Motixafortide + Natalizumab for Sickle Cell Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing two drugs, motixafortide and natalizumab, to help patients with sickle cell disease. These drugs aim to move more stem cells from the bone marrow into the blood, making it easier to collect them for gene therapy. Natalizumab is also used to treat multiple sclerosis.
Do I need to stop my current medications to join the trial?
Yes, you need to stop taking hydroxyurea, voxelotor, and/or crizanlizumab for at least 60 days before mobilization, and iron chelation for at least 7 days before mobilization.
What safety data exists for the treatment of Motixafortide + Natalizumab for Sickle Cell Disease?
The safety data for Motixafortide, a CXCR4 inhibitor, shows it is safe and well-tolerated when used with G-CSF for hematopoietic stem cell mobilization, with common adverse events being mild injection site reactions. Motixafortide is being developed for sickle cell disease, but specific safety data for its combination with Natalizumab in this context is not provided in the available research. However, Motixafortide has been shown to be a promising agent for stem cell mobilization in other conditions, and its safety profile in these contexts is favorable.12345
Is the drug Motixafortide a promising treatment for Sickle Cell Disease?
What data supports the idea that Motixafortide + Natalizumab for Sickle Cell Disease is an effective treatment?
The available research does not provide specific data on the effectiveness of Motixafortide + Natalizumab for Sickle Cell Disease. The studies mentioned focus on other conditions and treatments, such as autoimmune hemolytic anemia, hairy cell leukemia, idiopathic myelofibrosis, acute promyelocytic leukemia, and cancer patients undergoing stem cell collection. Therefore, there is no direct evidence from the provided information to support the effectiveness of Motixafortide + Natalizumab for Sickle Cell Disease.89101112
Who Is on the Research Team?
Zachary D Crees, M.D.
Principal Investigator
Washington University School of Medicine
Are You a Good Fit for This Trial?
Adults aged 18-40 with sickle cell disease (hemoglobin SS or Sβ0 genotype), able to pause certain medications, and have specific health criteria like a platelet count of at least 75,000/uL. They must not be pregnant, agree to use contraception during the study and for three months after, and cannot have had previous gene therapies or certain immunosuppressants.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Phase 1
Participants receive a single subcutaneous injection of motixafortide, followed by leukapheresis
Treatment Phase 2
Participants receive a single IV infusion of natalizumab, followed by a subcutaneous injection of motixafortide, then leukapheresis
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Leukapheresis
- Motixafortide
- Natalizumab
Leukapheresis is already approved in European Union, United States, Canada for the following indications:
- Acute myeloid leukemia (AML)
- Acute lymphoblastic leukemia (ALL)
- Chronic myeloid leukemia (CML)
- Hyperviscosity syndrome
- Acute myeloid leukemia (AML)
- Acute lymphoblastic leukemia (ALL)
- Chronic myeloid leukemia (CML)
- Hyperviscosity syndrome
- Acute myeloid leukemia (AML)
- Acute lymphoblastic leukemia (ALL)
- Chronic myeloid leukemia (CML)
- Hyperviscosity syndrome
Find a Clinic Near You
Who Is Running the Clinical Trial?
Washington University School of Medicine
Lead Sponsor
Biogen
Industry Sponsor
Daniel Quirk
Biogen
Chief Medical Officer
MD
Christopher A. Viehbacher
Biogen
Chief Executive Officer since 2022
Graduated from Queen's University, Kingston, Ontario, Canada
BioLineRx, Ltd.
Industry Sponsor