Cohort 2a: Conditioning Regimen B for Cystinosis With CKD Stage 3 Through 5

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Lucile Packard Children's Hospital, Palo Alto, CA
Cystinosis With CKD Stage 3 Through 5+6 More
CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System - Device
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

This is a single center, non-randomized, non-controlled open-label phase 1b/2a trial of performing sequential αβdepleted-HSCT and KT in patients requiring KT to prevent kidney rejection post-KT, in the absence of any post-KT immunosuppression, to abrogate the need for lifelong immunosuppression, the risk of chronic rejection and, ultimately, the need for repeated transplantation.

Eligible Conditions

  • Cystinosis With CKD Stage 3 Through 5
  • FSGS With CKD Stage 3 Through 5
  • SLE Nephritis With CKD Stage 3 Through 5
  • SIOD With CKD Stage 3 Through 5

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 9 Secondary · Reporting Duration: +1 year post-KT

+1 year post-HSCT
Number of patients with chronic GvHD
+1 year post-KT
Number of patients with de novo acute GVHD
Number of patients with de novo chronic GVHD
Number of patients with successful kidney function
+5 year post-KT
Number of cases of secondary malignancies
6- and 12-months post-KT
Number of patients with functional tolerance to donor cells
Year 1
Number of patients with persistent full donor chimerism
Day +42 post-HSCT
Number of patients with myloid engraftment
Day +90 and Day +180 post-HSCT
Number of patients with acute GvHD
Day +90 post-KT
Number of patients who are able to discontinue immunosuppression post-KT

Trial Safety

Safety Progress

1 of 3

Trial Design

4 Treatment Groups

Cohort 2a: Conditioning Regimen B
1 of 4
Cohort 1b: Conditioning Regimen A
1 of 4
Cohort 1b: Conditioning Regimen B
1 of 4
Cohort 2a: Conditioning Regimen A
1 of 4
Experimental Treatment

12 Total Participants · 4 Treatment Groups

Primary Treatment: Cohort 2a: Conditioning Regimen B · No Placebo Group · Phase 1 & 2

Cohort 2a: Conditioning Regimen BExperimental Group · 8 Interventions: CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System, ATG, Fludarabine, Melphalan, Rituximab, Cyclophosphamide 100 mg/Kg, Total Body Irradiation, Kidney Transplant · Intervention Types: Device, Drug, Drug, Drug, Drug, Drug, Radiation, Procedure
Cohort 1b: Conditioning Regimen AExperimental Group · 7 Interventions: CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System, ATG, Cyclophosphamide 1200 mg/Kg, Fludarabine, Rituximab, Total Body Irradiation, Kidney Transplant · Intervention Types: Device, Drug, Drug, Drug, Drug, Radiation, Procedure
Cohort 1b: Conditioning Regimen BExperimental Group · 8 Interventions: CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System, ATG, Fludarabine, Melphalan, Rituximab, Cyclophosphamide 100 mg/Kg, Total Body Irradiation, Kidney Transplant · Intervention Types: Device, Drug, Drug, Drug, Drug, Drug, Radiation, Procedure
Cohort 2a: Conditioning Regimen AExperimental Group · 7 Interventions: CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System, ATG, Cyclophosphamide 1200 mg/Kg, Fludarabine, Rituximab, Total Body Irradiation, Kidney Transplant · Intervention Types: Device, Drug, Drug, Drug, Drug, Radiation, Procedure
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
ATG
2016
Completed Phase 4
~870
Fludarabine
2012
Completed Phase 2
~1240
Melphalan
2008
Completed Phase 3
~1940
Rituximab
1999
Completed Phase 3
~1510
Total Body Irradiation
2006
Completed Phase 3
~810

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: +1 year post-kt
Closest Location: Lucile Packard Children's Hospital · Palo Alto, CA
2005First Recorded Clinical Trial
1 TrialsResearching Cystinosis With CKD Stage 3 Through 5
35 CompletedClinical Trials

Eligibility Criteria

Age < 65 · All Participants · 7 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are able to give informed consent or have an LAR available to provide consent.
You are male and female subjects of childbearing potential
You have a steroid intake of less than 0.5 mg/kg/day.
You have a score of at least 50 on the Lansky/Karnofsky Scale.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

References