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Motixafortide and Natalizumab to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD)

NCT05618301 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-08-11

No results posted yet for this 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.

Conditions

Interventions

DRUG

Motixafortide

Motixafortide is to be administered as a subcutaneous injection at a dose of 1.25 mg/kg

DRUG

Natalizumab

Natalizumab will be administered as an IV infusion at a flat dose of 300 mg

PROCEDURE

Leukapheresis

Leukapheresis consisting of a 1 Blood Volume procedure

Sponsors & Collaborators

  • BioLineRx, Ltd.

    collaborator INDUSTRY

  • Biogen

    collaborator INDUSTRY

  • Ayrmid Pharma

    collaborator UNKNOWN

  • Washington University School of Medicine

    lead OTHER

Principal Investigators

  • Zachary Crees, M.D. · Washington University School of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-07-07
Primary Completion
2025-06-24
Completion
2025-06-24
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05618301 on ClinicalTrials.gov