Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00590460
• Date of registration: 26/12/2007
• Prospective Registration: No
• Primary sponsor: Baylor College of Medicine
• Public title: Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia Mafia
• Scientific title: Cd45 (Yth-24 and Yth 54) and Cd52 (Campath-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Donor Stem Cell Transplantation of Patients With Fanconi Anemia
• Date of first enrolment: July 2001
• Target sample size: 5
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00590460
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Malcolm Brenner, M.B., Ph.D.,
• Affiliation: Baylor College of Medicine

Key inclusion & exclusion criteria

Inclusion Criteria:

Diagnosis of Fanconi Anemia or other suspected DNA breakage/chromosomal instability
syndromes, such as dyskeratosis congenita or Nijmegen breakage syndrome of all ages are
eligible.

Diagnosis of Fanconi anemia confirmed by studies of peripheral blood or bone marrow
sensitivity to mitomycin C or DEB or clinical evidence of other DNA breakage/chromosomal
instability syndrome as determined by genetic testing or clinical diagnosis by a geneticist

Severe aplasia anemia as evidenced by a hypocellular bone marrow and at least 1 of the 3
criteria below: ANC < 500/mm3 Hemoglobin < 10 gm/dl with reticulocyte count < 1% Platelet
count < 50,000/mm3

Availability of an HLA matched or mismatched (up to one haplotype) family member who has
been documented not to have Fanconi anemia or of an unrelated HLA matched stem cell donor.
Fully matched is defined at 6/6 match by high resolution DR based DNA typing.

Life expectancy greater than 6 weeks limited by diseases other than FA

Creatinine 2X normal for age or less

Karnofsky score 70% or more

Exclusion Criteria:

Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by
echocardiogram (i.e., shortening fraction less than 25%).

Patients with known allergy to rat serum products.

Patients with a severe infection that on evaluation by the Principal Investigator precludes
ablative chemotherapy or successful transplantation.

Patients with severe personality disorder or mental illness.

Patients with documented HIV positivity.

Pregnant

NOTE: Patients who would be excluded from the protocol strictly for laboratory
abnormalities can be included at the investigator's discretion after approval by the CCGT
Protocol Review Committee and the FDA Reviewer.



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fanconi Anemia

Severe Aplastic Anemia

Intervention(s)

Procedure: Stem cell infusion

Biological: CAMPATH-1H

Drug: Fludarabine

Biological: Anti-CD45

Primary Outcome(s)

Number of Patients With Donor Engraftment [Time Frame: 100 Days]

Secondary Outcome(s)

Number of Patients Alive at 1 Year Post Transplant [Time Frame: 1 year]

Patients With Extensive Chronic GVHD From Day 100 to 365 [Time Frame: 365 days]

Patients With Grade III - IV Acute GVHD [Time Frame: 100 days]

Patients With Limited Chronic GVHD From Day 100 to 365 [Time Frame: 365 days]

Patients With Grade II - IV Acute Graft Versus Host Disease (GVHD) [Time Frame: 100 days]

Days to Platelet Count of 20,000/mm3 Without Transfusions [Time Frame: 30 Days]

Patients With Treated Related Death [Time Frame: 100 days]

Days to Absolute Neutrophil Count (ANC) of 500/mm3 [Time Frame: 30 Days]

Number of Patients With Graft Failure [Time Frame: 100 days]

Secondary ID(s)

H-9938

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Center for Cell and Gene Therapy, Baylor College of Medicine

The Methodist Hospital System

Results

• Results available: Yes
• Date Posted: 09/08/2012
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00590460