotherapy administered as a part of the HSCT conditioning regimen.

Bone marrow processing
ATGDRUG

Chemotherapy administered as a part of the HSCT conditioning regimen.

Bone marrow processing

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • Pregnant or breastfeeding.
  • Active CNS leukemic involvement
  • Active uncontrolled viral, bacterial or fungal infection
  • Positive for HIV.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Fanconi Anemia

Interventions

BusulfanfludarabineCyclophosphamide

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, CongenitalAnemia, AplasticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesCongenital Bone Marrow Failure SyndromesBone Marrow Failure DisordersBone Marrow DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Butylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur CompoundsPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedPhosphoramidesOrganophosphorus Compounds

Study Officials

  • David A Margolis, MD

    Medical College of Wisconsin

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor of Pediatrics and BMT Program Director

Study Record Dates

First Submitted

May 20, 2009

First Posted

February 19, 2010

Study Start

April 1, 2009

Primary Completion

August 30, 2016

Study Completion

August 30, 2016

Last Updated

September 11, 2019

Record last verified: 2019-09

Locations

US(1)