Advancing Transplantation Outcomes in Children

NCT ID: NCT06055608

Last Updated: 2026-01-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

200 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-05-22

Study Completion Date

2028-06-30

Brief Summary

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This is a pediatric kidney transplant study comparing the safety and efficacy of an immunosuppressive regimen of belatacept and sirolimus to tacrolimus and Mycophenolate Mofetil (MMF). Two hundred participants will be randomized (1:1) to one of two groups within 24 hours following the transplant procedure. The duration of the study from time of transplant to the primary endpoint is 12-24 months.

Detailed Description

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Conditions

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Kidney Transplant

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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(Group 1): Belatacept+Sirolimus group

Participants in this group will receive antithymocyte globulin (ATG) + steroid taper + belatacept + (tacrolimus bridge, day 0-14) with conversion to sirolimus (day 30 +/-14 days)

Group Type EXPERIMENTAL

Sirolimus

Intervention Type DRUG

Participants in Group 1 will transition to sirolimus therapy on day 14 (+/- 5 days) - weight \<40 kg will receive 3mg/m\^ 2, with maintenance dose of 1 mg/m\^2 divided BID - weight \>= 40kg will receive 6mg/m\^ 2, with maintenance dose of 2 mg daily

Belatacept

Intervention Type BIOLOGICAL

Belatacept will be administered as an intravenous infusion over 30 minutes. The belatacept dose for the study is 10 mg/kg on post-operative day (POD) 1, 5, 14, 28, 56, 84 for the first 3 months, followed by 5 mg/kg every 4 weeks (+/-4 days), starting on month 4 until month 24

Tacrolimus (Group1)

Intervention Type DRUG

Participants will receive Prograf® (tacrolimus), or generic, initiated at 0.1 mg/kg BID within 48 hours of transplantation to attain target trough levels. Participants in Group 1 will be transitioned to sirolimus 2-4 weeks post-transplant

Anti-Thymocyte Globulin (ATG)

Intervention Type DRUG

Participants will receive induction therapy with anti-thymocyte globulin (1.5 mg/kg/dose, maximum 125 mg) starting intraoperatively on day 0 and continuing on days 2 and 3 (total dose 4.5 mg/kg). Total dose may be extended to 6 mg/kg over 1-2 days for delayed graft function

(Group 2): Tacrolimus + Mycophenolate Mofetil (MMF) group

Participants in this group will receive anti-thymocyte globulin (ATG) + steroid taper + tacrolimus + MMF

Group Type ACTIVE_COMPARATOR

Mycophenolate Mofetil

Intervention Type DRUG

Mycophenolate Mofetil-MMF will be initiated at 600 mg/m\^2 BID until tacrolimus is at therapeutic levels, then 450 mg/m\^2 BID

Anti-Thymocyte Globulin (ATG)

Intervention Type DRUG

Participants will receive induction therapy with anti-thymocyte globulin (1.5 mg/kg/dose, maximum 125 mg) starting intraoperatively on day 0 and continuing on days 2 and 3 (total dose 4.5 mg/kg). Total dose may be extended to 6 mg/kg over 1-2 days for delayed graft function

Tacrolimus (Group 2)

Intervention Type DRUG

Participants will receive Prograf® (tacrolimus), or generic, initiated at 0.1 mg/kg BID within 48 hours of transplantation to attain target trough levels

Interventions

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Sirolimus

Participants in Group 1 will transition to sirolimus therapy on day 14 (+/- 5 days) - weight \<40 kg will receive 3mg/m\^ 2, with maintenance dose of 1 mg/m\^2 divided BID - weight \>= 40kg will receive 6mg/m\^ 2, with maintenance dose of 2 mg daily

Intervention Type DRUG

Belatacept

Belatacept will be administered as an intravenous infusion over 30 minutes. The belatacept dose for the study is 10 mg/kg on post-operative day (POD) 1, 5, 14, 28, 56, 84 for the first 3 months, followed by 5 mg/kg every 4 weeks (+/-4 days), starting on month 4 until month 24

Intervention Type BIOLOGICAL

Mycophenolate Mofetil

Mycophenolate Mofetil-MMF will be initiated at 600 mg/m\^2 BID until tacrolimus is at therapeutic levels, then 450 mg/m\^2 BID

Intervention Type DRUG

Tacrolimus (Group1)

Participants will receive Prograf® (tacrolimus), or generic, initiated at 0.1 mg/kg BID within 48 hours of transplantation to attain target trough levels. Participants in Group 1 will be transitioned to sirolimus 2-4 weeks post-transplant

Intervention Type DRUG

Anti-Thymocyte Globulin (ATG)

Participants will receive induction therapy with anti-thymocyte globulin (1.5 mg/kg/dose, maximum 125 mg) starting intraoperatively on day 0 and continuing on days 2 and 3 (total dose 4.5 mg/kg). Total dose may be extended to 6 mg/kg over 1-2 days for delayed graft function

Intervention Type DRUG

Tacrolimus (Group 2)

Participants will receive Prograf® (tacrolimus), or generic, initiated at 0.1 mg/kg BID within 48 hours of transplantation to attain target trough levels

Intervention Type DRUG

Other Intervention Names

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AY 22-989 Rapamune Rapamycin Nulojix CellCept MMF FK-506 FR-900506 Prograf FK-506 FR-900506 Prograf

Eligibility Criteria

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Inclusion Criteria

1. Participant and/or parent/guardian must be able to understand and provide informed consent
2. Male or female, 13-20 years of age at time of enrollment
3. Candidate for primary renal allograft from a living or deceased donor
4. EBV IgG seropositive, defined as evidence of acquired immunity shown by the presence of IgG antibodies to viral capsid antigen (VCA) and EBV nuclear antigen (EBNA)
5. EBV VCA IgM seronegative OR EBV VCA IgM seropositive on two occasions at least 3 months apart and an undetectable EBV PCR result within 1 month prior to enrollment
6. If a female participant of childbearing potential, a negative pregnancy test prior to conducting any study procedures
7. If participant has reproductive potential, agrees to use Food and Drug Administration (FDA) approved methods of birth control for the duration of the study
8. Negative test result for latent tuberculosis infection by tuberculosis skin test (purified protein derivative \[PPD\]) or Tuberculosis (TB) blood test (interferon gamma release assay \[IGRA\] i.e., QuantiFERON, T- SPOT.TB) within 12 months
9. In the absence of contraindication, vaccinations must be up to date per the Centers for Disease Control and Prevention (CDC) Guidelines and Division of Allergy, Immunology, and Transplantation (DAIT) Guidance for Patients in Transplant Trials



Individuals who meet all of the following criteria are eligible for randomization.

1\. If EBV serology to meet enrollment criteria was performed within 8 weeks of receiving IVIG, EBV VCA IgG and EBV EBNA IgG seropositivity, confirmed between enrollment and time of transplant

Exclusion Criteria

1. Inability or unwillingness to comply with study protocol
2. Active infection requiring treatment, or viremia
3. History of malignancy
4. Receipt of any licensed or investigational live attenuated vaccine(s) within 4 weeks of enrollment
5. Prior history of organ transplantation
6. Listed for multi-organ transplant (e.g. heart- kidney, liver-kidney, multivisceral- kidney, lung- kidney)
7. Active systemic autoimmune disease at time of enrollment
8. Idiopathic Focal Segmental Glomerulosclerosis (FSGS), Membranoproliferative Glomerulonephritis (MPGN), C3 glomerulopathy, or atypical Hemolytic Uremic Syndrome (HUS) suspected at risk for recurrence
9. Use of immunosuppressants, biologics (including IVIG), chronic corticosteroids or investigational drug(s) within 8 weeks of enrollment
10. Known bleeding disorder
11. Sustained platelet count \< 75,000 cells/microliters within 3 months of enrollment
12. History of inherited hypercoagulability requiring therapy more than aspirin
13. Panel Reactive Antibody (cPRA) greater than 80 percent
14. Clinically significant unrepaired congenital heart disease causing hemodynamic compromise
15. Uncontrolled diagnosed psychiatric disorder or self-reported drug or alcohol abuse that, in the opinion of the investigator, would interfere with the participant's ability to comply with study requirements
16. Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study


Individuals who meet any of these criteria are not eligible for randomization.

1. Sustained WBC \<1500 or \>20,000 per microliter within 3 months of randomization
2. Sustained liver function tests (AST and/or ALT) \> 2x normal within 3 months of randomization
3. Active systemic autoimmune disease at time of transplant
4. Known bleeding disorder
5. Sustained platelet count \< 75,000 cells/microliters within 3 months of enrollment
6. Current (within 45 days) or historical anti-HLA antibody to the donor prior to randomization
7. Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of randomization
8. Panel Reactive Antibody (cPRA) greater than 80 percent at any point in time
9. If a female participant of childbearing potential, a positive pregnancy test within 48 hours of randomization (all female participants of childbearing potential must complete a pregnancy test within 48 hours of randomization)
10. Treatment with immunosuppressants within 8 weeks of randomization, except in the case of planned transplant standard of care
11. Treatment with biologics (including IVIG) within 8 weeks of randomization
Minimum Eligible Age

13 Years

Maximum Eligible Age

20 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Institute of Allergy and Infectious Diseases (NIAID)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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David Briscoe, MD

Role: STUDY_CHAIR

Boston Children's Hospital: Pediatric Transplantation

Eileen Chambers, MD

Role: STUDY_CHAIR

Duke University Medical Center: Department of Pediatrics

Locations

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University of Alabama at Birmingham (Site # 71038)

Birmingham, Alabama, United States

Site Status RECRUITING

Children's Hospital of Los Angeles (Site #: 71036)

Los Angeles, California, United States

Site Status NOT_YET_RECRUITING

Cedars-Sinai Medical Center (Site #: 71026)

Los Angeles, California, United States

Site Status RECRUITING

Mattel Children's Hospital, UCLA (Site #: 71012)

Los Angeles, California, United States

Site Status RECRUITING

UCSD Rady Children's Hospital (Site #: 71037)

San Diego, California, United States

Site Status RECRUITING

Children's Hospital of Colorado (Site #: 71019)

Aurora, Colorado, United States

Site Status RECRUITING

Nemours Children's Health (Site #: 71042)

Wilmington, Delaware, United States

Site Status RECRUITING

Children's National Medical Center (Site #: 71039)

Washington D.C., District of Columbia, United States

Site Status RECRUITING

Ann and Robert H. Lurie Children's Hospital of Chicago (Site #: 71016)

Chicago, Illinois, United States

Site Status RECRUITING

Johns Hopkins Children's Center (Site #: 71025)

Baltimore, Maryland, United States

Site Status RECRUITING

Boston Children's Hospital (Site #: 71001)

Boston, Massachusetts, United States

Site Status RECRUITING

Helen DeVos Children's Hospital (Site #: 71035)

Grand Rapids, Michigan, United States

Site Status RECRUITING

Washington University/St. Louis Children's Hospital (Site #: 71006)

St Louis, Missouri, United States

Site Status RECRUITING

New York Medical College/Boston Children's Health Physicians

Westchester, New York, United States

Site Status NOT_YET_RECRUITING

Duke University (Site #: 71033)

Durham, North Carolina, United States

Site Status RECRUITING

Cincinnati Children's Hospital Medical Center (Site #: 71017)

Cincinnati, Ohio, United States

Site Status RECRUITING

Children's Hospital of Philadelphia (Site #: 71091)

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

UPMC Children's Hospital of Pittsburgh (Site #: 71008)

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

Texas Children's Hospital (Baylor) (Site #: 71005)

Houston, Texas, United States

Site Status RECRUITING

Seattle Children's Hospital (Site #: 71041)

Seattle, Washington, United States

Site Status RECRUITING

Countries

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United States

Facility Contacts

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Scott House

Role: primary

205-638-9781

Ariana Teame

Role: primary

323-419-2774

Kelly Kirshner

Role: primary

310-825-0922

Serena Ridge

Role: primary

310-968-4225

Kristin Zeeb

Role: primary

858-966-1700 ext. 221657

Claire Giachino

Role: primary

720-777-0106

Aiyana Bullock

Role: primary

302-274-9333

Mirian Naybor

Role: primary

202-476-6659

Petula Grant

Role: primary

312-227-7401

Ryan Hutson

Role: primary

443-287-3214

Emily Toal, MPH

Role: primary

617-919-6535

Lena Sanfilippo

Role: primary

616-391-4305

Lujain Jaza

Role: primary

314-747-1846

Armando Ramirez

Role: primary

914-504-0152

Zachary Messer

Role: backup

Lynette Moats, MSN,RN,CCRC

Role: primary

919-681-3399

Nina Kanis

Role: primary

513-803-1053

Annie Chung

Role: primary

267-425-3934

Rehman Bajwa

Role: primary

412-692-6739

Valerie Okeke

Role: primary

832-824-3141

Megan Kelton

Role: primary

206-987-5539

Related Links

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https://www.niaid.nih.gov/

National Institute of Allergy and Infectious Diseases (NIAID)

http://www.niaid.nih.gov/about/dait

Division of Allergy, Immunology, and Transplantation (DAIT)

Other Identifiers

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DAIT CTOT-41

Identifier Type: -

Identifier Source: org_study_id

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