Appointments

866.223.8100

Submit a Form

Questions

866.223.8100

Submit a Form

Expand Content

View Cancer Clinical Trials

FHCRC 2064   |   12-090
(Pediatric) Nonmyeloablative Hematopoietic Cell Transplantation for Patients with Fanconi Anemia Using Alternative Marrow Donors: A Phase I/II Dose-Finding Study

Disease(s)
Blood & Marrow Transplant (BMT)
Fanconi Anemia
Hospital(s)
Main Campus
Phase(s)
Phase 1
Phase 2
Stage(s)
N/A
Type(s)
Therapeutic
Drug(s)
Cyclophosphamide
Cyclosporine
Fludarabine
Mesna
Mycophenolate Mofetil

Contact Information
Cancer Answer Line

866.223.8100

8:00 am - 4:30 pm, Monday - Friday


Description

A. Primary Objectives

  • Identify doses of total body irradiation (TBI) that lead to sufficient probability of donor engraftment (>5% donor CD3 chimerism) by day +200.
  • Evaluate the probability of severe acute graft-versus-host disease.

B. Secondary Objectives

  • Evaluate the probabilities of overall survival, regimen-related toxicity (RRT), and recurrent hematopoietic malignancy in those patients with a prior underlying history of such.
  • Examine the degree to which mixed chimerism provides for amelioration of symptoms (ie, infections due to neutropenia, hemorrhage due to thrombocytopenia) associated with bone marrow failure
  • Determine if the FA complementation group and % initial mosaicism predict engraftment and RRT outcomes

Inclusion Criteria

Patients must meet one of the following criteria (1-4) and 5 to be eligible for this study:

  • Any patient with FA and BM failure involving 2 of the following 3 lineages: granulocyte count <0.5 x 10^9/L, platelet count <20 x 10^9/L, or hemoglobin <8 g/dL.
  • Any patient with FA who requires red blood cell or platelet transfusions because of marrow failure
  • Any patient with FA who has a life-threatening BM failure involving a single hematopoietic lineage.
  • Any patient with FA and pre-existing cytogenetic abnormality including hematopoietic malignancy (AML or MDS) in morphological remission (defined as absence of circulating blasts and bone marrow blasts <5% as assessed by morphology). Note that hematopoietic recovery is not required for remission status.
  • Patients must have a negative cytotoxic crossmatch with donor.

Exclusion Criteria
  • Patients having available HLA-matched related donors.
  • Significant organ dysfunction that would prevent compliance with conditioning, GVHD prophylaxis, or would severely limit the probability of survival, such as liver disease/failure (active hepatitis, moderate to severe portal fibrosis/cirrhosis confirmed by biopsy or uncorrectable hepatic synthetic dysfunction), lung disease, or cardiac disease (ejection fraction <35%, or if unable to obtain ejection fraction, shortening fraction of <26%; if shortening is <26% a cardiology consult is required with PI having final approval of eligibility).
  • HIV seropositive patients
  • Fertile females who are unwilling to use contraceptive techniques during and for the twelve months following treatment, as well as females who are pregnant or actively breast feeding
  • Fertile males who are unwilling to use contraceptive techniques during and for the twelve months following treatment
  • AML/MDS in morphological relapse, defined as having circulating blasts or bone marrow blasts ≥5% as assessed by morphology
  • Active infectious disease concerns
  • Karnofsky performance score <50 or Lansky performance score <40

Cancer Answers & Appointments

Speak with a cancer nurse specialist for appointment assistance and for answers to your questions about cancer locally at 216.444.7923 or toll-free 1.866.223.8100.

Monday through Friday from 8 a.m. – 4:30 p.m. (ET).

Referrals

Resources for medical professionals

  • Outpatient appointment referrals: 216.444.7923 or 866.223.8100
  • Inpatient hospital transfers: 800.553.5056
  • Referring Physician Concierge: 216.444.6196 or 216.312.4910.

Clinical Trials

Search available cancer clinical trials by disease, hospital, phase or number.

This information is provided by Cleveland Clinic and is not intended to replace the medical advice of your doctor or health care provider. Please consult your health care provider for advice about a specific medical condition.

© Copyright 2014 Cleveland Clinic. All rights reserved.