Motixafortide followed by Motixafortide + Natalizumab for Anemia, Sickle Cell
Phase-Based Progress Estimates
Effectiveness
Safety
Anemia, Sickle Cell+1 More
Motixafortide - DrugEligibility
18 - 65
All Sexes
What conditions do you have?
Select
Eligibility
18 - 65
All Sexes
What conditions do you have?
Select
Study Summary
Hematopoietic stem cell (HSC)-based gene therapies now offer curative potential for patients with sickle cell disease (SCD), with decreased toxicity compared to allogeneic hematopoietic cell transplantation. However, effective HSC-based gene therapy depends on collecting sufficient HSCs to generate the therapeutic product, and currently available mobilization regimens carry unacceptable risk for patients with SCD or do not reliably yield optimal numbers of HSCs for gene therapy. The investigators hypothesize that HSC mobilization with motixafortide (CXCR4i) alone and the combination of motixafortide plus natalizumab (VLA-4i) will be safe and tolerable in SCD patients. In addition, the investigators hypothesize that combined CXCR4 and VLA-4 blockade with motixafortide plus natalizumab will result in a rapid, robust, and synergistic increase in HSC mobilization to peripheral blood (PB) in patients with SCD, when compared to motixafortide alone.
Eligible Conditions
- Anemia, Sickle Cell
Treatment Effectiveness
Effectiveness Progress
Study Objectives
1 Primary · 6 Secondary · Reporting Duration: From start of treatment through 6 weeks following completion of all treatment (estimated to be 3 months)
Day 44
Leukapheresis
Leukapheresis
Day 44
Natalizumab
Change in peak CD34+ HSC mobilization in response to motixafortide alone and motixafortide + natalizumab in SCD patients, as assessed by CD34+ cells/ul in peripheral blood
Month 3
Natalizumab
Week 8
Safety and tolerability as assessed by the incidence of dose-limiting toxicities (DLTs)
Month 5
Natalizumab
Trial Safety
Safety Progress
Trial Design
1 Treatment Group
Motixafortide followed by Motixafortide + Natalizumab
1 of 1
Experimental Treatment
5 Total Participants · 1 Treatment Group
Primary Treatment: Motixafortide followed by Motixafortide + Natalizumab · No Placebo Group · Phase 1
Motixafortide followed by Motixafortide + NatalizumabExperimental Group · 3 Interventions: Motixafortide, Natalizumab, Leukapheresis · Intervention Types: Drug, Drug, Procedure
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Natalizumab
2003
Completed Phase 4
~4250
Leukapheresis
2010
Completed Phase 2
~510
Trial Logistics
Trial Timeline
Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: from start of treatment through 6 weeks following completion of all treatment (estimated to be 3 months)
Who is running the clinical trial?
BioLineRx, Ltd.Industry Sponsor
21 Previous Clinical Trials
2,071 Total Patients Enrolled
Washington University School of MedicineLead Sponsor
1,789 Previous Clinical Trials
2,271,871 Total Patients Enrolled
16 Trials studying Anemia, Sickle Cell
11,138 Patients Enrolled for Anemia, Sickle Cell
Zachary Crees, M.D.Principal InvestigatorWashington University School of Medicine
Eligibility Criteria
Age 18 - 65 · All Participants · 10 Total Inclusion Criteria
Mark “yes” if the following statements are true for you:You have received a transfusion of RBCs via an apheresis-capable central venous access.
You are able to hold hydroxyurea for at least 4 weeks prior to mobilization.
You have a platelet count of at least 75,000/microliter.
AST/ALT ratio is greater than 3.0 x IULN at time of screening.
Creatinine clearance ≥ 30 mL/min.
About The Reviewer
Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.
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