Your session is about to expire
← Back to Search
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
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
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 belowSelect...
My kidney function is at least 30% of what is expected.
Select...
I am a suitable stem cell donor.
Select...
I need blood transfusions or medications to help with blood cell counts.
Select...
I have low blood counts that are not responding to treatment.
Select...
My platelet count is below 50,000 or I need transfusions.
Select...
My hemoglobin is below 9g/uL or I need blood transfusions.
Select...
I have been diagnosed with DC and show its three skin-related symptoms.
Select...
I have severe aplastic anemia and need a second transplant.
Select...
My heart pumps well and I don't have heart failure.
Select...
My white blood cell count is very low without taking any growth factor medicine.
Select...
My cancer has a mutation in one of the telomerase genes.
Select...
I have severe aplastic anemia and my bone marrow is failing.
Select...
I have been diagnosed with severe aplastic anemia and my condition is resistant to treatment.
Select...
My blood disorder shows early signs of abnormal cell changes.
Select...
My lung function is good and I don't need extra oxygen.
Select...
I have Dyskeratosis Congenita with signs of bone marrow failure.
Select...
I have unusual net-like darkening of my skin.
Select...
My platelet count is below 20,000/uL or I need regular transfusions.
Select...
My genetic test shows a mutation in the TINF2 gene.
Select...
I have white patches inside my mouth.
Select...
My cancer has a CTC1 mutation.
Select...
I have abnormal nail growth.
Select...
I am 70 years old or younger.
Select...
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:Select...
I have severe liver damage or hepatitis.
Select...
I have been diagnosed with Fanconi anemia.
Select...
I have dyskeratosis congenita with advanced MDS or AML with more than 30% blasts.
Select...
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
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 moreTrial 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
~1020
Alemtuzumab
2004
Completed Phase 4
~1890
Cyclophosphamide
1995
Completed Phase 3
~3770
Stem Cell Transplant
2007
Completed Phase 3
~1350
Anti-thymocyte globulin
2010
Completed Phase 4
~470
Total Body Irradiation
2006
Completed Phase 3
~820
Find a Location
Who is running the clinical trial?
Masonic Cancer Center, University of MinnesotaLead Sponsor
276 Previous Clinical Trials
14,813 Total Patients Enrolled
1 Trials studying Aplastic Anemia
36 Patients Enrolled for Aplastic Anemia
Jakub Tolar, MDPrincipal InvestigatorUniversity of Minnesota
1 Previous Clinical Trials
12 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My kidney function is at least 30% of what is expected.I have severe liver damage or hepatitis.I have received radiation therapy before (applies only if I have severe aplastic anemia).Your body is rejecting a transplanted organ, as shown by a specific test.My heart pumps well and I don't have heart failure.My white blood cell count is very low without taking any growth factor medicine.My cancer has a mutation in one of the telomerase genes.I have severe aplastic anemia and my bone marrow is failing.I have been diagnosed with severe aplastic anemia and my condition is resistant to treatment.Your absolute reticulocyte count is less than 20,000 per microliter.My blood disorder shows early signs of abnormal cell changes.My organs are working well.My lung function is good and I don't need extra oxygen.I have severe aplastic anemia and need a second transplant.I have been diagnosed with Fanconi anemia.I have dyskeratosis congenita with advanced MDS or AML with more than 30% blasts.I am a suitable stem cell donor.I need blood transfusions or medications to help with blood cell counts.I have low blood counts that are not responding to treatment.My platelet count is below 50,000 or I need transfusions.My hemoglobin is below 9g/uL or I need blood transfusions.I have been diagnosed with DC and show its three skin-related symptoms.I have Dyskeratosis Congenita with signs of bone marrow failure.I have unusual net-like darkening of my skin.I do not have any infections that are not responding to treatment.My platelet count is below 20,000/uL or I need regular transfusions.My genetic test shows a mutation in the TINF2 gene.I have been diagnosed with DC.I have white patches inside my mouth.Your telomeres are shorter than what is being studied.My cancer has a CTC1 mutation.I have abnormal nail growth.I am 70 years old or younger.My bone marrow is underperforming with very few blood-making cells.
Research Study Groups:
This trial has the following groups:- Group 1: Treatment Plan for Dyskeratosis Congenita
- Group 2: Treatment for Severe Aplastic Anemia
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Share this study with friends
Copy Link
Messenger