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Stem Cell Transplant for Aplastic Anemia
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.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
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- 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).My white blood cell count is very low without taking any growth factor medicine.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.
- Group 1: Treatment Plan for Dyskeratosis Congenita
- Group 2: Treatment for Severe Aplastic Anemia
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- 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.
Frequently Asked Questions
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."
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.."
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."
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."
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"
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."
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."
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