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Monoclonal Antibodies

Bone Marrow Transplant for Dyskeratosis Congenita

Phase 2
Waitlist Available
Led By Suneet Agarwal, MD, PHD
Research Sponsored by Boston Children's Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Bone marrow hypocellular for age
Moderate or severe aplastic anemia defined by specific criteria
Screening 3 weeks
Treatment Varies
Follow Up up to 15 years post-bmt
Awards & highlights
All Individual Drugs Already Approved
Approved for 5 Other Conditions
No Placebo-Only Group

Study Summary

This trial will test whether a bone marrow transplantation (BMT) can be successful without using DNA damaging agents, which can make the lung and liver disease and risk of cancer worse, in patients with dyskeratosis congenita (DC).

Who is the study for?
This trial is for patients with Dyskeratosis Congenita, specifically those who have moderate or severe aplastic anemia but not Fanconi anemia. Participants need a matching bone marrow donor and good kidney function. It's not for those with prior transplants, significant allergies to the drugs used, HIV, uncontrolled infections, pregnant or breastfeeding women, certain bone marrow abnormalities, or very poor health status.Check my eligibility
What is being tested?
The study tests a new Bone Marrow Transplantation (BMT) regimen without radiation and alkylators in Dyskeratosis Congenita patients. The aim is to see if this less damaging approach can still successfully treat the blood system issues without worsening lung or liver disease or increasing cancer risk.See study design
What are the potential side effects?
Potential side effects may include immune system reactions due to alemtuzumab; digestive problems from mycophenolate mofetil; nervous system effects from fludarabine; and kidney dysfunction from cyclosporins. Tacrolimus might cause high blood pressure and tremors.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
My bone marrow is less active than usual for my age.
I have been diagnosed with moderate or severe aplastic anemia.
I have been diagnosed with dyskeratosis congenita.
I have a donor match for a transplant.
My donor will provide a bone marrow transplant.
I have been tested and do not have Fanconi anemia.
My kidney function is good, with a filtration rate of at least 30 ml/min.


Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 15 years post-bmt
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 15 years post-bmt for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Primary engraftment
Secondary outcome measures
Acute and chronic graft-versus-host disease (GVHD)
Changes in pulmonary function as assessed by pulmonary function testing
Engraftment monitoring (chimerism)
+7 more

Side effects data

From 2021 Phase 3 trial • 30 Patients • NCT01877837
Infection (grade 3 and above)
Graft versus host disease
Renal insufficiency
SupraVentricular Tachycardia
Alerted mental status
Posterior Reversible Encephalopathy Syndrome
Gastrointestinal bleed
Respiratory failure
Study treatment Arm
Patients With Sickle Cell Anemia

Awards & Highlights

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 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: alemtuzumab/fludarabine conditioningExperimental Treatment5 Interventions
alemtuzumab/fludarabine conditioning; calcineurin-inhibitor/mycophenolate mofetil GVHD prophylaxis
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved
Mycophenolate mofetil
Completed Phase 4
Completed Phase 4
Completed Phase 3
Completed Phase 4

Find a Location

Who is running the clinical trial?

Karolinska University HospitalOTHER
466 Previous Clinical Trials
1,303,281 Total Patients Enrolled
Children's Mercy Hospital Kansas CityOTHER
243 Previous Clinical Trials
1,271,788 Total Patients Enrolled
Hackensack Meridian HealthOTHER
131 Previous Clinical Trials
28,204 Total Patients Enrolled

Media Library

Alemtuzumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT01659606 — Phase 2
Dyskeratosis Congenita Clinical Trial 2023: Alemtuzumab Highlights & Side Effects. Trial Name: NCT01659606 — Phase 2
Alemtuzumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01659606 — Phase 2
Dyskeratosis Congenita Research Study Groups: alemtuzumab/fludarabine conditioning

Frequently Asked Questions

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

Does this research initiative permit persons under 80 to take part?

"The eligibility requirements for this research project necessitate that participants are aged between 30 days and 65 years old."

Answered by AI

How many participants have been accepted into this experiment?

"Affirmative. According to clinicaltrials.gov, this clinical trial is accepting applications from eligible candidates since its posting on July 1st 2012 and last updated April 23rd 2022. 40 participants are required between 14 different sites."

Answered by AI

Are there multiple facilities administering this clinical experiment in Canada?

"This clinical trial is currently enrolling patients from a selection of sites, including Duke University Medical Center and Pediatric BMT in Durham (North carolina), Hackensack University Medical Center in Hackensack (New jersey), as well as Children's Mercy Hospital Kansas City located in Missouri. Additionally, there are another 14 locations taking part."

Answered by AI

What reports have been made on the outcomes of Fludarabine research?

"At this time, there are 503 Fludarabine studies being conducted. Of those, 47 have progressed to Phase 3 trials and the bulk of them take place in Philadelphia, Pennsylvania; however, research is happening all over with 4995 sites involved overall."

Answered by AI

In which medical situations is Fludarabine typically prescribed?

"Fludarabine, a common treatment for transplantation patients, can also be beneficial in managing excessive tearing, lupus nephritis and bulla."

Answered by AI

Are there current vacancies for this experimental therapy?

"Affirmative. Per the information uploaded on clinicaltrials.gov, this medical research is currently recruiting participants. It was first posted on July 1st 2012 and most recently edited on April 23rd 2022, with 40 patients sought at 14 different sites."

Answered by AI

What potential risks are associated with the utilization of fludarabine?

"Fludarabine has been allocated a score of 2, as clinical evidence exists to suggest it is safe but the data supporting its efficacy remains inconclusive."

Answered by AI

Am I eligible to join this research project?

"This clinical trial seeks 40 individuals with aplastic anemia between one month and sixty-five years of age. To qualify, patients must meet certain criteria: Diagnosis of dyskeratosis congenita based on the threefold abnormality of skin pigmentation, nail dystrophy or oral leukoplakia; OR one from these triad along with two associated features; OR documented presence of pathogenic mutation in DKC1,TERC, TERT, NOP10 etc.; OR telomere length < 1%ile for their age as reported by CLIA approved lab; OR patient having Hoyeraal-Hre"

Answered by AI
~0 spots leftby Apr 2024