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Alkylating agents

Stem Cell Transplant for Sickle Cell Disease

Phase 2
Recruiting
Led By Damiano Rondelli, MD
Research Sponsored by University of Illinois at Chicago
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients with sickle cell disease who have the following complications: stroke or central nervous system event lasting longer than 24 hours, frequent vaso-occlusive pain episodes, recurrent episodes of priapism, acute chest syndrome with recurrent hospitalizations, red-cell alloimmunization, bilateral proliferative retinopathy with major visual impairment, osteonecrosis of 2 or more joints, sickle cell nephropathy, pulmonary hypertension
Karnofsky performance status of 60 or higher
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to day +60
Awards & highlights

Study Summary

This trial is looking at a new way to do a stem cell transplant for sickle cell disease. The old way is not working as well as they'd like, so they are trying something new. This study is to see if the new way is better.

Who is the study for?
This trial is for people aged 16-60 with severe sickle cell disease, who have complications like stroke, recurrent pain episodes, or organ damage. They must not be pregnant, HIV-negative, and without a fully HLA-matched sibling donor but have an HLA-haploidentical relative willing to donate stem cells.Check my eligibility
What is being tested?
The study tests a new way of transplanting stem cells from half-matched relatives in patients with aggressive sickle cell disease. It involves low-dose body radiation and drugs like fludarabine and cyclophosphamide before the transplant, followed by post-transplant medication to help the body accept the new cells.See study design
What are the potential side effects?
Possible side effects include reactions to medications such as mycophenolate mofetil and sirolimus (like nausea or diarrhea), risks from total body irradiation (like fatigue or skin changes), and complications related to stem cell infusion (such as infection risk).

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have sickle cell disease with serious complications like stroke, frequent pain, or vision loss.
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I can care for myself but cannot do normal activities or work.
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My heart pumps well enough (ejection fraction is 40% or more).
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My lungs work well enough for treatment (DLCO ≥ 50% predicted).
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I have a family member who is a partial genetic match for me.
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I am between 16 and 60 years old.
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I do not have a sibling who is a complete HLA match for me.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to day +60
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to day +60 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Estimate the number of patients who engraft by Day +60
Secondary outcome measures
Adverse Effects
Disease free survival
Overall survival

Trial Design

1Treatment groups
Experimental Treatment
Group I: Subject treatmentExperimental Treatment7 Interventions
Patients will receive the following conditioning regimen: ATG, fludarabine (6 days before stem cell infusion), cyclophosphamide, and total body irradiation. The stem cell product will be infused according to BMT unit policy. Patients will also receive GVHD prophylaxis which will consist of cyclophosphamide, sirolimus, and mycophenolate mofetil according to the protocol. Post-transplant evaluation will be done as per standard care with study data collected at days 30, 60, 100, 180, 365, and annually thereafter.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Mycophenolate mofetil
FDA approved
Antithymocyte immunoglobulin (rabbit)
FDA approved
Stem cell infusion
2002
Completed Phase 3
~60
Sirolimus
FDA approved
Fludarabine
FDA approved
Cyclophosphamide
FDA approved
Total body irradiation
2008
Completed Phase 3
~1080

Find a Location

Who is running the clinical trial?

University of Illinois at ChicagoLead Sponsor
604 Previous Clinical Trials
1,558,945 Total Patients Enrolled
Damiano Rondelli, MDPrincipal Investigator - University of Illinois at Chicago
University of Illinois at Chicago Eye & Ear Infirmary
6 Previous Clinical Trials
235 Total Patients Enrolled

Media Library

Cyclophosphamide (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT03121001 — Phase 2
Sickle Cell Disease Research Study Groups: Subject treatment
Sickle Cell Disease Clinical Trial 2023: Cyclophosphamide Highlights & Side Effects. Trial Name: NCT03121001 — Phase 2
Cyclophosphamide (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03121001 — Phase 2

Frequently Asked Questions

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

Is recruiting underway for this research effort?

"Correct. According to the clinicaltrials.gov website, this investigation is still open for enrollment and was initially announced on March 20th 2017. Furthermore, 50 participants are required from 1 site before October 27 2022."

Answered by AI

What is the scale of participation in this clinical experiment?

"Affirmative. The information found on clinicaltrials.gov confirms that this experiment is actively enrolling participants, with a post date of March 20th 2017 and an update from October 27th 2022. 50 individuals need to be recruited from 1 medical facility for the study's conclusion."

Answered by AI

Is this research open to participants below the age of 85?

"To participate eligibly in this trial, the patient must be between 16 and 60 years of age. Meanwhile, there are 499 trials available for individuals under 18 as well as 868 studies open to seniors aged 65 or older."

Answered by AI

What past research has occurred in regards to Stem cell infusion?

"Currently, 1039 investigations related to stem cell infusion are underway with 186 of them in their third phase. Though there is a concentration of trials centered around the city Philadelphia, Pennsylvania, this medical intervention can be accessed at 2920 different sites worldwide."

Answered by AI

What conditions are typically remedied through stem cell infusion?

"Stem cell infusion is a common therapeutic approach used to facilitate organ transplants, as well as address immunosuppression, multiple sclerosis, leukemia, myelocytic and acute conditions."

Answered by AI

What are the eligibility criteria to partake in this research project?

"This trial, which is recruiting a maximum of 50 participants, requires potential enrollees to be between 16 and 60 years old with anemia or sickle cell."

Answered by AI

What risks have been observed with Stem cell infusion treatments?

"The safety of stem cell infusion was evaluated and given a rating of 2 since this is merely a Phase 2 trial with preliminary evidence towards its security but no data on efficacy."

Answered by AI

Who else is applying?

What state do they live in?
New Jersey
What site did they apply to?
University of Illinois at Chicago
What portion of applicants met pre-screening criteria?
Met criteria
~9 spots leftby Nov 2025