61 Participants Needed

Stem Cell Transplant for Aplastic Anemia

TK
Overseen ByTimothy Krepski
Age: Any Age
Sex: Any
Trial Phase: Academic
Sponsor: Masonic Cancer Center, University of Minnesota
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

What is the purpose of this trial?

Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens are included based on disease and donor type.

Research Team

JT

Jakub Tolar, MD

Principal Investigator

University of Minnesota

Eligibility Criteria

This trial is for people aged 0-70 with dyskeratosis congenita or severe aplastic anemia needing blood transfusions. Participants must have specific blood counts, organ function levels, and genetic mutations related to the conditions. Pregnant individuals, those with uncontrolled infections, certain liver issues, or a diagnosis of Fanconi anemia are excluded.

Inclusion Criteria

My kidney function is at least 30% of what is expected.
Your body is rejecting a transplanted organ, as shown by a specific test.
My heart pumps well and I don't have heart failure.
See 27 more

Exclusion Criteria

I have severe liver damage or hepatitis.
I have received radiation therapy before (applies only if I have severe aplastic anemia).
I have been diagnosed with Fanconi anemia.
See 3 more

Treatment Details

Interventions

  • Alemtuzumab
  • Anti-thymocyte globulin
  • Cyclophosphamide
  • Fludarabine
  • Hematopoietic Stem Cell Transplant
  • Total Body Irradiation
Trial OverviewThe study tests a fludarabine-based regimen followed by stem cell transplant from donors in patients with bone marrow failure due to dyskeratosis congenita or aplastic anemia. It compares three regimens based on disease severity and donor match.
Participant Groups
5Treatment groups
Experimental Treatment
Group I: Arm E: Severe Aplastic Anemia (SAA), PTCy platformExperimental Treatment6 Interventions
Fludarabine based preparative regimen which includes: cyclophosphamide, fludarabine, rabbit ATG and total body irradiation. Followed by stem cell transplant.
Group II: Arm D: Dyskeratosis Congenita (DKC), PTCy platformExperimental Treatment4 Interventions
Fludarabine based preparative regimen, including alemtuzumab, cyclophosphamide, fludarabine, followed by stem cell transplant for the treatment of dyskeratosis congenita.
Group III: Arm C: Severe Aplastic Anemia (matched related donor)Experimental Treatment6 Interventions
Fludarabine based preparative regimen which includes: cyclophosphamide, fludarabine, rabbit ATG and total body irradiation. Followed by stem cell transplant.
Group IV: Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)Experimental Treatment6 Interventions
Fludarabine based preparative regimen which includes: cyclophosphamide, fludarabine, rabbit ATG and total body irradiation. Followed by stem cell transplant.
Group V: Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)Experimental Treatment4 Interventions
Fludarabine based preparative regimen, including alemtuzumab, cyclophosphamide, fludarabine, followed by stem cell transplant for the treatment of dyskeratosis congenita.

Alemtuzumab is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Campath for:
  • Chronic lymphocytic leukemia
  • Multiple sclerosis
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Approved in European Union as Lemtrada for:
  • Multiple sclerosis
🇪🇺
Approved in European Union as Campath for:
  • Chronic lymphocytic leukemia

Find a Clinic Near You

Who Is Running the Clinical Trial?

Masonic Cancer Center, University of Minnesota

Lead Sponsor

Trials
285
Recruited
15,700+