Bone Marrow Transplant for Dyskeratosis Congenita

Recruiting in Palo Alto (17 mi)

+17 other locations

Overseen by Suneet Agarwal, MD, PHD

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Boston Children's Hospital

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial tests a new bone marrow transplant method for patients with Dyskeratosis congenita. It uses fludarabine and antibodies to avoid harmful side effects, aiming to improve survival and reduce complications. Fludarabine-based regimens have been shown to be effective and feasible in reducing transplant-related morbidity in patients with Dyskeratosis congenita.

Research Team

Suneet Agarwal, MD, PHD

Principal Investigator

Boston Children's Hospital

Eligibility Criteria

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.

Inclusion Criteria

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

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.

Exclusion Criteria

You have a specific type of antibody that reacts against the donor's tissue, and it could cause health problems.

I have had a bone marrow or stem cell transplant from another person.

My bone marrow test shows changes linked to MDS or AML.

Treatment Details

Interventions

• Alemtuzumab (Monoclonal Antibodies)
• Cyclosporins (Immunosuppressant)
• Fludarabine (Antimetabolites)
• Mycophenolate Mofetil (Antimetabolites)
• Tacrolimus (Immunosuppressant)

Trial Overview 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.

Participant Groups

Group I: alemtuzumab/fludarabine conditioning Experimental Treatment 5 Interventions

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

Who Is Running the Clinical Trial?

Boston Children's Hospital

Lead Sponsor

Trials

801

Recruited

5,584,000+

Dr. Kevin B. Churchwell

Boston Children's Hospital

Chief Executive Officer since 2021

MD from Vanderbilt Medical School

Dr. Sarah Pitts

Boston Children's Hospital

Chief Medical Officer since 2019

MD from Harvard Medical School

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

Collaborator

Trials

1

Recruited

40+

Children's Hospital of Philadelphia

Collaborator

Trials

749

Recruited

11,400,000+

Joseph W. St. Geme III

Children's Hospital of Philadelphia

Chief Medical Officer since 2021

MD, PhD, MPH

Madeline Bell

Children's Hospital of Philadelphia

Chief Executive Officer since 2015

BSc in Nursing from Villanova University, MSc in Organizational Dynamics from the University of Pennsylvania

Karolinska University Hospital

Collaborator

Trials

509

Recruited

1,319,000+

Björn Zoëga

Karolinska University Hospital

Chief Executive Officer since 2019

PhD in Medicine from the University of Gothenburg, MBBS from Reykjavik University

Christophe Pedroletti

Karolinska University Hospital

Chief Medical Officer

MD, PhD

Mayo Clinic

Collaborator

Trials

3,427

Recruited

3,221,000+

Dr. Gianrico Farrugia

Mayo Clinic

Chief Executive Officer since 2019

MD from University of Malta Medical School

Dr. Richard Afable

Mayo Clinic

Chief Medical Officer

MD from Loyola Stritch School of Medicine

University of Chicago

Collaborator

Trials

1,086

Recruited

844,000+

Pete Salzmann

University of Chicago

Chief Executive Officer since 2018

MD from University of Chicago’s Pritzker School of Medicine, MBA from Stanford University’s Graduate School of Business

Anh Nguyen

University of Chicago

Chief Medical Officer

MD from Rutgers New Jersey Medical School, MBA from University of Chicago

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Collaborator

Trials

10

Recruited

2,100+

Children's Hospital Los Angeles

Collaborator

Trials

257

Recruited

5,075,000+

Paul S. Viviano

Children's Hospital Los Angeles

Chief Executive Officer since 2015

Master of Public Health from UCLA Fielding School of Public Health

Alan S. Wayne

Children's Hospital Los Angeles

Chief Medical Officer since 2023

MD

Children's Mercy Hospital Kansas City

Collaborator

Trials

261

Recruited

941,000+

Dr. Alejandro Quiroga

Children's Mercy Hospital Kansas City

Chief Executive Officer

MD from Universidad Del Rosario, MBA

Dr. Doug Blowey

Children's Mercy Hospital Kansas City

Chief Medical Officer since 2023

MD from University of Kansas

University of Wisconsin, Madison

Collaborator

Trials

1,249

Recruited

3,255,000+

Robert Drape

University of Wisconsin, Madison

Chief Executive Officer since 2007

Executive MBA from the University of Wisconsin – Madison, Bachelor's degree in Biology from Augustana College (IL)

Dr. Ciara Barclay-Buchanan

University of Wisconsin, Madison

Chief Medical Officer since 2023

MD from Wayne State University School of Medicine