Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05618301 | Motixafortide and Natalizumab to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD) | PHASE1 | COMPLETED | 10 | — | — | Jul 7, 2023 | Jun 24, 2025 | Aug 11, 2025 | 1 | United States |
* 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, erythrocytosis, thrombocytosis, anemia, leukopenia, or febrile neutropenia * Non-hematologic criteria * Any Grade 4 or 5 adverse event. * Any Grade 3 or higher arterial or venous thromboembolic event * Any Grade 2 or 3 adverse event that does not resolve within 4 weeks; with the exception of \<grade 2 injection site reactions.
| Arm | Type | Description |
|---|---|---|
| Motixafortide followed by Motixafortide + Natalizumab | EXPERIMENTAL | * 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. |
| Name | Type | Description |
|---|---|---|
| Motixafortide | DRUG | Motixafortide is to be administered as a subcutaneous injection at a dose of 1.25 mg/kg |
| Natalizumab | DRUG | Natalizumab will be administered as an IV infusion at a flat dose of 300 mg |
| Leukapheresis | PROCEDURE | Leukapheresis consisting of a 1 Blood Volume procedure |
Inclusion Criteria: * Adult patients at least 18 years old * Diagnosis of sickle cell disease (hemoglobin SS or Sβ0 genotype) * Receiving automated RBC exchanges via apheresis-capable central venous access or willing to have apheresis-capable venous access placed. * Able to hold hydroxyurea, voxelo...