Actively Recruiting
SCD Stem Cell Mobilization and Apheresis Using Motixafortide
Led by St. Jude Children's Research Hospital · Updated on 2026-04-27
15
Participants Needed
2
Research Sites
189 weeks
Total Duration
On this page
Sponsors
S
St. Jude Children's Research Hospital
Lead Sponsor
A
Ayrmid Ltd
Collaborating Sponsor
AI-Summary
What this Trial Is About
This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use. PRIMARY OBJECTIVE * To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events (AEs). SECONDARY OBJECTIVES * To characterize the efficacy of a single dose (Part A) or two doses (Part B) of motixafortide for hematopoietic stem cell (HSC) mobilization and apheresis collection in participants with SCD as determined by the yield of CD34+ cells (CD34+ cells/kg). * To measure the mobilization effects of single-day (Part A) or daily dosing (Part B) dosing with motixafortide in the peripheral blood in participants with SCD as determined by peak peripheral blood CD34+ counts * To recommend a phase 2 dosing strategy based on safety, efficacy, and mobilization effects
CONDITIONS
Official Title
SCD Stem Cell Mobilization and Apheresis Using Motixafortide
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants with severe sickle cell disease (SCD) aged 18 years or older willing to donate autologous hematopoietic stem cells for future gene therapy
- Documented diagnosis of SCD with confirmed genotype by medical history
- Have or agree to have adequate intravenous access for apheresis (central line or other)
- ECOG performance status, Karnofsky score, or Lansky score greater than 80
- White blood cell count above 3.0 x 10^9/L and absolute neutrophil count above 1.0 x 10^9/L
- Platelet count above 150 x 10^9/L and hemoglobin above 7.0 gm/dL
- Adequate kidney function with creatinine less than 1.5 mg/dL and estimated glomerular filtration rate at least 60 mL/min/1.73 m^2
- Adequate liver function with direct bilirubin less than 2.5 times upper normal limit; AST and ALT less than 5 times upper normal limit
- Cardiac function with ejection fraction above 40% and no pulmonary hypertension within last 6 months
- Negative tests for syphilis, hepatitis B and C, HIV, and HTLV-1/II
- Feasible plan for exchange transfusion to reduce hemoglobin S to near 30% within one week of mobilization
- Negative pregnancy test for females of childbearing age
- Agreement to use two methods of effective contraception during treatment and for at least 1 month after last dose (for women of childbearing potential)
You will not qualify if you...
- Active or painful enlarged spleen larger than normal on exam
- Need for rare donor registry red blood cells or inability to receive routine transfusion
- Known allergy or contraindication to motixafortide or medications used during apheresis
- Prior autologous or allogeneic transplantation including gene therapy
- Active infections including viral, bacterial, fungal, or parasitic
- History of cancer except skin squamous carcinoma or cervical carcinoma in situ
- Received experimental therapy within 4 weeks before consent
- Poorly controlled diabetes
- Concurrent treatment with another investigational agent that cannot be stopped for 30 days
- Unwillingness to use effective contraception for at least 1 month after motixafortide
- Pregnancy
- Unable or unwilling to provide informed consent
- Unable or unwilling to stop hydroxyurea treatment for 30 days before first dose
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 2 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
Research Team
A
Alexis Leonard, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
OTHER
Number of Arms
2
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