Treatment for Myeloid Chimerism

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
National Institutes of Health Clinical Center, Bethesda, MD
Myeloid Chimerism+1 More
Eligibility
Any Age
All Sexes
Eligible conditions
Select

Study Summary

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

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Eligible Conditions

  • Myeloid Chimerism

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Treatment will improve 1 primary outcome and 1 secondary outcome in patients with Myeloid Chimerism. Measurement will happen over the course of 5 years.

5 years
Incidence of graft rejection defined as HbS >10% when donors have HbAA and HbS >50% when donors have sickle cell trait
dichotomous positive/negative outcome where a positive response is defined by absence of graft rejection

Trial Safety

Safety Estimate

1 of 3

Trial Design

0 Treatment Group

This trial requires 30 total participants across 0 different treatment group

Trial Logistics

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 5 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 5 years for reporting.

Closest Location

National Institutes of Health Clinical Center - Bethesda, MD

Eligibility Criteria

This trial is for patients born any sex of any age. You must have received 1 prior treatment for Myeloid Chimerism or the other condition listed above. There are 9 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Inclusion criteria- recipient
You have a history of SCD or beta thalassemia and you have undergone allogeneic HSCT. show original
Patient with recurrent SCD defined as HbS greater than or equal to 50% for donors with sickle cell trait and greater than or equal to 10% for donors with HbAA with recurrent clinical manifestations (for example but not limited to recurrent painful crises, acute chest syndrome, priapism, or severe anemia) or patients with recurrent beta thalassemia defined as clinical manifestations such as transfusion-dependence or evidence of extramedullary hematopoiesis. The HbS requirement may be waived by the PI or designee for reasons such as the patient is requiring chronic transfusion therapy or otherwise meets clinical criteria for return of SCD.
You have persistent donor chimerism levels. show original
You are 4 years of age or older. show original
Negative beta-HCG
Ejection fraction greater than or equal to 35%
DLCO greater than or equal to 35%
Inclusion- donor Related donor from original transplant, deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood for research. Related donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Age (Bullet) 2 years <80 years old

Patient Q&A Section

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Are there any geographic restrictions to consider when applying for this trial?

Patients from any state can participate in this trial, so long as they are able to attend all treatment sessions.

Will my insurance cover participating in this trial?

In order to understand how insurance coverage works for this trial, you will need to contact the study coordinator.

See if you qualify for this trial
Get access to this novel treatment for Myeloid Chimerism by sharing your contact details with the study coordinator.