Trial Outcomes & Findings for Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100 (NCT NCT00479115)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

1 participants

Primary outcome timeframe

30 days

Results posted on

2020-10-30

Participant Flow

Participant milestones

Participant milestones
Measure	AMD3100 AMD3100: 240 mcg/kg subcutaneously, minimum of two days; maximum of eight days AmCell CliniMACs: CD34+ cell selection from peripheral collection
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	AMD3100 n=1 Participants AMD3100: 240 mcg/kg subcutaneously, minimum of two days; maximum of eight days AmCell CliniMACs: CD34+ cell selection from peripheral collection
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 30 days

Population: Please refer to the adverse event tables for specifics.

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.

Outcome measures

Outcome measures
Measure	AMD3100 n=1 Participants AMD3100: 240 mcg/kg subcutaneously, minimum of two days; maximum of eight days AmCell CliniMACs: CD34+ cell selection from peripheral collection
Number of Participants Who Safely Received AMD3100 Used in Combination With Standard Dose G-CSF	1 participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	AMD3100 n=1 Participants AMD3100: 240 mcg/kg subcutaneously, minimum of two days; maximum of eight days AmCell CliniMACs: CD34+ cell selection from peripheral collection
Number of Participants for Whom a Sufficient Number of CD34+ Cells Were Mobilized Into the Peripheral Blood	1 participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	AMD3100 n=1 Participants AMD3100: 240 mcg/kg subcutaneously, minimum of two days; maximum of eight days AmCell CliniMACs: CD34+ cell selection from peripheral collection
Number of Participants for Whom the Target Number of Hematopoietic Cells Were Collected by Peripheral Blood Apheresis	1 participants

SECONDARY outcome

Timeframe: 3 days

Population: One patient was enrolled and this patient met the target for collection.

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.

Outcome measures

Outcome measures
Measure	AMD3100 n=1 Participants AMD3100: 240 mcg/kg subcutaneously, minimum of two days; maximum of eight days AmCell CliniMACs: CD34+ cell selection from peripheral collection
Number of Participants for Whom the Target Number of CD34+ Cells Were Collected From Their Bone Marrow	1 participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	AMD3100 n=1 Participants AMD3100: 240 mcg/kg subcutaneously, minimum of two days; maximum of eight days AmCell CliniMACs: CD34+ cell selection from peripheral collection
Number of Participants for Whom the Target Number of CD34+ Cells Were Isolated.	1 participants

SECONDARY outcome

Timeframe: 3 days

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

Outcome measures

Outcome measures
Measure	AMD3100 n=1 Participants AMD3100: 240 mcg/kg subcutaneously, minimum of two days; maximum of eight days AmCell CliniMACs: CD34+ cell selection from peripheral collection
Number of Participants Whose Product Was Used for Preclinical Biological Investigations.	1 participants

Adverse Events

AMD3100

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	AMD3100 n=1 participants at risk AMD3100: 240 mcg/kg subcutaneously, minimum of two days; maximum of eight days AmCell CliniMACs: CD34+ cell selection from peripheral collection
Skin and subcutaneous tissue disorders Injection Site Reaction	100.0% 1/1 • Number of events 1 • 30 days following the last dose of AMD3100
Gastrointestinal disorders Vomiting	100.0% 1/1 • Number of events 3 • 30 days following the last dose of AMD3100
Cardiac disorders Hypotension	100.0% 1/1 • Number of events 1 • 30 days following the last dose of AMD3100
General disorders Fatigue	100.0% 1/1 • Number of events 1 • 30 days following the last dose of AMD3100
Gastrointestinal disorders Nausea	100.0% 1/1 • Number of events 1 • 30 days following the last dose of AMD3100
Metabolism and nutrition disorders Hyponatremia	100.0% 1/1 • Number of events 1 • 30 days following the last dose of AMD3100
Nervous system disorders Neuropathy: sensory	100.0% 1/1 • Number of events 1 • 30 days following the last dose of AMD3100
Eye disorders Blurred vision	100.0% 1/1 • Number of events 1 • 30 days following the last dose of AMD3100

Additional Information

Stella Davies, MBBS, PhD, MRCP

Cincinnati Children's Hospital Medical Center

Phone: 513-636-1371

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place