Hematopoietic Stem Cell Transplant for Sickle Cell Disease

Phase 1

Waitlist Available

Led By Kirsten Boughan, DO

Research Sponsored by Case Comprehensive Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing if fludarabine can help patients with sickle cell disease accept new stem cells from a donor. The drug weakens the immune system to reduce the chances of rejecting the new cells. The goal is to see if this approach can improve or cure their condition.

Eligible Conditions

Sickle Cell Disease
Sickle cell disease

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Probability of Engraftment

Secondary study objectives

Cerebral Vasculopathy

Disease Progression

Erythropoiesis

+9 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Immunomodulation with Fludarabine prior to HSCTExperimental Treatment2 Interventions

Fludarabine given beginning at 25mgm/m2 three times per day. Patients may be escalated up to 25mgm/m2 five times per day depending on dose-limiting toxicity

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fludarabine

2012

Completed Phase 4

~1860

Hematopoietic Stem Cell Transplant (HSCT)

2015

Completed Phase 2

~40