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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 between 18 and 65 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

Study Summary

This trial will test if a new combination of drugs can safely and effectively mobilize hematopoietic stem cells for gene therapy in sickle cell disease patients.

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.Check my eligibility

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.See study design

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)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from start of treatment through 8 weeks following completion of all treatment (estimated to be 16 weeks and 4 days) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Safety and tolerability as assessed by the incidence of dose-limiting toxicities (DLTs)

Secondary outcome measures

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

Frequency of adverse events

+2 more

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

~690

Natalizumab

2003

Completed Phase 4

~4900

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Washington University School of MedicineLead Sponsor

1,935 Previous Clinical Trials

2,299,868 Total Patients Enrolled

BiogenIndustry Sponsor

639 Previous Clinical Trials

467,317 Total Patients Enrolled

BioLineRx, Ltd.Industry Sponsor

21 Previous Clinical Trials

2,115 Total Patients Enrolled

Media Library

Leukapheresis (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05618301 — Phase 1

Sickle Cell Disease Research Study Groups: Motixafortide followed by Motixafortide + Natalizumab

Sickle Cell Disease Clinical Trial 2023: Leukapheresis Highlights & Side Effects. Trial Name: NCT05618301 — Phase 1

Leukapheresis (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05618301 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the recruitment process for this experiment still ongoing?

"Unfortunately, the clinicaltrial.gov records demonstrate that this investigation is no longer recruiting patients due to its last update on November 8th 2022. However, there are an additional 198 active studies seeking participants at present."

Answered by AI

Which demographic is most likely to benefit from participating in this trial?

"Patients that meet the criteria of anemia, sickle cell and are between 18-40 years in age may be eligible for this trial. Currently, 5 participants have been recruited so far."

Answered by AI

Does the FDA sanction a combination of Motixafortide and Natalizumab after administration of Motixafortide?

"Our evaluation at Power awards Motixafortide and the combination of Motixafortide + Natalizumab a score of 1, as this is an experimental Phase 1 trial with limited evidence for their safety or effectiveness."

Answered by AI