AMD3100: 240 mcg/kg subcutaneously, minimum of two days; maximum of eight days
AmCell CliniMACs: CD34+ cell selection from peripheral collection

AMD3100

Sex: Female, Male

Race (NIH/OMB)

Region of Enrollment

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.

Number of Participants Who Safely Received AMD3100 Used in Combination With Standard Dose G-CSF

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.

Number of Participants for Whom a Sufficient Number of CD34+ Cells Were Mobilized Into the Peripheral Blood

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.

Number of Participants for Whom the Target Number of Hematopoietic Cells Were Collected by Peripheral Blood Apheresis

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.

Number of Participants for Whom the Target Number of CD34+ Cells Were Collected From Their Bone Marrow

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.

Number of Participants for Whom the Target Number of CD34+ Cells Were Isolated.

To provide cells for preclinical biological investigations (up to 10% of the final product may be used).

Number of Participants Whose Product Was Used for Preclinical Biological Investigations.

Overall Study

Stella Davies, MBBS, PhD, MRCP

Fanconi Anemia

240 mcg/kg subcutaneously, minimum of two days; maximum of eight days

CD34+ cell selection from peripheral collection

cafe-au-lait spots/mitomycin c

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

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