Sickle Cell DiseasePhase 1NCT06442761
SCD Stem Cell Mobilization and Apheresis Using Motixafortide
A Phase 1 study testing Motixafortide in people with Sickle Cell Disease, run by St. Jude Children's Research Hospital at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Motixafortide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesUnited States
- Study dates
- 14 Nov 2024to Jul 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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)
- Intrauterine hormone-releasing system (IUS)
- Bilateral tubal occlusion
- Vasectomised partner
- Sexual abstinence.
Who cannot join
- 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.
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- 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
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
National Heart, Blood and Lung Institute-Cellular and Molecular Therapy Branch
Bethesda, Maryland, 20814
Matthew Hsieh, MD, 301-273-5102, Matthewhs@nhlbi.nih.gov
Matthew Hsieh, MD
- Recruiting
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Julie Kanter, MD, 205-975-2837, jkanter@uabmc.edu
Julie Kanter, MD
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Alexis Leonard, MD, 888-226-4343, referralinfo@stjude.org
Alexis Leonard, MD
About this study
Official title: Sickle Cell Disease Stem Cell Mobilization and Apheresis Using Motixafortide
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
Read more
- 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
Read the detailed description
This study is divided into 2 parts. Participants will be assigned to a part based on when they enroll. Early in the study, participants will be assigned to part A. Later in the study, participants will be assigned to part B. In both parts, participants will receive the study drug, motixafortide, by an injection under the skin. About 8 hours after the injection, stem cells will be collected. Participants will follow-up within 7-10 days after the study drug and stem cell collection. Study staff will contact participants about 30 (±3) days after the last drug dose administration. The follow up may be conducted in person if the participant is in clinic during that follow up period.
- Conditions
- Sickle Cell Disease
- Being tested
- Motixafortide
- Keywords
- Stem Cell Mobilization, Apheresis, Motixafortide
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MotixafortideDrug
Also called APHEXDA™
Given Subcutaneously (under the skin).
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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). Receives: Drug: Motixafortide |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the safety and tolerability of motixafortide in participants with sickle cell disease (SCD) as determined by the incidence of adverse events.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2024
- Study start14 Nov 2024
- Primary completionJan 2028 (estimated)
- Study completionJul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Ayrmid Ltd
- Responsible party
- Sponsor
- Investigators
- Alexis Leonard, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06442761
- Sponsor's protocol ID
- SCDSTEMM
- Status last verified
- Sep 2026
- First submitted
- 13 May 2024
- Last updated
- 8 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sickle Cell Disease Stem Cell Mobilization and Apheresis Using Motixafortide
- ClinicalTrials.gov ID
- NCT06442761
- Acronym / sponsor ID
- SCDSTEMM
- Conditions
- Sickle Cell Disease
- Keywords
- Stem Cell Mobilization, Apheresis, Motixafortide
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- Ayrmid Ltd
- Study start
- 14 Nov 2024 (actual)
- Primary completion
- Jan 2028 (estimated)
- Study completion
- Jul 2028 (estimated)
- First posted
- 4 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
This study is divided into 2 parts. Participants will be assigned to a part based on when they enroll. Early in the study, participants will be assigned to part A. Later in the study, participants will be assigned to part B. In both parts, participants will receive the study drug, motixafortide, by an injection under the skin. About 8 hours after the injection, stem cells will be collected. Participants will follow-up within 7-10 days after the study drug and stem cell collection. Study staff will contact participants about 30 (±3) days after the last drug dose administration. The follow up may be conducted in person if the participant is in clinic during that follow up period.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment-Arm A-Single Dose | Experimental | Drug: MotixafortidePart 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. |
| Treatment-Arm B-Two Daily Doses | Experimental | Drug: MotixafortidePart 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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- 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
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MotixafortideAlso: APHEXDA™Given Subcutaneously (under the skin). | Drug | Treatment-Arm A-Single Dose, Treatment-Arm B-Two Daily Doses |
Primary outcome
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
4 secondary outcomes
To determine the yield of CD34+ cells/kg after a single or daily dose of motixafortide in participants with SCD.
Time frame: 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.
Time frame: 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
Time frame: 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
Time frame: through study completion, an average of 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2026.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Alexis Leonard, MD principal investigator
St. Jude Children's Research Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Motixafortide in people with Sickle Cell Disease, run by St. Jude Children's Research Hospital at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alexis Leonard, MD (888-226-4343, referralinfo@stjude.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2026.