SCD Stem Cell Mobilization and Apheresis Using Motixafortide

NCT06442761 | Recruiting Phase 1 FDA Drug

• 1 other identifier: SCDSTEMM
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 2

Brief Summary

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

Sickle Cell Disease

Keywords

Stem Cell Mobilization; Apheresis; Motixafortide

Outcome Measures

Primary Outcomes (1)

• To assess the safety and tolerability of motixafortide in participants with sickle cell disease (SCD) as determined by the incidence of adverse events.

  0 - 30 days

Secondary Outcomes (4)

• To determine the yield of CD34+ cells/kg after a single or daily dose of motixafortide in participants with SCD.

  1 - 2 days

• To determine the mobilization effects as determined by peripheral blood CD34+ counts/uL after a single or daily dose of motixafortide in participants with SCD.

  1 - 2 days

• To recommend a phase 2 dosing strategy of one or two doses motixafortide in participants with SCD based on the incidence of adverse events

  through study completion, an average of 1 year

• To recommend a phase 2 dosing strategy for timing of apheresis based on peripheral blood CD34+ counts/uL after motixafortide administration in participants with SCD

  through study completion, an average of 1 year

Study Arms (2)

Treatment-Arm A-Single Dose

EXPERIMENTAL

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.

Drug: Motixafortide

Treatment-Arm B-Two Daily Doses

EXPERIMENTAL

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).

Drug: Motixafortide

Interventions

Motixafortide DRUG

Given Subcutaneously (under the skin).

Also known as: APHEXDA™

Treatment-Arm A-Single Dose; Treatment-Arm B-Two Daily Doses

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants with severe sickle cell disease (SCD) who are ≥18 years of age and willing to donate autologous hematopoietic stem cells (HSCs) for advancing future gene therapy for SCD after collection of back-up product. Severe SCD, for the purpose of this study, will be defined as participants who are receiving chronic transfusion therapy due to SCD related complications or are eligible for or currently enrolled on an allogeneic transplant protocol.
• Participant must have a documented diagnosis of SCD with documentation of SCD genotype by medical history
• Participants should either have a central line in place, be able to undergo apheresis without the necessity of the insertion of a central venous catheter, or agree to have a central line placed if IV access is inadequate.
• ECOG performance status/Karnofsky score/Lansky score \>80
• White blood cell (WBC) count \>3.0 x 10\^9/L, absolute neutrophil count (ANC) \>1.0 x 10\^9/L, and platelet count \>150 x 10\^9/L, and hemoglobin \>7.0 gm/dL
• Adequate renal function defined as serum/plasma creatinine \< 1.5 mg/dL and an estimated glomerular filtration rate (eGFR) of at least 60 mL/min/1.73 m\^2 based on the CKD-Epi equation or the St. Jude equation.
• Adequate liver function defined as direct bilirubin \< 2.5 times the upper limit of normal range; aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 5 times the upper limit of normal range.
• Participant's cardiac function (i.e., ejection fraction \>40%) and pulmonary status (i.e., no evidence of pulmonary hypertension) within the last 6 months must be sufficient to undergo apheresis, as assessed by the Principal Investigator or an independent physician evaluating the participant. If an assessment has not been done within the last 6 months, an echocardiogram will be performed.
• Negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1/II
• Feasible manual or automated exchange transfusion plan to achieve hemoglobin S (HbS) near 30% within one week of mobilization
• Female participants of childbearing age should have a negative pregnancy test.
• Participants of childbearing potential should agree to use of a highly effective form of contraception during treatment and for at least 1 month after the last dose of motixafortide. Women of childbearing potential must agree to use 2 methods of effective contraception: One barrier method (e.g. diaphragm, or condom or sponge, each of which are to be combined with a spermicide) and one hormonal method, unless she uses a highly effective method. Highly effective methods of contraception include:
• Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: oral, intravaginal, transdermal
• Progestogen-only hormonal contraception associated with inhibition of ovulation: oral, injectable, implantable
• Intrauterine device (IUD)

You may not qualify if:

• Active and painful splenomegaly or splenomegaly (size greater than upper limit of normal on examination).
• Participant who, by medical history, requires rare donor registry RBC units for transfusion, or is unable to receive routine transfusion. Eligible study participants must have undergone prior work-up for the presence of red cell alloantibodies and confirmation of available compatible blood product support
• Known allergy to or contraindication for motixafortide administration, or medications routinely administered during apheresis
• Participant who has had a prior autologous or allogeneic transplantation, inclusive of gene therapy
• Active viral, bacterial, fungal, or parasitic infection.
• History of cancer, excluding squamous carcinoma of the skin and cervical carcinoma in situ.
• Participant who has received experimental therapy within 4 weeks prior to providing informed consent
• Poorly controlled diabetes mellitus, as assessed by the Investigator
• Concomitant treatment with alternative investigational agent unable to be held for 30 days
• Unwillingness to use a highly effective method of contraception for 1 month after motixafortide
• Pregnancy
• Inability or unwillingness of research participant or legal guardian/ representative to give written informed consent.
• Inability or unwillingness of research participant to hold hydroxyurea for 30 days prior to first dose of study drug

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead
• Ayrmid Ltd: collaborator

Study Sites (2)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

RECRUITING

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Alexis Leonard, MD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Alexis Leonard, MD

CONTACT

888-226-4343; referralinfo@stjude.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 13, 2024
• First Posted: June 4, 2024
• Study Start: November 14, 2024
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): July 1, 2028
• Last Updated: April 27, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be made available at the time of article publication.
• Access Criteria: Data will be provided to researchers following a formal request with the following information: full name of requestor, affiliation, data set requested, and timing of when data is needed. As an informational point, the lead statistician and study principal investigator will be informed that primary results datasets have been requested.

Locations

US (2)