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Hematopoietic Stem Cell Transplant for Sickle Cell Disease

NCT02065596 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2024-04-29

No results posted yet for this study

Summary

This is a study of patients with sickle cell disease. It aims to find out if people with sickle cell disease can be cured by changing their immune system before they have blood stem cell transplants. Doctors will give patients a new drug (fludarabine) to see if this drug changes patients immune system and reduces the patient's cells (host) from rejecting donor cells (graft) after the patient gets a Hematopoietic (blood) stem cell transplant.

Conditions

Interventions

DRUG

Fludarabine

the study will begin with enrollment of an initial safety cohort of at least 10 subjects at the lowest dose, after which enrollment will pause until the dose-limiting toxicity (DLT) period has been completed. If a patient experiences DLT, defined as failure to engraft. In which case, the patient may be advanced to two higher doses.

PROCEDURE

Hematopoietic Stem Cell Transplant (HSCT)

Three weeks after Immunomodulation patients will be infused with matched bone marrow from a sibling, unrelated donor, haploidentical donor, or cord blood. Patients will be followed for the following year.

Sponsors & Collaborators

  • Case Comprehensive Cancer Center

    lead OTHER

Principal Investigators

  • Molly Gallogly, MD, PhD · Case Comprehensive Cancer Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-10-19
Primary Completion
2023-02-17
Completion
2023-02-17
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Diseases

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 NCT02065596 on ClinicalTrials.gov