SCD Stem Cell Mobilization and Apheresis Using Motixafortide
Sickle Cell Disease Stem Cell Mobilization and Apheresis Using Motixafortide
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Procedures mentioned: Injections
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "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."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests whether motixafortide is safe for adults with severe sickle cell disease. It also tests whether the drug helps move more blood-forming stem cells into the blood so they can be collected for possible future transplants.
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
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 18 or older with a confirmed sickle cell disease type. You must get regular blood transfusions for sickle cell problems, or qualify for or be enrolled in a donor transplant study.
- Be willing to donate your own blood-forming stem cells for future sickle cell gene therapy research after backup cells are collected.
- Have suitable vein access for apheresis, a process that collects cells from blood, or agree to have a tube placed in a large vein.
- Meet the study’s daily activity score and blood count requirements.
- Meet kidney and liver test requirements. Your heart must pump well enough, and you must have no signs of high blood pressure in the lungs.
- Test negative for syphilis, hepatitis B and C, HIV, and HTLV-1/II.
- Have a workable plan to replace some blood through transfusion to bring sickle hemoglobin near 30% within one week of the drug treatment.
- If you could become pregnant, have a negative pregnancy test. If you could have children, agree to the required birth control during treatment and for at least 1 month after the last dose.
Who can't join
- Have an enlarged spleen, including one that is painful.
- Need rare-donor red blood cells or be unable to receive routine blood transfusions.
- Have an allergy or another medical reason you cannot receive motixafortide or medicines used during blood cell collection.
- Have had a stem cell transplant using your own or a donor’s cells, including gene therapy.
- Have an active infection, poorly controlled diabetes, or a history of cancer other than squamous cell skin cancer or cervical cancer that has not spread beyond its starting layer.
- Have received an experimental treatment within 4 weeks before consent, or be unable to stop another experimental treatment for 30 days.
- Be pregnant or unwilling to use highly effective birth control for 1 month after motixafortide.
- Be unable or unwilling to give written consent, either yourself or through a legal representative, or to stop hydroxyurea for 30 days before the first study dose.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: * 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) * Intrauterine hormone-releasing system (IUS) * Bilateral tubal occlusion * Vasectomised partner * Sexual abstinence. Exclusion Criteria: * 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
Study sites (3)
AL
- University of Alabama at Birmingham · Birmingham, AL 35294Julie Kanter, MD · Study contact · 205-975-2837 · jkanter@uabmc.eduJulie Kanter, MD · Principal investigatorMore trials in Birmingham
MD
- National Heart, Blood and Lung Institute-Cellular and Molecular Therapy Branch · Bethesda, MD 20814Matthew Hsieh, MD · Study contact · 301-273-5102 · Matthewhs@nhlbi.nih.govMatthew Hsieh, MD · Principal investigatorMore trials in Bethesda
TN
- St. Jude Children's Research Hospital · Memphis, TN 38105Alexis Leonard, MD · Study contact · 888-226-4343 · referralinfo@stjude.orgAlexis Leonard, MD · Principal investigatorMore trials in Memphis