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NCT05618301 · ClinicalTrials.gov registry record · Phase 1
Motixafortide and Natalizumab to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD)
A Phase 1 study, sponsored by Washington University School of Medicine.
- Completed
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
NCT05618301: Completed Phase 1 study, sponsored by Washington University School of Medicine.
NCT05618301 is a Phase 1 study that has completed, run by Washington University School of Medicine. The registered enrollment target is 10 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05618301, a Phase 1 study, has completed, sponsored by Washington University School of Medicine.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
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.
Primary Outcome
* All toxicities will be graded using NCI-CTCAE Version 5.0 * A DLT is an event occurring during the DLT period of 28 days following administration of either motixafortide and/or natalizumab that is considered to be at least possibly, probably or definitely related to study treatment by the investigator, and that meets the criteria below: * Hematologic criteria * Any Grade 5 adverse event * Any Grade 4 adverse event, excluding Grade 4 hemolysis, bilirubin increase, leukocytosis, eryt
Interventions
- PROCEDURE Leukapheresis
- DRUG Motixafortide
- DRUG Natalizumab
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2023-07-07 |
| Est. Completion | 2025-06-24 |
| Phase | Phase 1 |
What the finished NCT05618301 record still lists
NCT05618301 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower).
The record links to 0 conditions, and to 3 interventions - of which Leukapheresis is the first listed.
NCT05618301 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT05618301 about?
NCT05618301 is a clinical study titled "Motixafortide and Natalizumab to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD)". 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 ...
What is the current status of trial NCT05618301?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2023-07-07. Estimated completion is 2025-06-24.
What interventions are being tested in trial NCT05618301?
The interventions under investigation include: Leukapheresis (PROCEDURE), Motixafortide (DRUG), Natalizumab (DRUG).
Who is sponsoring clinical trial NCT05618301?
This trial is sponsored by Washington University School of Medicine, which has 1,502 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT05618301's enrollment target sits among peer trials
10 1913th of 2000 higher than 46 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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