Actively Recruiting

Early Phase 1
Age: 0 - 22Years
All Genders
NCT02928991

Fludarabine Based RIC for Bone Marrow Failure Syndromes

Led by Children's Hospital of Philadelphia · Updated on 2025-05-20

25

Participants Needed

1

Research Sites

661 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This is a pilot study to determine whether fludarabine-based reduced intensity conditioning (RIC) regimens facilitate successful donor engraftment of patients with acquired aplastic anemia (AA) and Inherited bone marrow failure (iBMF) syndromes undergoing Matched related donor bone marrow transplant (MRD-BMT).

CONDITIONS

Official Title

Fludarabine Based RIC for Bone Marrow Failure Syndromes

Who Can Participate

Age: 0 - 22Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patient is 0 to 22 years old at enrollment
  • Diagnosis of severe or very severe acquired aplastic anemia with bone marrow cellularity below 25% plus low neutrophils, platelets, and reticulocytes
  • Negative tests for inherited bone marrow failure and myelodysplastic syndromes
  • Patients with paroxysmal nocturnal hemoglobinuria clones meeting severe aplastic anemia criteria
  • Diagnosis of inherited bone marrow failure syndromes causing chronic transfusion dependence or low neutrophils, including Fanconi Anemia, Dyskeratosis Congenita, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Congenital Dyserythropoietic/Sideroblastic Anemias, Congenital Amegakaryocytic Thrombocytopenia, or Shwachman-Diamond Syndrome
  • Performance score above 60 on Lansky or Karnofsky scale
  • Availability of a fully HLA-matched related donor
  • No active untreated infection
  • Negative pregnancy test for females of childbearing potential
  • Kidney function with serum creatinine less than 1.5 times the upper limit of normal for age
  • Liver enzymes less than 5 times normal
  • Cardiac shortening fraction above 27%
  • Bilirubin less than 2.5 times normal (unless due to Gilbert's disease)
  • Donor must be at least 6 months old and meet eligibility including infectious disease screening
  • Donor testing negative for inherited bone marrow failure if recipient has iBMF syndrome
  • Donor and collection procedures follow institutional and FDA regulations
Not Eligible

You will not qualify if you...

  • Uncontrolled bacterial, viral, or fungal infections
  • Unable to find or use a fully HLA-matched related donor
  • Donor unable to donate bone marrow
  • Pregnancy
  • Diagnosis of myelodysplastic syndrome with bone marrow dysplasia and cytogenetic abnormalities
  • Paroxysmal nocturnal hemoglobinuria without underlying bone marrow aplasia

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 1 location

1

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

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Research Team

M

Megan Atkinson

CONTACT

P

Patricia Hankins, BSN, RN, CCRC

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

3

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