Stem Cell Transplant for Aplastic Anemia

Overseen By Timothy Krepski

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Masonic Cancer Center, University of Minnesota

Disqualifiers: Acute hepatitis, Pregnant, Uncontrolled infection, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

61 Participants Needed

What You Need to Know Before You Apply

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.

Who Is on the Research Team?

Jakub Tolar, MD

Principal Investigator

University of Minnesota

Are You a Good Fit for This Trial?

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.

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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Preparative Regimen

Participants receive a fludarabine-based preparative regimen including alemtuzumab, cyclophosphamide, and fludarabine, followed by a stem cell transplant

6 weeks

Engraftment Monitoring

Participants are monitored for neutrophil and platelet engraftment

42 days

Follow-up

Participants are monitored for regimen-related mortality, acute and chronic graft-versus-host disease, and secondary malignancies

1 year

What Are the Treatments Tested in This Trial?

Interventions

• Alemtuzumab
• Anti-thymocyte globulin
• Cyclophosphamide
• Fludarabine
• Hematopoietic Stem Cell Transplant
• Total Body Irradiation

Trial Overview The 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.

How Is the Trial Designed?

Group I: Arm E: Severe Aplastic Anemia (SAA), PTCy platform Experimental Treatment 6 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 platform Experimental Treatment 4 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 Treatment 6 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 Treatment 6 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 Treatment 4 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

Approved in European Union as Lemtrada for:

• Multiple sclerosis

Approved in European Union as Campath for:

• Chronic lymphocytic leukemia

Who Is Running the Clinical Trial?

Masonic Cancer Center, University of Minnesota

Lead Sponsor

Trials

285

Recruited

15,700+