Trial Outcomes & Findings for G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers (NCT NCT00082329)

Last Updated: 2021-07-22

Results Overview

Healthy volunteers will be administered AMD 3100 (Mozobil plerixafor) and granulocyte colony stimulating factor (G-CSF) to determine cytokine polarization status of cluster of differentiation (CD 4) T-cells collected by apheresis. We propose that the combination of single dose AMD 3100 and G-CSF as combined mobilizing agents will improve the peripheral blood progenitor cells mobilization. Successful treatment responders is defined by completing study treatment with cell mobilization and cell collection. Non-responders is defined by having completed the study treatment and having cell mobilization without cell collection.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Day 1 (cells are counted 24 hours after AMD3100)

Results posted on

2021-07-22

Participant Flow

Participant milestones

Participant milestones
Measure	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers Participants received subcutaneous injection of granulocyte colony-stimulating factor (G-CSF) at 10 mcg/kg/day for 5 days followed by a single subcutaneous injection of AMD3100 (240 mcg/kg) given 12 hours prior to apheresis peripheral blood stem cell collection. Peripheral blood stem cell collection was performed on the fifth day following G-CSF administration.
Overall Study STARTED	9
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers n=9 Participants Participants received subcutaneous injection of granulocyte colony-stimulating factor (G-CSF) at 10 mcg/kg/day for 5 days followed by a single subcutaneous injection of AMD3100 (240 mcg/kg) given 12 hours prior to apheresis peripheral blood stem cell collection. Peripheral blood stem cell collection was performed on the fifth day following G-CSF administration.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Region of Enrollment United States	9 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 (cells are counted 24 hours after AMD3100)

Outcome measures

Outcome measures
Measure	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers n=9 Participants Participants received subcutaneous injection of granulocyte colony-stimulating factor (G-CSF) at 10 mcg/kg/day for 5 days followed by a single subcutaneous injection of AMD3100 (240 mcg/kg) given 12 hours prior to apheresis peripheral blood stem cell collection. Peripheral blood stem cell collection was performed on the fifth day following G-CSF administration.
Number of Participants With Successful Apheresis Collection Following Combination of AMD3100 and G-CSF. AMD & G-CSF Responder	8 participants
Number of Participants With Successful Apheresis Collection Following Combination of AMD3100 and G-CSF. AMD& G-CSF Non-Responder	1 participants

SECONDARY outcome

Timeframe: Day 7

Average fold change from baseline of mobilized cells that contained immune properties and other cellular content following G-CSF and AMD3100 to mobilize stem cells in healthy volunteers. The mobilized cells are defined as: white blood cells, lymphocytes, polys and monocytes. Polys (also known as segs, segmented neutrophils, neutrophils, granulocytes) are the most numerous of our white blood cells.

Outcome measures

Outcome measures
Measure	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers n=9 Participants Participants received subcutaneous injection of granulocyte colony-stimulating factor (G-CSF) at 10 mcg/kg/day for 5 days followed by a single subcutaneous injection of AMD3100 (240 mcg/kg) given 12 hours prior to apheresis peripheral blood stem cell collection. Peripheral blood stem cell collection was performed on the fifth day following G-CSF administration.
Average Fold Change From Baseline of Mobilized Cells Following G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers Lymphocytes	3.8 Mean Fold Change from Baseline Interval 2.4 to 5.3
Average Fold Change From Baseline of Mobilized Cells Following G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers Polys	11.9 Mean Fold Change from Baseline Interval 7.2 to 17.8
Average Fold Change From Baseline of Mobilized Cells Following G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers Monocytes	6.6 Mean Fold Change from Baseline Interval 2.2 to 12.2
Average Fold Change From Baseline of Mobilized Cells Following G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers White Blood Cells	9.3 Mean Fold Change from Baseline Interval 6.6 to 14.0

SECONDARY outcome

Timeframe: Day 7

Population: 1 participant did not complete apheresis, therefore 1 participant was not included in analysis

Number of participants with increase in yields of hematopoietic progenitor cells, immune cells, and other cellular subsets collected by apheresis following G-CSF and AMD3100 to mobilize stem cells.

Outcome measures

Outcome measures
Measure	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers n=9 Participants Participants received subcutaneous injection of granulocyte colony-stimulating factor (G-CSF) at 10 mcg/kg/day for 5 days followed by a single subcutaneous injection of AMD3100 (240 mcg/kg) given 12 hours prior to apheresis peripheral blood stem cell collection. Peripheral blood stem cell collection was performed on the fifth day following G-CSF administration.
Number of Participants With Increased the Levels of Circulating Hematopoietic Progenitor Cells, Immune Cells, and Other Cellular Subsets Collected by Apheresis.	8 participants

Adverse Events

G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers n=9 participants at risk Participants received subcutaneous injection of granulocyte colony-stimulating factor (G-CSF) at 10 mcg/kg/day for 5 days followed by a single subcutaneous injection of AMD3100 (240 mcg/kg) given 12 hours prior to apheresis peripheral blood stem cell collection. Peripheral blood stem cell collection was performed on the fifth day following G-CSF administration.
Vascular disorders deep vein thrombosis	11.1% 1/9 • 30 days

Other adverse events

Other adverse events
Measure	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers n=9 participants at risk Participants received subcutaneous injection of granulocyte colony-stimulating factor (G-CSF) at 10 mcg/kg/day for 5 days followed by a single subcutaneous injection of AMD3100 (240 mcg/kg) given 12 hours prior to apheresis peripheral blood stem cell collection. Peripheral blood stem cell collection was performed on the fifth day following G-CSF administration.
Blood and lymphatic system disorders Elevated Alk Phos	88.9% 8/9 • 30 days
Blood and lymphatic system disorders Elevated LDH	88.9% 8/9 • 30 days
Blood and lymphatic system disorders Elevated WBCs	11.1% 1/9 • 30 days
Cardiac disorders tachycardia	11.1% 1/9 • 30 days
Gastrointestinal disorders Diarrhea	44.4% 4/9 • 30 days
General disorders achy	11.1% 1/9 • 30 days
General disorders Appetite decr'd	11.1% 1/9 • 30 days
General disorders Body ache	11.1% 1/9 • 30 days
General disorders diaphoresis	11.1% 1/9 • 30 days
General disorders fatigue	33.3% 3/9 • 30 days
General disorders Headache	33.3% 3/9 • 30 days
General disorders low back pain	11.1% 1/9 • 30 days
General disorders Tiredness	11.1% 1/9 • 30 days
General disorders Tiredness (fatigue)	44.4% 4/9 • 30 days
Musculoskeletal and connective tissue disorders Back pain	11.1% 1/9 • 30 days
Musculoskeletal and connective tissue disorders bone pain	33.3% 3/9 • 30 days
Musculoskeletal and connective tissue disorders low back pain	11.1% 1/9 • 30 days
Musculoskeletal and connective tissue disorders Muscle pain	11.1% 1/9 • 30 days
Nervous system disorders paresthia (facial tingling)	11.1% 1/9 • 30 days
Psychiatric disorders Mood alteration	11.1% 1/9 • 30 days
Skin and subcutaneous tissue disorders Bruising, local at IV site	11.1% 1/9 • 30 days

Additional Information

Dr. Richard Childs

NHLBI NIH

Phone: 301-594-8008

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place