Stem Cell Transplant for Aplastic Anemia

Recruiting in Palo Alto (17 mi)

Overseen byJakub Tolar, MD

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Waitlist Available

Sponsor: Masonic Cancer Center, University of Minnesota

No Placebo Group

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

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 (Monoclonal Antibodies)
Anti-thymocyte globulin (Immunosuppressant)
Cyclophosphamide (Alkylating agents)
Fludarabine (Alkylating agents)
Hematopoietic Stem Cell Transplant (Stem Cell Transplant)
Total Body Irradiation (Radiation)

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

2Treatment groups

Experimental Treatment

Group I: Treatment for Severe Aplastic AnemiaExperimental Treatment5 Interventions

Fludarabine based preparative regimen which includes: cyclophosphamide, fludarabine, rabbit ATG and total body irradiation. Followed by stem cell transplant.

Group II: Treatment Plan for Dyskeratosis CongenitaExperimental Treatment4 Interventions

Fludarabine based preparative regimen, including alemtuzumab, cyclophosphamide, fludarabine, and total body irradiation, followed by stem cell transplant for the treatment of dyskeratosis congenita.

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

🇺🇸 Approved in United States as Campath for: