Hematopoietic Cell Transplantation for Sickle Cell Disease

Phase-Based Progress Estimates
City of Hope Comprehensive Cancer Center, Duarte, CASickle Cell DiseaseHematopoietic Cell Transplantation - Procedure
12 - 40
All Sexes
What conditions do you have?

Study Summary

This trial is testing the safety and effectiveness of a new conditioning regimen for stem cell transplantation in patients with sickle cell disease. The conditioning regimen includes total marrow and lymphoid irradiation (TMLI) and alemtuzumab. Alemtuzumab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. The goal of this trial is to reduce organ damage that can be caused by radiation and decrease the risk of graft-versus-host disease (GVHD).

Eligible Conditions
  • Sickle Cell Disease

Treatment Effectiveness

Study Objectives

2 Primary · 6 Secondary · Reporting Duration: Up to 2 years

Baseline only (pre-conditioning)
Bone marrow environment inflation - levels of inflammatory cytokines
Bone Marrow
Year 2
Disease-free Survival (DFS)
Year 2
Event-free survival (EFS)
Year 2
Overall survival (OS)
Up to 1 year
Treatment response on bone marrow environment and CBF
Up to 2 years
Immune cell reconstitution
Platelet engraftment
Quality of life - PedsQL
Time to acute graft-versus-host disease (grades 2-4 and 3-4) until day +100 after transplant
Time to chronic graft-versus-host disease for up to one year after transplant
Day 100
Incidence of adverse events

Trial Safety

Side Effects for

Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)
10%Death following disease progression post transplant
5%Typhlitis & Bowel Perforation
5%Death following progression of GVHD
5%Death: Sepsis/Renal failure/ with history of GVHD
5%Minimal hydronephosis
5%Renal failure
5%Cardiac Arrhythmia and Seizure
5%Perforated sigmoid diverticulitis
5%Acute Pulmonary Embolism
5%Severe abnormal pain due to gut GVH
5%Tumor lysis syndrome
5%Increased creatinine
This histogram enumerates side effects from a completed 2010 Phase 2 trial (NCT00060424) in the Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis) ARM group. Side effects include: Neutropenia with 48%, Death following disease progression post transplant with 10%, Hyperbilirubinemia with 10%, Thrombocytopenia with 10%, Hypoxia with 10%.

Trial Design

1 Treatment Group

Treatment (TMLI, alemtuzumab)
1 of 1

Experimental Treatment

20 Total Participants · 1 Treatment Group

Primary Treatment: Hematopoietic Cell Transplantation · No Placebo Group · Phase 1

Treatment (TMLI, alemtuzumab)Experimental Group · 4 Interventions: Hematopoietic Cell Transplantation, Sirolimus, Alemtuzumab, Intensity-Modulated Radiation Therapy · Intervention Types: Procedure, Drug, Drug, Radiation
First Studied
Drug Approval Stage
How many patients have taken this drug
Hematopoietic Cell Transplantation
Completed Phase 2
Completed Phase 4
Completed Phase 4
Intensity-Modulated Radiation Therapy
Completed Phase 3

Trial Logistics


Participation is compensated

You will be compensated for participating in this trial.

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: up to 2 years

Who is running the clinical trial?

National Cancer Institute (NCI)NIH
13,153 Previous Clinical Trials
41,162,904 Total Patients Enrolled
City of Hope Medical CenterLead Sponsor
524 Previous Clinical Trials
2,249,958 Total Patients Enrolled
Joseph RosenthalPrincipal InvestigatorCity of Hope Comprehensive Cancer Center
1 Previous Clinical Trials
24 Total Patients Enrolled

Eligibility Criteria

Age 12 - 40 · All Participants · 39 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a condition called osteonecrosis where two or more of your joints have died despite receiving treatment.
You have experienced acute chest syndrome, which is a serious complication of sickle cell disease, within the past two years despite receiving treatment.
You have a condition called priapism, which causes painful and prolonged erections that require medical treatment.

Frequently Asked Questions

Is this experiment enlisting participants aged twenty or above?

"In order to be eligable for this medical trial, applicants must between 12 and 40 years old. There are 131 trials available exclusively to those under 18 while 86 of the studies are accessible only by individuals above 65 years of age." - Anonymous Online Contributor

Unverified Answer

Can I take part in this experimental procedure?

"This clinical trial seeks twenty individuals aged between 12 and 40 who currently suffer from anemia due to sickle cell. In addition, patients must be willing to assent as per institutional guidelines; registered into the REMS program; possess an ECOG performance status lower than or equal to 2; have experienced a stroke or neurological deficit lasting longer than 24 hours with increased transcranial Doppler velocity greater than 200 m/s; suffered recurrent priapism necessitating medical intervention; provided informed consent of their own volition or by legal representative if applicable; experienced one more episodes of acute chest syndrome in the past two years despite supportive care measures" - Anonymous Online Contributor

Unverified Answer

Has the FDA sanctioned Hematopoietic Cell Transplantation?

"Our team has rated the safety of Hematopoietic Cell Transplantation a 1 as this is currently in its Phase 1 trial, which suggests that there are only limited data to support its efficacy and safety." - Anonymous Online Contributor

Unverified Answer

Does this clinical experiment still have the capacity to enroll new participants?

"Affirmative. The clinical trial registry contains evidence that this particular experiment has been recruiting since July 13th 2022, with the most recent update on July 31st 2022. Around 20 patients are being sought from a single medical facility." - Anonymous Online Contributor

Unverified Answer

What outcomes is this clinical trial aiming to achieve?

"This trial will have two-year follow up and the primary outcome being monitored is incidence of adverse events. Secondary endpoints include cumulative incidence of chronic GVHD, aGVHD within 100 days post-transplant, and platelet engraftment (defined as independence from transfusions with more than 20x10^9/L)." - Anonymous Online Contributor

Unverified Answer

How many participants are sought for this research program?

"Affirmative. The information on clinicaltrials.gov indicates that this medical trial is in a state of active enrollment, having been initially posted on July 13th 2022 and updated most recently on the 31st of July. They are hoping to acquire 20 participants from one site." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.