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Hematopoietic Stem Cell Transplantation

ECT-001-CB (UM171-Expanded Cord Blood Transplant) for Sickle Cell Disease

Phase 1
Waitlist Available
Research Sponsored by ExCellThera inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 24-months
Awards & highlights

Study Summary

US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease

Eligible Conditions
  • Sickle Cell Disease
  • Umbilical Cord Blood
  • Hematopoiesis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 24-months
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 24-months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Evaluate feasibility of a successfully performing ECT-001-CB transplantation in the context of SCD.
Evaluate feasibility of finding a suitable ECT-001-CB graft for transplantation in the context of SCD.
Evaluate the Safety of ECT-001-CB by calculating incidence of acute and chronic GvHD
+4 more
Secondary outcome measures
Determine the pharmacologic effect of ECT-001-CB by evaluating donor chimerism
Determine the pharmacologic effect of ECT-001-CB by measuring sickle hemoglobin (HbS) in peripheral blood.
Evaluate impact of ECT-001-CB on SCD-related events during the study period.

Trial Design

1Treatment groups
Experimental Treatment
Group I: ECT-001-CBExperimental Treatment1 Intervention
An Umbilical Cord Blood for transplant will undergo CD34+ selection and expansion. The CD34- fraction is infused on Day +1 post-transplant. Patients will receive standard supportive care and GVHD prophylaxis

Find a Location

Who is running the clinical trial?

ExCellThera inc.Lead Sponsor
4 Previous Clinical Trials
92 Total Patients Enrolled
California Institute for Regenerative Medicine (CIRM)OTHER
63 Previous Clinical Trials
3,148 Total Patients Enrolled
Medical MonitorStudy DirectorExCellThera inc.
1,649 Previous Clinical Trials
979,725 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease
2021. "US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04594031.
~0 spots leftby Apr 2025
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