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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

Diagnosis of Fanconi anemia must be excluded by specific testing

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 15 years post-bmt

Awards & highlights

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

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My bone marrow is less active than usual for my age.

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I have been tested and do not have Fanconi anemia.

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My kidney function is good, with a filtration rate of at least 30 ml/min.

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I have been diagnosed with moderate or severe aplastic anemia.

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My donor will provide a bone marrow transplant.

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I have a donor match for a transplant.

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I have been diagnosed with dyskeratosis congenita.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 15 years post-bmt

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

36%

Infection (grade 3 and above)

24%

Graft versus host disease

Renal insufficiency

SupraVentricular Tachycardia

Alerted mental status

Posterior Reversible Encephalopathy Syndrome

Gastrointestinal bleed

Respiratory failure

Hypokalemia

100%

80%

60%

40%

20%

Study treatment Arm

Patients With Sickle Cell Anemia

Trial Design

1Treatment groups

Experimental Treatment

Group I: alemtuzumab/fludarabine conditioningExperimental Treatment5 Interventions

alemtuzumab/fludarabine conditioning; calcineurin-inhibitor/mycophenolate mofetil GVHD prophylaxis

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Tacrolimus

FDA approved

Mycophenolate mofetil

2014

Completed Phase 4

~2350

alemtuzumab

2004

Completed Phase 4

~2760

Fludarabine

2012

Completed Phase 3

~1100

Cyclosporins

2018

Completed Phase 4

~220

0 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

Fred Hutch/University of Washington/Seattle Children's Cancer ConsortiumUNKNOWN

Children's Hospital of PhiladelphiaOTHER

707 Previous Clinical Trials

8,581,251 Total Patients Enrolled

1 Trials studying Dyskeratosis Congenita

1,716 Patients Enrolled for Dyskeratosis Congenita

Karolinska University HospitalOTHER

476 Previous Clinical Trials

1,304,897 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

Recent research and studies

Pediatric Blood & CancerJournal

Reduced‐intensity Conditioning–based Hematopoietic Cell Transplantation for Dyskeratosis Congenita: Single‐center Experience and Literature Review

Bhoopalan, Senthil Velan, Marcin Wlodarski, Ulrike Reiss, Brandon Triplett, and Akshay Sharma. 2021. “Reduced‐intensity Conditioning–based Hematopoietic Cell Transplantation for Dyskeratosis Congenita: Single‐center Experience and Literature Review”. Pediatric Blood & Cancer. Wiley. doi:10.1002/pbc.29177.

Pediatric Blood & CancerJournal

Reduced‐intensity Conditioning–based Hematopoietic Cell Transplantation for Dyskeratosis Congenita: Single‐center Experience and Literature Review

clinicaltrials.govStudy

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

Suneet Agarwal 2012. "Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01659606.

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