Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients

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ClinicalTrials.gov Identifier: NCT03226691

Recruitment Status : Completed

First Posted : July 24, 2017

Results First Posted : April 20, 2020

Last Update Posted : April 20, 2020

View this study on Beta.ClinicalTrials.gov

Sponsor:

Collaborator:

St. Jude Children's Research Hospital

Information provided by (Responsible Party):

National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Study Type	Interventional
Study Design	Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Supportive Care
Condition	Sickle Cell Disease
Intervention	Drug: Plerixafor
Enrollment	15

Participant Flow

Recruitment Details
Pre-assignment Details


Arm/Group Title	Experimental: Plerixafor
Arm/Group Description	Plerixafor at a single dose of 240 ...
Arm/Group Description	Plerixafor at a single dose of 240 microgram/kg
Period Title: Overall Study
Started	15
Completed	15
Not Completed	0

Baseline Characteristics

Arm/Group Title		Experimental: Plerixafor
Arm/Group Description		Plerixafor at a single dose of 240 ...
Arm/Group Description		Plerixafor at a single dose of 240 microgram/kg
Overall Number of Baseline Participants		15
Baseline Analysis Population Description		[Not Specified]
Baseline Analysis Population Description		[Not Specified]
Age, Categorical Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	15 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	15 100.0%
	>=65 years	0 0.0%
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	15 participants
	Female	8 53.3%
	Male	7 46.7%
Ethnicity (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	15 participants
	Hispanic or Latino	0 0.0%
	Not Hispanic or Latino	15 100.0%
	Unknown or Not Reported	0 0.0%
Race (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	15 participants
	American Indian or Alaska Native	0 0.0%
	Asian	0 0.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%
	Black or African American	15 100.0%
	White	0 0.0%
	More than one race	0 0.0%
	Unknown or Not Reported	0 0.0%

Outcome Measures

1.Primary Outcome


Title	Number of Participants With Sufficient Collection of Hemopoietic Stem Cells (HSCs) Without Serious Adverse Events
Description	Sufficient collection of HSCs (target 2.0x106 CD34+ cells/kg) from the...
Description	Sufficient collection of HSCs (target 2.0x106 CD34+ cells/kg) from the PB after plerixafor mobilization without serious adverse events (SAEs)
Time Frame	1 day

Outcome Measure Data

Analysis Population Description

Individuals with SCD age 18 or greater and are willing to donate HSC collection for future gene therapy or gene editing study, if no allogeneic HSC transplantation study was currently available


Arm/Group Title	Experimental: Plerixafor
Arm/Group Description:	Plerixafor at a single dose of 240 ...
Arm/Group Description:	Plerixafor at a single dose of 240 microgram/kg
Overall Number of Participants Analyzed	15
Measure Type: Count of Participants Unit of Measure: Participants
	14 93.3%

Adverse Events

Time Frame	10 days
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	Experimental: Plerixafor
Arm/Group Description	Plerixafor at a single dose of 240 ...
Arm/Group Description	Plerixafor at a single dose of 240 microgram/kg
All-Cause Mortality
	Experimental: Plerixafor
	Affected / at Risk (%)
Total	0/15 (0.00%)
Serious Adverse Events Serious Adverse Events
	Experimental: Plerixafor
	Affected / at Risk (%)
Total	3/15 (20.00%)
Blood and lymphatic system disorders
Hemolysis ^†	1/15 (6.67%)
General disorders
Pain ^†	2/15 (13.33%)
† Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Experimental: Plerixafor
	Affected / at Risk (%)
Total	15/15 (100.00%)
Blood and lymphatic system disorders
Anemia ^†	1/15 (6.67%)
Cardiac disorders
Cardiac disorders - Other specify: irregular heart rate ^†	1/15 (6.67%)
Ear and labyrinth disorders
Tinnitus ^†	1/15 (6.67%)
Eye disorders
Blurred vision ^†	1/15 (6.67%)
Eye disorders - Other specify: double vision ^†	1/15 (6.67%)
Gastrointestinal disorders
Abdominal pain ^†	2/15 (13.33%)
Bloating ^†	2/15 (13.33%)
Constipation ^†	1/15 (6.67%)
Dyspepsia ^†	1/15 (6.67%)
Nausea ^†	2/15 (13.33%)
Vomiting ^†	1/15 (6.67%)
General disorders
Edema face ^†	1/15 (6.67%)
Edema limbs ^†	1/15 (6.67%)
Fatigue ^†	4/15 (26.67%)
Fever ^†	1/15 (6.67%)
General disorders and administration site conditions - Other specify: Leg pain ^†	1/15 (6.67%)
General disorders and administration site conditions - Other specify: Pain ^†	1/15 (6.67%)
Pain at the subcutaneous port site ^†	1/15 (6.67%)
Injection site reaction ^†	2/15 (13.33%)
Non-cardiac chest pain ^†	1/15 (6.67%)
Pain ^†	6/15 (40.00%)
Injury, poisoning and procedural complications
Postoperative hemorrhage ^†	1/15 (6.67%)
Investigations
Activated partial thromboplastin time prolonged ^†	1/15 (6.67%)
Blood bilirubin increased ^†	1/15 (6.67%)
Platelet count decreased ^†	2/15 (13.33%)
Metabolism and nutrition disorders
Anorexia ^†	1/15 (6.67%)
Hyperglycemia ^†	1/15 (6.67%)
Hypocalcemia ^†	4/15 (26.67%)
Hypokalemia ^†	3/15 (20.00%)
Hypomagnesemia ^†	6/15 (40.00%)
Hyponatremia ^†	2/15 (13.33%)
Hypophosphatemia ^†	1/15 (6.67%)
Musculoskeletal and connective tissue disorders
Bone pain ^†	1/15 (6.67%)
Myalgia ^†	1/15 (6.67%)
Nervous system disorders
Brachial plexopathy ^†	1/15 (6.67%)
Headache ^†	6/15 (40.00%)
Paresthesia ^†	6/15 (40.00%)
Spasticity ^†	1/15 (6.67%)
Psychiatric disorders
Anxiety ^†	1/15 (6.67%)
Confusion ^†	1/15 (6.67%)
Insomnia ^†	2/15 (13.33%)
Renal and urinary disorders
Acute kidney injury ^†	1/15 (6.67%)
Cystitis noninfective ^†	1/15 (6.67%)
Hematuria ^†	1/15 (6.67%)
Urinary retention ^†	1/15 (6.67%)
Reproductive system and breast disorders
Vaginal dryness ^†	1/15 (6.67%)
Skin and subcutaneous tissue disorders
Pruritus ^†	1/15 (6.67%)
Swelling soft tissue left hand, dorsal interspace between thumb and index finger ^†	1/15 (6.67%)
Vascular disorders
Flushing ^†	1/15 (6.67%)
Hypotension ^†	1/15 (6.67%)
† Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	John Tisdale
Organization:	National Heart Lung and Blood Institute
Phone:	+1 301 402 6497
EMail:	johntis@nhlbi.nih.gov

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Leonard A, Sharma A, Uchida N, Stroncek D, Panch SR, West K, Molloy E, Hughes TE, Hauffe S, Taylor T, Fitzhugh C, Hankins JS, Wilson M, Tsai SQ, Weiss MJ, Hsieh M, Tisdale JF. Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease. Blood Adv. 2021 May 11;5(9):2403-2411. doi: 10.1182/bloodadvances.2021004232.

Layout table for additonal information

Responsible Party:	National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier:	NCT03226691
Other Study ID Numbers:	170124 17-H-0124
First Submitted:	July 21, 2017
First Posted:	July 24, 2017
Results First Submitted:	April 3, 2020
Results First Posted:	April 20, 2020
Last Update Posted:	April 20, 2020

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