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Sickle Cell DiseasePhase 2NCT03121001

Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

A Phase 2 study testing ATG, fludarabine and cyclophosphamide in people with Sickle Cell Disease, run by University of Illinois Chicago at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v. cyclophosphamide conditioning prior to human leukocyte antigen (HLA)-haploidentical hematopoietic stem cell transplant (HSCT). Read more about this study

Who is it for?
Ages 16 to 60 years
Conditions the study is for
Sickle Cell Disease
What is being tested?
ATG, fludarabine and cyclophosphamide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
1 siteUnited States
Study dates
20 Mar 2017to May 2029
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 16 to 60 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Patient Eligibility:

  1. Patients with sickle cell disease are eligible if they have any of the following complications: 1.1 Stroke or central nervous system event lasting longer than 24 hours 1.2 Frequent vaso-occlusive pain episodes, defined as ≥ 3 per year requiring emergency room, acute care center, hospital admissions, or home bedrest leading to absence from work or school. 1.3 Recurrent episodes of priapism, defined as ≥ 2 per year requiring emergency room visits 1.4 Acute chest syndrome with recurrent hospitalizations, defined as ≥ 2 lifetime events 1.5 Red-cell alloimmunization (≥ 2 antibodies) during long-term transfusion therapy 1.6 Bilateral proliferative retinopathy with major visual impairment in at least one eye 1.7 Osteonecrosis of 2 or more joints 1.8 Sickle cell nephropathy, defined by a GFR < 90mL/min/1.73m2 or the presence of macroalbuminuria (urine albumin > 300 mg/g creatinine) 1.9 Pulmonary hypertension, defined by a mean pulmonary arterypressure >25mmHg
  2. Age 16-60 years
  3. Karnofsky performance status of 60 or higher (Appendix A)
  4. Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40%
  5. Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥ 50% predicted (after adjustment for hemoglobin concentration)
  6. Estimated GFR ≥ 50mL/min/1.73m2 as calculated by the modified MDRD equation
  7. ALT ≤ 3x upper limit of normal
  8. HIV-negative
  9. Patient is not pregnant
  10. Patient is able and willing to sign informed consent
  11. Patient does not have a fully HLA-matched sibling donor
  12. Patient has an HLA-haploidentical relative

Donor Eligibility Relatives (parents, offspring, siblings, aunts/uncles, cousins) will be tested by molecular typing of HLA class I (A, B, and C) and class II (DRB1) at low resolution. Only those that are an HLA-haploidentical match (≥ 4/8) will be considered as a potential donor. NOTE: If during testing, a fully HLA-matched sibling donor is found and is willing to donate his/her stem cells, the potential subject will not be eligible for this protocol.

Donor consent will be obtained as per standard protocol of the bone marrow transplant unit.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Damiano Rondelli, MD contact

312 413-3547

drond@uic.edu

United States1 site, 1 recruiting

  • University of Illinois at Chicago

    Chicago, Illinois, 60612

    Damiano Rondelli, MD, 312-413-3547, drond@uic.edu

    Recruiting

Other trials in United States

About this study

Official title: A Phase II Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v. cyclophosphamide conditioning prior to human leukocyte antigen (HLA)-haploidentical hematopoietic stem cell transplant (HSCT).

The primary objective of the study is to determine the engraftment at Day +60 following HLA-haploidentical hematopoietic stem cell transplant protocol using immunosuppressive agents and low-dose total body irradiation (TBI) for conditioning and post-transplant cyclophosphamide in patients with sickle cell disease.

Being tested
ATG, fludarabine, cyclophosphamide, Total body irradiation, Stem cell infusion
Medical subject headings
Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ATGDrug

    Also called Thymoglobulin®

    0.5 mg/kg IV on day -9, and 2 mg/kg on days -8 and day -7

  • fludarabineDrug

    30 mg/m2 IVPB daily for day -6 (6 days before stem cell infusion) through day -2

  • cyclophosphamideDrug

    14.5 mg/kg IV on days -6 and -5 and 50 mg/kg/d on days +3 and +4

  • Total body irradiationRadiation

    3 Gy on day -1

  • Stem cell infusionProcedure or surgery

    Stem cell product infused according to BMT unit policy on day 0.

  • SirolimusDrug

    loading dose of 15 mg followed by 5 mg per day on day +5

  • mycophenolate mofetilDrug

    1 g every 8 h (until day 35) will be started on day 5

Study groups in NCT03121001
Study groupRoleWhat they receive
Subject treatmentExperimentalPatients 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.
Receives: Drug: ATG; Drug: fludarabine; Drug: cyclophosphamide; Radiation: Total body irradiation; Procedure: Stem cell infusion; Drug: Sirolimus; Drug: mycophenolate mofetil

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Estimate the number of patients who engraft by Day +60

    Patients who achieve < 5% peripheral blood donor chimerism by Day +30 and do not have a Day +60 measure will be regarded as failing to achieve full donor chimerism by Day +60; patients who achieve > 5% donor chimerism by Day +30 but do not have a Day +60 measure will be considered nonevaluable for the primary endpoint.

    Measured: Up to Day +60

Secondary outcomes (3)

  1. Disease free survival

    Using the Kaplan-Meier method, the probability of EFS will be estimated and reported with 90% confidence intervals. The proportion of patients who are alive will also be estimated with a 90% exact binomial confidence interval. Cumulative incidences of transplant related mortality will be estimated separately using Grey's method.

    Measured: Up to Day +60

  2. Overall survival

    Using the Kaplan-Meier method, the probability of overall survival will be estimated and reported with 90% confidence intervals. The proportion of patients who are alive will also be estimated with a 90% exact binomial confidence interval. Cumulative incidences of transplant related mortality will be estimated separately using Grey's method.

    Measured: Up to Day +60

  3. Adverse Effects

    The cumulative incidence of acute (grade II-IV, grade III-IV) and chronic GVHD will be estimated through competing-risk analysis using Grey's method, wherein graft failure, and death are competing risks for GVHD. Other selected toxicities (including rates of infection) will be reported descriptively.

    Measured: Up to Day +60

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Apr 2017
  2. Study start20 Mar 2017
  3. Primary completionMay 2029 (estimated)
  4. Study completionMay 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03121001
Sponsor's protocol ID
2016-1152
Status last verified
Jul 2026
First submitted
14 Apr 2017
Last updated
8 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ATG, fludarabine and cyclophosphamide in people with Sickle Cell Disease, run by University of Illinois Chicago at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 8 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Damiano Rondelli, MD (312 413-3547, drond@uic.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Estimate the number of patients who engraft by Day +60 (time frame: Up to Day +60). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03121001.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2026.

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