RECRUITING

Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease

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Conditions

Sickle Cell Disease

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).

Official Title

Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease

Quick Facts

Study Start:2018-04-17
Study Completion:2025-11
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT03587272

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 25 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm/sec by the non-imaging technique (or ≥185 cm/sec by the imaging technique) measured at a minimum of two separate occasions.
  2. * History of cerebral infarction on brain MRI (overt stroke, or silent stroke if ≥3 mm in one dimension, visible in two planes on fluid-attenuated inversion recovery T2-weighted images).
  3. * History of two or more episodes of acute chest syndrome (ACS) in lifetime.
  4. * History of three or more SCD pain events requiring treatment with an opiate or IV pain medication (inpatient or outpatient) in lifetime.
  5. * History of any hospitalization for SCD pain or ACS while receiving hydroxyurea treatment.
  6. * History of two or more episodes of priapism (erection lasting ≥4 hours or requiring emergent medical care).
  7. * Administration of regular RBC transfusions (≥8 transfusions in the previous 12 months).
  8. * At least two episodes of splenic sequestration requiring red blood cell transfusion or splenectomy after at least one episode of splenic sequestration.
  9. * Clinically significant neurologic event (overt stroke).
  10. * History of two or more episodes of ACS in the 2-years period preceding enrollment.
  11. * History of three or more SCD pain events requiring treatment with an opiate or IV pain medication (inpatient or outpatient) in the 1-year period preceding enrollment.
  12. * History of any hospitalization for SCD pain or ACS while receiving hydroxyurea treatment.
  13. * History of two or more episodes of priapism (erection lasting ≥4 hours or requiring emergent medical care).
  14. * Administration of regular RBC transfusions (≥8 transfusions in the previous 12 months).
  15. * At least two episodes of splenic sequestration requiring red blood cell transfusion or splenectomy after at least one episode of splenic sequestration.
  1. * • General: Life expectancy less than 6 months. Pregnant or breastfeeding patients.
  2. * Infection Disease: Uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms) within 1 month prior to conditioning. Patients with febrile illness or suspected minor infection should await clinical resolution prior to starting conditioning. Patients with confirmed seropositivity for HIV and patients with active Hepatitis B or C determined by serology and/or NAAT are excluded.
  3. * Liver: Direct (conjugated) bilirubin \> 1.5 mg/dL, transaminases \>5x upper limit of normal for age.
  4. * Cardiac: Left ventricular shortening fraction \<25% or ejection fraction \<50% by ECHO.
  5. * Kidney: Estimated creatinine clearance less than 60 mL/min/1.73m2.
  6. * Pulmonary function: Diffusion capacity of carbon monoxide (DLCO) \<35% (adjusted for hemoglobin). Baseline oxygen saturation \<85% or PaO2 \<70.
  7. * Heme: History of RBC alloantibodies against donor RBC antigens (even if current antibody screen is negative). Major ABO incompatibility with donor.

Contacts and Locations

Study Contact

Robert Nickel, MD
CONTACT
202-476-5000
RNickel@childrensnational.org
Fahmida Hoq, MBBS
CONTACT
202-476-5000
FHoq@childrensnational.org

Principal Investigator

Rober Nickel, MD
PRINCIPAL_INVESTIGATOR
Children's National Health System

Study Locations (Sites)

Children's National Health System
Washington, District of Columbia, 20010
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
Levine Children's Hospital
Charlotte, North Carolina, 28203
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States

Collaborators and Investigators

Sponsor: Robert Nickel

  • Rober Nickel, MD, PRINCIPAL_INVESTIGATOR, Children's National Health System

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2018-04-17
Study Completion Date2025-11

Study Record Updates

Study Start Date2018-04-17
Study Completion Date2025-11

Terms related to this study

Additional Relevant MeSH Terms

  • Sickle Cell Disease