SCD Stem Cell Mobilization and Apheresis Using Motixafortide

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
St. Jude Children's Research Hospital
Study ID
NCT06442761
Phase
PHASE1
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Motixafortide — DRUG
    Given Subcutaneously (under the skin).

Study Details

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

Key Dates

Start date
Nov 14, 2024
Status verified
Jun 2026
Primary completion
Jan 31, 2028
Completion
Jul 31, 2028

Study Design

Enrollment
15 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
OTHER

Arms

  • Experimental: Treatment-Arm A-Single Dose
    Part A: Participants who enroll early will be assigned to Part A. Part A participants will get one dose of the study drug and one stem cell collection process.
  • Experimental: Treatment-Arm B-Two Daily Doses
    Part B: Participants who enroll later in the study will be assigned to Part B. Part B participants will get two doses of the study drug and two stem cell collection processes over two days (one on each day).

Primary Outcome Measure

To assess the safety and tolerability of motixafortide in participants with sickle cell disease (SCD) as determined by the incidence of adverse events. [ Time Frame: 0 - 30 days ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
University of Alabama at BirminghamBirminghamAlabama35294
Julie Kanter, MD
[email protected]
205-975-2837
Julie Kanter, MD (PRINCIPAL_INVESTIGATOR)
National Heart, Blood and Lung Institute-Cellular and Molecular Therapy BranchBethesdaMaryland20814
Matthew Hsieh, MD
[email protected]
301-273-5102
Matthew Hsieh, MD (PRINCIPAL_INVESTIGATOR)
St. Jude Children's Research HospitalMemphisTennessee38105
Alexis Leonard, MD
[email protected]
888-226-4343
Alexis Leonard, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Birmingham, AL

By condition
Sickle cell disease
By research site
University of Alabama at Birmingham· Birmingham, ALNational Heart, Blood and Lung Institute-Cellular and Molecular Therapy Branch· Bethesda, MDSt. Jude Children's Research Hospital· Memphis, TN

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