IBMFS for Bone Marrow Failure

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Bone Marrow Failure+2 MoreCyclophosphamide - Drug
Eligibility
4 - 40
All Sexes
What conditions do you have?
Select

Study Summary

This trial will study how well a reduced intensity conditioning hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) work in patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), and inherited bone marrow failure syndromes (IBMFS).

Eligible Conditions
  • Bone Marrow Failure
  • Primary Immunodeficiency
  • Immune Deficiency

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 20 Secondary · Reporting Duration: 2 years

1 year
Disease Free Survival at 1 year
Event-Free Survival at 1 year
Number of patients that have survived at 1 year
The impact of BMT on disease-specific testing
Transplant-related complications
120 days
Feasibility of early cessation of tacrolimus in a subset of patients (with matched related or matched unrelated donors)
14-30 days
Median day of neutrophil engraftment
14-45 days
Median day of platelet engraftment
2 years
Cumulative incidence of Chronic GVHD
Disease Free Survival at 2 years
Donor T-cell alloreactivity
Event-Free Survival at 2 years
Incidence of Infectious complications
Kinetics of Immune reconstitution
Number of patients that have survived at 2 years
Number of patients with Graft failure
Number of patients with Sustained Donor Engraftment
Recipient T-cell alloreactivity
Transplant-related mortality
30-180 days
Graft-vs-Host Disease
60 Days
Donor Engraftment

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

2 Treatment Groups

IBMFS
1 of 2
PID/IDS
1 of 2

Experimental Treatment

27 Total Participants · 2 Treatment Groups

Primary Treatment: IBMFS · No Placebo Group · Phase 2

IBMFSExperimental Group · 6 Interventions: Cyclophosphamide, Fludarabine, Alemtuzumab, Melphalan, Tacrolimus, Mycophenolate Mofetil · Intervention Types: Drug, Drug, Drug, Drug, Drug, Drug
PID/IDSExperimental Group · 7 Interventions: Cyclophosphamide, Fludarabine, Alemtuzumab, Low Dose Total Body Irradiation, Melphalan, Tacrolimus, Mycophenolate Mofetil · Intervention Types: Drug, Drug, Drug, Radiation, Drug, Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cyclophosphamide
FDA approved
Fludarabine
FDA approved
Alemtuzumab
FDA approved
Melphalan
FDA approved
Tacrolimus
FDA approved
Mycophenolate mofetil
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 2 years

Who is running the clinical trial?

Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsLead Sponsor
536 Previous Clinical Trials
31,238 Total Patients Enrolled
1 Trials studying Bone Marrow Failure
21 Patients Enrolled for Bone Marrow Failure
Heather J Symons, MD, MHSPrincipal InvestigatorJohns Hopkins University

Eligibility Criteria

Age 4 - 40 · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a bone marrow failure disorder.