Fludarabine for Dyskeratosis Congenita

University of Minnesota Medical Center, Fairview, Minneapolis, MN
Dyskeratosis Congenita+1 More ConditionsFludarabine - Drug
No minimum age - 70
All Sexes

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.

Eligible Conditions
  • Dyskeratosis Congenita
  • Aplastic Anemia

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3

Study Objectives

2 Primary · 4 Secondary · Reporting Duration: 1 Year

1 Year
Incidence of chronic graft-versus-host disease
Incidence of secondary malignancies
1 year
Incidence of platelet engraftment
Day 100
Incidence of acute graft-versus-host disease
Incidence of regimen related mortality
Day 42
Incidence of neutrophil engraftment

Trial Safety

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 20 Other Conditions
This treatment demonstrated efficacy for 20 other conditions.

Trial Design

2 Treatment Groups

Treatment for Severe Aplastic Anemia
1 of 2
Treatment Plan for Dyskeratosis Congenita
1 of 2

Experimental Treatment

50 Total Participants · 2 Treatment Groups

Primary Treatment: Fludarabine · No Placebo Group · N/A

Treatment for Severe Aplastic AnemiaExperimental Group · 5 Interventions: Fludarabine, Stem Cell Transplant, Anti-thymocyte globulin, Cyclophosphamide, Total Body Irradiation · Intervention Types: Drug, Biological, Drug, Drug, Radiation
Treatment Plan for Dyskeratosis CongenitaExperimental Group · 4 Interventions: Fludarabine, Stem Cell Transplant, Cyclophosphamide, Alemtuzumab · Intervention Types: Drug, Biological, Drug, Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved
Stem Cell Transplant
Completed Phase 3
Antithymocyte immunoglobulin (rabbit)
FDA approved
FDA approved
FDA approved
Total Body Irradiation
Completed Phase 3

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor
265 Previous Clinical Trials
15,972 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

Eligibility Criteria

Age No minimum age - 70 · All Participants · 2 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a rare condition called Dyskeratosis Congenita that causes changes in the skin, nails, and mouth.
Your heart, lungs, and kidneys are functioning well enough to participate in the study. You can also provide your consent in writing.

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.