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Motixafortide + Natalizumab for Sickle Cell Disease
Phase 1
Recruiting
Led By Zachary Crees, M.D.
Research Sponsored by Washington University School of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from start of treatment through 8 weeks following completion of all treatment (estimated to be 16 weeks and 4 days)
Awards & highlights
No Placebo-Only Group
Summary
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.
Who is the study for?
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.
What is being tested?
The trial is testing Motixafortide and Natalizumab's ability to mobilize hematopoietic stem cells in preparation for gene therapy in sickle cell disease patients. It aims to see if these drugs can safely increase the number of stem cells available for treatment without unacceptable risks.
What are the potential side effects?
Potential side effects may include reactions related to immune system suppression given that Natalizumab affects immune function. Specific side effects are not listed but could align with those typical of agents affecting blood cell mobilization such as bone pain, fatigue, headache, nausea.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ from start of treatment through 8 weeks following completion of all treatment (estimated to be 16 weeks and 4 days)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from start of treatment through 8 weeks following completion of all treatment (estimated to be 16 weeks and 4 days)
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Safety and tolerability as assessed by the incidence of dose-limiting toxicities (DLTs)
Secondary study objectives
Frequency of adverse events
Leukapheresis
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: Motixafortide followed by Motixafortide + NatalizumabExperimental Treatment3 Interventions
* Consenting and eligible patients will receive a single subcutaneous injection of motixafortide, followed by leukapheresis. Patient will then be followed for 8 weeks for adverse event monitoring.
* Following the 8-week monitoring period, patients will receive a single IV infusion natalizumab, then approximately 32 hours later, a single subcutaneous injection of motixafortide, followed by leukapheresis. Patients will then be followed for 8 weeks for adverse event monitoring.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Leukapheresis
2016
Completed Phase 2
~710
Natalizumab
2003
Completed Phase 4
~9310
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, which increases fetal hemoglobin (HbF) to reduce red blood cell sickling and vaso-occlusive episodes, and chronic transfusions, which dilute sickle hemoglobin (HbS) with normal hemoglobin to prevent complications. Investigational therapies like Motixafortide (CXCR4 inhibition) and Natalizumab (VLA-4 inhibition) focus on mobilizing hematopoietic stem cells and reducing cell adhesion, respectively, to improve blood flow and reduce vaso-occlusion.
These treatments are vital as they target the underlying mechanisms of SCD, aiming to alleviate pain, prevent complications, and enhance the quality of life for patients.
Regular long-term red blood cell transfusions for managing chronic chest complications in sickle cell disease.Sickle-cell disease.
Regular long-term red blood cell transfusions for managing chronic chest complications in sickle cell disease.Sickle-cell disease.
Find a Location
Who is running the clinical trial?
Washington University School of MedicineLead Sponsor
1,999 Previous Clinical Trials
2,335,478 Total Patients Enrolled
BiogenIndustry Sponsor
646 Previous Clinical Trials
466,682 Total Patients Enrolled
BioLineRx, Ltd.Industry Sponsor
23 Previous Clinical Trials
2,150 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am currently taking drugs that suppress my immune system.I have never had a stem cell transplant or gene therapy.My bone marrow and organs are functioning normally.I am undergoing or willing to undergo a procedure for blood exchange through a central vein.I can stop my iron chelation therapy for at least 7 days before treatment.I am fully active and can carry on all my pre-disease activities without restriction.I have had a brain infection called progressive multifocal leukoencephalopathy.I do not have any uncontrolled illnesses or infections.I agree to use birth control during and for 3 months after the study.My kidneys are functioning well enough (creatinine clearance ≥ 30 mL/min).I am between 18 and 40 years old.I can stop taking hydroxyurea, voxelotor, or crizanlizumab for 60 days before the next treatment step.I have been diagnosed with sickle cell disease.I can understand and am willing to sign the consent form.
Research Study Groups:
This trial has the following groups:- Group 1: Motixafortide followed by Motixafortide + Natalizumab
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.