An interventional study of Reduced Toxicity Ablative Regimen and Reduced Intensity Preparative Regimen in Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia and Diamond Blackfan Anemia, sponsored by Masonic Cancer Center, University of Minnesota. Terminated at 1 site in United States. Open to participants aged Up to 55 Years. Per ClinicalTrials.gov, last updated 2025-03-03.
Sponsored by Masonic Cancer Center, University of Minnesota · Not applicable, Interventional, and Treatment
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Among 733 studies in Anemia, Sickle Cell with a site in United States — 134 open to participants now, 185 under way, 16 of them phase 3. Anemia, Sickle Cell · United States.
Among the 733 studies in Anemia, Sickle Cell with a site in United States, this one is the 250th largest by enrollment of the 559 interventional ones that give a count, is one of 36 that started in 2014, is one of 165 with no phase, and was the 270th registered.
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
Browse Anemia, Sickle Cell studies →134 of the 733 studies in Anemia, Sickle Cell with a site in United States are open now. Recruiting first, the newest first:
University of Maryland, Baltimore · Sites in United States
Ages 18–88
Mohsen Saidinejad · Sites in United States
Ages 5–20
St. Jude Children's Research Hospital · Sites in United States
Ages up to 22
Emory University · Sites in United States
Ages 8–18
University of Alabama at Birmingham · Sites in United States
Ages 18–50 · Women only
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 177 Anemia, Sickle Cell studies recruiting now, 64 (36%) set no upper age limit and 97 (55%) stop at 75 or younger; 127 take children under 18; 29 take healthy volunteers.
This study: Up to 55 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
733 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 158 · Phase 2 228 · Phase 3 57 · Phase 4 20
First in 1984; this study’s year is marked. 36 started in 2014.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 38 is close to the median of 40 across 769 interventional studies indexed under Anemia, Sickle Cell.
Median enrollment 31 across the 559 interventional studies, 100 across the 168 observational. Of the 506 that completed or were terminated, 207 (41%) have results posted.
This tab as a report to print or save as PDF → With an account.
168 of 733 have an industry lead sponsor. The 10 most frequent:
Masonic Cancer Center, University of Minnesota is the lead sponsor of 286 studies on the registry; 35 are open to participants now.
Of its 39 completed or terminated interventional studies of FDA-regulated products, 28 (72%) have results posted.
Masonic Cancer Center, University of Minnesota has 4 studies filed under Anemia, Sickle Cell — fewer than ten, so there is no comparison to draw.
57 of the studies in Anemia, Sickle Cell with a site in United States that are under way reach primary completion within a year, 7 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07247188Phase 3SanofiStudy of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease | 2027-01-07 |
| NCT04293185Phase 3bluebird bioA Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease | 2027-05 |
| NCT06989567Phase 3Asklepion Pharmaceuticals, LLCA Study of Intravenous L-Citrulline in Patients Aged 6-21 Years Old With Sickle Cell Disease Presenting to Emergency Departments in Acute Vaso-Occlusive Crisis | 2027-06 |
| NCT05329649Phase 3Vertex Pharmaceuticals IncorporatedEvaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) | 2027-06-06 |
| NCT05477563Phase 3Vertex Pharmaceuticals IncorporatedEvaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | 2027-06-09 |
Add all 57 to your calendar (.ics) → With an account.
Among the 733 studies in Anemia, Sickle Cell with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
For use in patients with a matched sibling donor or unrelated UCB donor and DBA patients who are \<12 years and/or have mild/moderate iron exposure.
Drug: Reduced Toxicity Ablative Regimen
For use in patients with unrelated donor bone marrow and for DBA patients who are \>12 years and/or have significant iron exposure.
Drug: Reduced Intensity Preparative Regimen
For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.
Drug: Myeloablative Preparative Regimen
* Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan * Stem Cell Infusion Day 0
* Alemtuzumab * Cyclophosphamide * Fludarabine * Total Body Irradiation (TBI) * Stem Cell Infusion Day 0
* Alemtuzumab * Cyclophosphamide * Busulfan * Stem Cell Infusion Day 0
incidence of graft failure
Time frame: 42 days
overall survival
Time frame: 6 months, 1 and 2 years
disease free survival
patient no longer needing red blood cell transfusion and/or a hemoglobin S level at that of the donor ( sickle cell disease only)
Time frame: 6 months, 1 and 2 years
The record gives a start of Sep 2014, a primary completion of Nov 2024 and a completion of Nov 2024. It does not say whether these dates are actual or estimated.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Feb 2025. You cannot join it, but the record below documents what was studied.
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Masonic Cancer Center, University of Minnesota →