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Monoclonal Antibodies

Stem Cell Transplant for Aplastic Anemia

N/A

Waitlist Available

Led By Jakub Tolar, MD

Research Sponsored by Masonic Cancer Center, University of Minnesota

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Renal: Glomerular filtration rate (GFR) ≥30% predicted

Acceptable hematopoeitic stem cell donor

Must not have

Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy

Diagnosis of Fanconi anemia based on DEB

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing different ways to prepare for and do a stem cell transplant for people with dyskeratosis congenita or severe aplastic anemia.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects include immune reactions from the body against the new cells (graft-versus-host disease), infection risks due to weakened immunity post-transplant, chemotherapy-related nausea and hair loss, as well as complications from total body irradiation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My kidney function is at least 30% of what is expected.

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I am a suitable stem cell donor.

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I need blood transfusions or medications to help with blood cell counts.

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I have low blood counts that are not responding to treatment.

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My platelet count is below 50,000 or I need transfusions.

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My hemoglobin is below 9g/uL or I need blood transfusions.

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I have been diagnosed with DC and show its three skin-related symptoms.

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I have severe aplastic anemia and need a second transplant.

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My heart pumps well and I don't have heart failure.

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My white blood cell count is very low without taking any growth factor medicine.

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My cancer has a mutation in one of the telomerase genes.

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I have severe aplastic anemia and my bone marrow is failing.

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I have been diagnosed with severe aplastic anemia and my condition is resistant to treatment.

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My blood disorder shows early signs of abnormal cell changes.

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My lung function is good and I don't need extra oxygen.

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I have Dyskeratosis Congenita with signs of bone marrow failure.

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I have unusual net-like darkening of my skin.

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My platelet count is below 20,000/uL or I need regular transfusions.

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My genetic test shows a mutation in the TINF2 gene.

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I have white patches inside my mouth.

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My cancer has a CTC1 mutation.

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I have abnormal nail growth.

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I am 70 years old or younger.

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My bone marrow is underperforming with very few blood-making cells.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have severe liver damage or hepatitis.

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I have been diagnosed with Fanconi anemia.

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I have dyskeratosis congenita with advanced MDS or AML with more than 30% blasts.

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I do not have any infections that are not responding to treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of neutrophil engraftment

Incidence of platelet engraftment

Secondary study objectives

Incidence of acute graft-versus-host disease

Incidence of chronic graft-versus-host disease

Incidence of regimen related mortality

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fludarabine

2012

Completed Phase 4

~1860

Alemtuzumab

2004

Completed Phase 4

~1880