← Back to Search

Cell Therapy

Bone Marrow Transplant for Sickle Cell Disease

Q: What effects has the utilization of CD3/CD19 depleted leukocytes had on patient treatment?

<p><a href="https://www.withpower.com/clinical-trials/sickle-cell-anemia">Sickle cell anemia</a>, DLBCL, and inoperable <a href="https://www.withpower.com/clinical-trials/ovarian-cancer">ovarian cancer</a> may be addressed by utilizing CD3/CD19 depleted leukocytes.</p>

Phase 1 & 2

Recruiting

Led By Paul Szabolcs, MD

Research Sponsored by Paul Szabolcs

Eligibility Criteria Checklist

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

Must have

Agreement to use FDA approved birth control for specified duration

Ages 5 years to 40 years, at time of consent

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline through study completion, an average of 2 years

Awards & highlights

Study Summary

This trial will test whether stem cell transplants from mismatched donors can help people with severe sickle cell disease or other transfusion-dependent anemias.

Who is the study for?

This trial is for people aged 5-40 with severe sickle cell disease or other anemias needing many blood transfusions and showing organ damage. They must have tried Hydroxyurea without success, not be pregnant, HIV negative, and agree to birth control post-transplant. A suitable donor match is also required.Check my eligibility

What is being tested?

The study tests if stem cell transplants from mismatched unrelated or related donors can help treat severe sickle cell disease and other anemias requiring frequent transfusions. It aims to reduce graft-versus-host disease using a T-cell depleted method in the transplant process.See study design

What are the potential side effects?

Potential side effects may include immune system reactions due to the depletion of T-cells, infusion-related reactions from drugs like Alemtuzumab, Rituximab, Fludarabine; as well as possible organ toxicity from Thiotepa.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I agree to use approved birth control methods for the required time.

Select...

I am between 5 and 40 years old.

Select...

I have Sickle Cell Disease or beta-thalassemia with complications or need regular blood transfusions.

Select...

My donor is a match for me.

Select...

I have tried hydroxyurea for my sickle cell disease, but it didn't work.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline through study completion, an average of 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline through study completion, an average of 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through study completion, an average of 2 years for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Acute Graft versus host disease

Bone Transplantation

Graft rejection

+1 more

Secondary outcome measures

Adult Sickle Cell Quality of Life Measurement System (ASCQ)

Cytomegalovirus (CMV) infection

Donor Cell Engraftment

+11 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Hematopoietic Stem Cell TransplantationExperimental Treatment7 Interventions

All patients will receive a CD3+/CD19+ depleted stem cell transplant. In this study, the investigators will use HLA mismatched unrelated or haploidentical related donor peripheral blood stem cells. Prior to transplantation, the marrow (90-95%) will be negatively selected for CD3/CD19 using the ClinicMACs® depletion device. The remaining (5-10%) will undergo CD45+RA+ depletion and be frozen for future use as an immune boost. Subjects will undergo hematopoietic stem cell transplant utilizing CD3+/CD19+ depleted cells following conditioning therapy.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Thiotepa

2008

Completed Phase 3

~2210

Hydroxyurea

2006

Completed Phase 4

~3620

Rituximab

1999

Completed Phase 4

~1880

Alemtuzumab

2004

Completed Phase 4

~1890

Fludarabine

2012

Completed Phase 3

~1100

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Paul SzabolcsLead Sponsor

7 Previous Clinical Trials

227 Total Patients Enrolled

Paul Szabolcs, MDPrincipal Investigator - University of Pittsburgh

University of Pittsburgh

7 Previous Clinical Trials

224 Total Patients Enrolled

Media Library

T-Cell Depleted Alternative Donor Bone Marrow Transplant (Cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03653338 — Phase 1 & 2

Sickle Cell Anemia Research Study Groups: Hematopoietic Stem Cell Transplantation

Sickle Cell Anemia Clinical Trial 2023: T-Cell Depleted Alternative Donor Bone Marrow Transplant Highlights & Side Effects. Trial Name: NCT03653338 — Phase 1 & 2

T-Cell Depleted Alternative Donor Bone Marrow Transplant (Cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03653338 — Phase 1 & 2

Frequently Asked Questions

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

Am I eligible to volunteer for this experiment?

"This clinical trial seeks to enroll up to 5 participants who have been diagnosed with thalassemia, and are aged between five and forty years old."

Answered by AI

What effects has the utilization of CD3/CD19 depleted leukocytes had on patient treatment?

"Sickle cell anemia, DLBCL, and inoperable ovarian cancer may be addressed by utilizing CD3/CD19 depleted leukocytes."

Answered by AI

What key outcomes is this clinical trial seeking to achieve?

"Over the course of two years, this medical trial will primarily monitor for Acute Graft versus host disease. Additionally, inspecting Sickle cell Disease recurrence, neutrophil recovery times, and donor cell engraftment are all secondary goals for this study. To qualify as a success case in these regards, subjects must present with ≥ 0.5 x 103/μL neutrophils over three days; HgbS>25%; an acute chest syndrome; > 50% donor chimerism at 180 days; and ≥ 5% donor cells on day +42 and ≥ 10% donor cells on day +100 respectively."

Answered by AI

Are there any vacancies available for potential participants in this research?

"Affirmative. According to clinicaltrials.gov, this research project was first made available on August 2nd 2018 and is still actively enrolling participants with a goal of recruiting 5 people at 1 site."

Answered by AI

Are the elderly in their mid-seventies being included as participants for this trial?

"This trial seeks participants aged between 5 and 40 years old. For those under 18, there are 454 alternate studies available. Similarly, patients over 65 can choose from 911 other clinical trials."

Answered by AI

Has there been previous research involving the depletion of CD3/CD19 leukocytes?

"Presently, 801 active studies exist for CD3/CD19 depleted leukocytes with 124 of them being in the advanced Phase 3. Most are located around Philadelphia, Pennsylvania; however, trials can be found at 17734 sites globally."

Answered by AI

What is the participant threshold for this trial?

"Indeed, clinicaltrials.gov affirms that this research is presently searching for subjects to enrol. The experiment was initially announced on August 2nd 2018 and revised most recently on May 4th 2022; it seeks 5 patients at a single site."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Paul Szabolcs 2018. "T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03653338.

All > Sickle Cell Disease > Sickle Cell Anemia