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Monoclonal Antibodies

Stem Cell Transplant for Aplastic Anemia

N/A
Recruiting
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights

Study 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.Check my eligibility
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.See study design
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 white blood cell count is very low without taking any growth factor medicine.
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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 organs are working well.
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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 been diagnosed with DC.
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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.

Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of neutrophil engraftment
Incidence of platelet engraftment
Secondary outcome measures
Incidence of acute graft-versus-host disease
Incidence of chronic graft-versus-host disease
Incidence of regimen related mortality
+1 more

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 3
~1100
Stem Cell Transplant
2007
Completed Phase 3
~1350
Anti-thymocyte globulin
2010
Completed Phase 4
~470
Cyclophosphamide
1995
Completed Phase 3
~3780
Alemtuzumab
2004
Completed Phase 4
~1890
Total Body Irradiation
2006
Completed Phase 3
~820

Find a Location

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor
270 Previous Clinical Trials
14,542 Total Patients Enrolled
1 Trials studying Dyskeratosis Congenita
36 Patients Enrolled for Dyskeratosis Congenita
Jakub Tolar, MDPrincipal InvestigatorUniversity of Minnesota
1 Previous Clinical Trials
12 Total Patients Enrolled

Media Library

Alemtuzumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT02162420 — N/A
Dyskeratosis Congenita Research Study Groups: Treatment Plan for Dyskeratosis Congenita, Treatment for Severe Aplastic Anemia
Dyskeratosis Congenita Clinical Trial 2023: Alemtuzumab Highlights & Side Effects. Trial Name: NCT02162420 — N/A
Alemtuzumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02162420 — N/A

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the eligibility for this clinical trial limited to participants under 60 years of age?

"As per the specified enrollment guidelines, all participants must be 0-70 years old in order to partake."

Answered by AI

What outcomes is this research endeavor aiming to accomplish?

"As the primary outcome, this clinical trial will monitor the rate of platelet engraftment after Day 42. Additionally, researchers are keeping an eye out for acute graft-versus-host disease by day 100., secondary malignancies and regimen related mortality also at day 100.."

Answered by AI

What conditions has Fludarabine demonstrated efficacy in ameliorating?

"Fludarabine is frequently administered to suppress the immune system, but also can be employed in cases of multiple sclerosis, leukemia, myelocytic acute rejection, and kidney transplantation."

Answered by AI

Are there any prior investigations that have utilized Fludarabine?

"At present, the number of active studies for Fludarabine is 919 with 165 in Phase 3. Philadelphia, Pennsylvania has the highest concentration of research; however, there are 28 615 locations conducting trials for this drug globally."

Answered by AI

To whom may this research project be administered?

"This clinical trial will include fifty individuals in the 0-70 age range who have been diagnosed with aplastic anemia. To be eligible to participate, they must also meet additional criteria such as displaying mutations found in telomerase holoenzyme (DKC1, TERT, TERC, NOP10, NHP2 and TCAB1) genes; presenting transfusion requirements for red blood cells or platelets; exhibiting oral leukoplakia symptoms; having nail dystrophy issues; showcasing shelterin complex mutation characteristics (TINF2); and/or developing refractory cytopenias featuring platelet counts"

Answered by AI

Is enrollment still possible for participants in this scientific experiment?

"As detailed on clinicaltrials.gov, this particular medical trial is still open for recruitment having been posted on January 1st 2015 and recently revised on the 20th of January 2022."

Answered by AI

How many participants have been recruited to partake in this research?

"Affirmative. Per the info available on clinicaltrials.gov, this research endeavour is currently seeking enrolment from participants. It was announced to the public on January 1st 2015 and has had its most recent amendment made on January 20th 2022. The investigation requires 50 people across a single location for completion."

Answered by AI
~6 spots leftby Jul 2025