Trial Outcomes & Findings for Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients (NCT NCT03226691)
NCT ID: NCT03226691
Last Updated: 2023-09-21
Results Overview
Sufficient collection of HSCs (target 2.0x106 CD34+ cells/kg) from the PB after plerixafor mobilization without serious adverse events (SAEs)
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
15 participants
Primary outcome timeframe
1 day
Results posted on
2023-09-21
Participant Flow
Participant milestones
| Measure |
Experimental: Plerixafor
Plerixafor at a single dose of 240 microgram/kg
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
COMPLETED
|
15
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients
Baseline characteristics by cohort
| Measure |
Experimental: Plerixafor
n=15 Participants
Plerixafor at a single dose of 240 microgram/kg
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
15 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
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
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 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
|
PRIMARY outcome
Timeframe: 1 dayPopulation: 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
Sufficient collection of HSCs (target 2.0x106 CD34+ cells/kg) from the PB after plerixafor mobilization without serious adverse events (SAEs)
Outcome measures
| Measure |
Experimental: Plerixafor
n=15 Participants
Plerixafor at a single dose of 240 microgram/kg
|
|---|---|
|
Number of Participants With Sufficient Collection of Hemopoietic Stem Cells (HSCs) Without Serious Adverse Events
|
14 Participants
|
Adverse Events
Experimental: Plerixafor
Serious events: 3 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Experimental: Plerixafor
n=15 participants at risk
Plerixafor at a single dose of 240 microgram/kg
|
|---|---|
|
Blood and lymphatic system disorders
Hemolysis
|
6.7%
1/15 • 10 days
|
|
General disorders
Pain
|
13.3%
2/15 • 10 days
|
Other adverse events
| Measure |
Experimental: Plerixafor
n=15 participants at risk
Plerixafor at a single dose of 240 microgram/kg
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
6.7%
1/15 • 10 days
|
|
Cardiac disorders
Cardiac disorders - Other specify: irregular heart rate
|
6.7%
1/15 • 10 days
|
|
Ear and labyrinth disorders
Tinnitus
|
6.7%
1/15 • 10 days
|
|
Eye disorders
Blurred vision
|
6.7%
1/15 • 10 days
|
|
Eye disorders
Eye disorders - Other specify: double vision
|
6.7%
1/15 • 10 days
|
|
Gastrointestinal disorders
Abdominal pain
|
13.3%
2/15 • 10 days
|
|
Gastrointestinal disorders
Bloating
|
13.3%
2/15 • 10 days
|
|
Gastrointestinal disorders
Constipation
|
6.7%
1/15 • 10 days
|
|
Gastrointestinal disorders
Dyspepsia
|
6.7%
1/15 • 10 days
|
|
Gastrointestinal disorders
Nausea
|
13.3%
2/15 • 10 days
|
|
Gastrointestinal disorders
Vomiting
|
6.7%
1/15 • 10 days
|
|
General disorders
Edema face
|
6.7%
1/15 • 10 days
|
|
General disorders
Edema limbs
|
6.7%
1/15 • 10 days
|
|
General disorders
Fatigue
|
26.7%
4/15 • 10 days
|
|
General disorders
Fever
|
6.7%
1/15 • 10 days
|
|
General disorders
General disorders and administration site conditions - Other specify: Leg pain
|
6.7%
1/15 • 10 days
|
|
General disorders
General disorders and administration site conditions - Other specify: Pain
|
6.7%
1/15 • 10 days
|
|
General disorders
Pain at the subcutaneous port site
|
6.7%
1/15 • 10 days
|
|
General disorders
Injection site reaction
|
13.3%
2/15 • 10 days
|
|
General disorders
Non-cardiac chest pain
|
6.7%
1/15 • 10 days
|
|
General disorders
Pain
|
40.0%
6/15 • 10 days
|
|
Injury, poisoning and procedural complications
Postoperative hemorrhage
|
6.7%
1/15 • 10 days
|
|
Investigations
Activated partial thromboplastin time prolonged
|
6.7%
1/15 • 10 days
|
|
Investigations
Blood bilirubin increased
|
6.7%
1/15 • 10 days
|
|
Investigations
Platelet count decreased
|
13.3%
2/15 • 10 days
|
|
Metabolism and nutrition disorders
Anorexia
|
6.7%
1/15 • 10 days
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
6.7%
1/15 • 10 days
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
26.7%
4/15 • 10 days
|
|
Metabolism and nutrition disorders
Hypokalemia
|
20.0%
3/15 • 10 days
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
40.0%
6/15 • 10 days
|
|
Metabolism and nutrition disorders
Hyponatremia
|
13.3%
2/15 • 10 days
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
6.7%
1/15 • 10 days
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
6.7%
1/15 • 10 days
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.7%
1/15 • 10 days
|
|
Nervous system disorders
Brachial plexopathy
|
6.7%
1/15 • 10 days
|
|
Nervous system disorders
Headache
|
40.0%
6/15 • 10 days
|
|
Nervous system disorders
Paresthesia
|
40.0%
6/15 • 10 days
|
|
Nervous system disorders
Spasticity
|
6.7%
1/15 • 10 days
|
|
Psychiatric disorders
Anxiety
|
6.7%
1/15 • 10 days
|
|
Psychiatric disorders
Confusion
|
6.7%
1/15 • 10 days
|
|
Psychiatric disorders
Insomnia
|
13.3%
2/15 • 10 days
|
|
Renal and urinary disorders
Acute kidney injury
|
6.7%
1/15 • 10 days
|
|
Renal and urinary disorders
Cystitis noninfective
|
6.7%
1/15 • 10 days
|
|
Renal and urinary disorders
Hematuria
|
6.7%
1/15 • 10 days
|
|
Renal and urinary disorders
Urinary retention
|
6.7%
1/15 • 10 days
|
|
Reproductive system and breast disorders
Vaginal dryness
|
6.7%
1/15 • 10 days
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
6.7%
1/15 • 10 days
|
|
Skin and subcutaneous tissue disorders
Swelling soft tissue left hand, dorsal interspace between thumb and index finger
|
6.7%
1/15 • 10 days
|
|
Vascular disorders
Flushing
|
6.7%
1/15 • 10 days
|
|
Vascular disorders
Hypotension
|
6.7%
1/15 • 10 days
|
Additional Information
John Tisdale
National Heart Lung and Blood Institute
Phone: +1 301 402 6497
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place