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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT00479115 |
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Date of registration:
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23/05/2007 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100
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Scientific title:
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AMD3100 in Combination With G-CSF to Mobilize Peripheral Blood Stem Cells in Patients With Fanconi Anemia(FA): A Phase I/II Study |
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Date of first enrolment:
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May 2007 |
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Target sample size:
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1 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00479115 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Supportive Care. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Stella Davies, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Children's Hospital Medical Center, Cincinnati |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Patients must have had a diagnosis of Fanconi anemia confirmed by a positive test for
increased chromosomal breakage with mitomycin C or diepoxybutane from peripheral
blood, bone marrow or amniotic fluid.
2. Bone marrow biopsy/aspirate with cellularity (mononuclear cells per ml of bone marrow
obtained), CD34+ content (% of MNC), and normal cytogenetics within three months of
collection.
3. For the first two cohorts: Absolute neutrophil count > 750/mm3, Hemoglobin > 8 gm/dl
without transfusion, platelet count > 50,000/mm3 without transfusion (within 30 days
prior to bone marrow collection or PB stem cell mobilization). For the final cohort,
the platelet count will be >30,000/mm3 without transfusion (within 30 days prior to
bone marrow collection or PB stem cell mobilization).
4. Minimum weight: 7.5 kg.
5. Age:
First cohort - > 7 Second cohort - > 3 Third cohort - >1.
6. Ability of patient or parent/legal guardian to consent for bone marrow harvest.
7. Ability of patient or parent/legal guardian to consent for placement of temporary
apheresis catheter.
8. Ability of patient or parent/legal guardian to consent for apheresis collection.
9. Ability of patient or parent/legal guardian to consent for PRBC/platelet transfusions.
Exclusion Criteria:
1. Myeloid or lymphoid leukemia.
2. Clonal cytogenetic abnormality of bone marrow or peripheral blood lymphocytes (in >2
metaphases by G-banded karyotype or any chromosome deletions of chromosome 7 by
Fluorescence in situ hybridization or FISH).
3. Pregnancy or lactation. Women with childbearing potential who are to be collected will
be advised that the marrow harvest procedure or the risk of G-CSF used for stem cell
mobilization may be teratogenic and will be required to take adequate measures to
prevent contraception.
4. Concurrent enrollment in any study using an investigational drug (defined as a drug
not approved by the FDA) with the exception of androgens or thyroxine.
5. Physical or emotional status that would prevent compliance, ability to understand
treatment plan or adequate follow-up.
6. HIV positive patients.
7. Patients with neoplastic or non-neoplastic disease of any major organ system that
would compromise their ability to withstand the bone marrow harvest or apheresis
procedure.
8. Patients with uncontrolled (culture or biopsy positive) infection requiring
intravenous antivirals, antibiotics, or antifungals. Patients on prolonged antifungal
therapy are still eligible if they are culture or biopsy negative in residual
radiographic lesions, and they meet the other organ function criteria.
9. Patients unable to tolerate general anesthesia.
10. Known adverse reaction to E. coli products or G-CSF and any contraindication to
leukocytosis or hypocalcemia or (where indicated) central line placement.
Age minimum:
1 Year
Age maximum:
30 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Fanconi Anemia
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Intervention(s)
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Device: AmCell CliniMACs
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Drug: AMD3100
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Primary Outcome(s)
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Number of Participants Who Safely Received AMD3100 Used in Combination With Standard Dose G-CSF
[Time Frame: 30 days]
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Number of Participants for Whom a Sufficient Number of CD34+ Cells Were Mobilized Into the Peripheral Blood
[Time Frame: 3 days]
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Secondary Outcome(s)
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Number of Participants for Whom the Target Number of CD34+ Cells Were Collected From Their Bone Marrow
[Time Frame: 3 days]
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Number of Participants for Whom the Target Number of Hematopoietic Cells Were Collected by Peripheral Blood Apheresis
[Time Frame: 3 days]
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Number of Participants Whose Product Was Used for Preclinical Biological Investigations.
[Time Frame: 3 days]
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Number of Participants for Whom the Target Number of CD34+ Cells Were Isolated.
[Time Frame: 3 days]
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Secondary ID(s)
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R01HL081499
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CCHMCEH004
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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