This trial is testing different ways to prepare for and do a stem cell transplant for people with dyskeratosis congenita or severe aplastic anemia.
- Dyskeratosis Congenita
- Aplastic Anemia
2 Primary · 4 Secondary · Reporting Duration: 1 Year
Awards & Highlights
2 Treatment Groups
Treatment for Severe Aplastic Anemia
1 of 2
Treatment Plan for Dyskeratosis Congenita
1 of 2
50 Total Participants · 2 Treatment Groups
Primary Treatment: Fludarabine · No Placebo Group · N/A
Who is running the clinical trial?
Age No minimum age - 70 · All Participants · 2 Total Inclusion CriteriaMark “Yes” if the following statements are true for you:
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." - Anonymous Online Contributor
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.." - Anonymous Online Contributor
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." - Anonymous Online Contributor
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." - Anonymous Online Contributor
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" - Anonymous Online Contributor
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." - Anonymous Online Contributor
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." - Anonymous Online Contributor