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Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00479115
Recruitment Status : Completed
First Posted : May 25, 2007
Results First Posted : October 30, 2020
Last Update Posted : October 30, 2020
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

Brief Summary:

The purpose of this research study is to determine whether an experimental drug called AMD3100 used in combination with another medication called G-CSF is safe and can help to increase the amount of blood stem cells (called CD34+ stem cells) found in the peripheral blood of patients with Fanconi anemia. While AMD3100 has been used successfully in adult volunteers and cancer patients, it has not been used in children or patients with Fanconi anemia and in only a few children with cancer.

Fanconi anemia is a rare genetic disease. Most Fanconi anemia patients eventually develop bone marrow failure, a condition in which the bone marrow no longer produces red blood cells (to carry oxygen), white blood cells (to fight infection), and platelets (to help blood clot). The only successful treatment for patients with Fanconi anemia with bone marrow failure is bone marrow transplantation. However, this treatment has many risks and is not available to all patients with Fanconi anemia.

CD34+ cells include stem cells found in the bone marrow or peripheral blood which are capable of making the red blood cells, white blood cells, and platelets. CD34+ stem cells can be collected from bone marrow or peripheral blood and purified using an experimental device called the CliniMACS. However, most Fanconi anemia patients do not have enough CD34+ stem cells in their bone marrow or peripheral blood to be collected using standard methods that work well in children and adults who don't have Fanconi anemia.


Condition or disease Intervention/treatment Phase
Fanconi Anemia Drug: AMD3100 Device: AmCell CliniMACs Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: AMD3100 in Combination With G-CSF to Mobilize Peripheral Blood Stem Cells in Patients With Fanconi Anemia(FA): A Phase I/II Study
Study Start Date : May 2007
Actual Primary Completion Date : December 2010
Actual Study Completion Date : December 2010


Arm Intervention/treatment
Experimental: AMD3100 Drug: AMD3100
240 mcg/kg subcutaneously, minimum of two days; maximum of eight days

Device: AmCell CliniMACs
CD34+ cell selection from peripheral collection




Primary Outcome Measures :
  1. Number of Participants Who Safely Received AMD3100 Used in Combination With Standard Dose G-CSF [ Time Frame: 30 days ]
    The safety of AMD3100 used in combination with standard dose G-CSF in Fanconi anemia patients to mobilize peripheral blood CD34+ cells for peripheral blood apheresis.

  2. Number of Participants for Whom a Sufficient Number of CD34+ Cells Were Mobilized Into the Peripheral Blood [ Time Frame: 3 days ]
    The effectiveness of AMD3100 used in combination with standard dose G-CSF in Fanconi anemia patients to mobilize a sufficient number of CD34+ cells into the peripheral blood. This target number is defined as > 5 CD34+ cells/mm3.


Secondary Outcome Measures :
  1. Number of Participants for Whom the Target Number of Hematopoietic Cells Were Collected by Peripheral Blood Apheresis [ Time Frame: 3 days ]
    Collecting a sufficient number of hematopoietic cells by peripheral blood apheresis, in patients with Fanconi anemia mobilized by AMD3100/G-CSF. This target number is defined as 2 x 106 CD34+ cells per kg patient weight based on the predicted weight in 5 years.

  2. Number of Participants for Whom the Target Number of CD34+ Cells Were Collected From Their Bone Marrow [ Time Frame: 3 days ]
    To collect a sufficient number of CD34+ cells from the bone marrow of Fanconi anemia patients who have failed AMD3100/G-CSF mobilization. This target number is defined as 2 x 106 CD34+ cells per kg patient weight based on the predicted weight in 5 years.

  3. Number of Participants for Whom the Target Number of CD34+ Cells Were Isolated. [ Time Frame: 3 days ]
    Isolating a sufficient number of CD34+ cells for clinical studies using the AmCell CliniMACs selection system. This target number is defined as 1 x 106 CD34+ cells per kg patient weight based on the predicted weight in 5 years.

  4. Number of Participants Whose Product Was Used for Preclinical Biological Investigations. [ Time Frame: 3 days ]
    To provide cells for preclinical biological investigations (up to 10% of the final product may be used).



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Ages Eligible for Study:   1 Year to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00479115


Locations
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United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
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Principal Investigator: Stella Davies, MD Children's Hospital Medical Center, Cincinnati
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Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT00479115    
Other Study ID Numbers: CCHMCEH004
R01HL081499 ( U.S. NIH Grant/Contract )
First Posted: May 25, 2007    Key Record Dates
Results First Posted: October 30, 2020
Last Update Posted: October 30, 2020
Last Verified: October 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Fanconi Syndrome
Anemia
Fanconi Anemia
Hematologic Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Congenital Bone Marrow Failure Syndromes
Bone Marrow Failure Disorders
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Plerixafor
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents