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New treatment option for Sickle Cell Disease
Official title SCD Stem Cell Mobilization and Apheresis Using Motixafortide
ClinicalTrials.gov ID: NCT06442761
What this study is testing
What is Motixafortide?
Motixafortide is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for sickle cell disease.
Also referred to as APHEXDA™.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Participants with severe sickle cell disease (SCD) who are ≥18 years of age and willing to donate autologous hematopoietic stem cells (HSCs) for...
- 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...
- 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...
You likely can't join 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...
- 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.
See the full eligibility 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...
- 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...
- 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...
- 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...
- 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.
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Bethesda, Maryland, United States
- Memphis, Tennessee, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Bethesda, Maryland, United States; Memphis, Tennessee, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.