A Study of a Renal Autologous Cell Therapy (REACT) in Patients With Chronic Kidney Disease (CKD) From Congenital Anomalies of the Kidney and Urinary Tract (CAKUT).

A Phase 1 Open-Label Safety, Tolerability, and Early Efficacy Study of a Renal Autologous Cell Therapy (REACT) in Patients With Chronic Kidney Disease From Congenital Anomalies of the Kidney and Urinary Tract (CAKUT) (REGEN-004)

A phase 1, open-label safety, tolerability and early efficacy study of a Renal Autologous Cell Therapy (REACT) in patients with Chronic Kidney Disease from Congenital Anomalies of the Kidney and Urinary Tract (CAKUT) (REGEN-004)

Study Overview

• Status: Completed
• Conditions: Chronic Kidney Disease; Congenital Anomalies of Kidney and Urinary Tract
• Intervention / Treatment: Biological: Renal Autologous Cell Therapy (REACT)

Detailed Description

It is anticipated that all subjects will receive two planned Renal Autologous Cell Therapy (REACT) injections to allow dose-finding and evaluate the duration of effects. The scientific rationale, based on non-clinical studies, is that the biologically active component of REACT (homologous, autologous, SRC) delays progression of experimental models of Chronic Kidney Disease (CKD) by augmenting renal structure and function. As a result, the more cells that can be infused, the greater the potential improvement in renal function.

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Amy Holman; Phone Number: 859-609-2817; Email: aholman@ctifacts.com
• Study Contact Backup: Name: Kyle West; Phone Number: 919-619-5242; Email: kyle.west@prokidney.com

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University Hospital
  • Idaho
    • Meridian, Idaho, United States, 83642
      • Boise Kidney & Hypertension Institute
  • New Jersey
    • Neptune, New Jersey, United States, 07753
      • Hackensack Meridian Jersey Shore University Medical Center
  • New York
    • New York, New York, United States, 45246
      • Mt. Sinai Hospital
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • University of North Carolina- Chapel Hill

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. The patient is male or female, 18 to 65 years of age on the date of informed consent.
2. The patient has a documented history of a corrected abnormality of the kidney and/or urinary tract (CAKUT) leading to renal dysfunction.
3. The patient has an established diagnosis of Stage III/IV CKD not requiring renal dialysis.
4. The subject has blood pressure less than 150/90 at the Screening Visit, prior to renal biopsy, and prior to REACT injection(s).

Exclusion Criteria:

1. The patient has a history of renal transplantation.
2. The patient has a diagnosis of hydronephrosis, SFU Grade 4 or 5.
3. The patient has an uncorrected VUR Grade 5.
4. The patient has a diffuse cortical thickness throughout the kidney measuring < 5 mm on MRI that prevents safe cortical biopsy.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Renal Autologous Cell Therapy (REACT); The volume of REACT to be administered will be determined by pre-procedure MRI volumetric 3D evaluation or ellipsoid formula. The participant will receive a second injection 6 months after the first injection into the same kidney.	Biological: Renal Autologous Cell Therapy (REACT); Autologous selected renal cells (SRC)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assess change in eGFR and observe incidence of renal-specific procedure and/or product related adverse events (AEs) through 18 months following two Renal Autologous Cell Therapy (REACT) injections [Safety].	The primary objective is to assess the safety and optimal delivery of Renal Autologous Cell Therapy (REACT) injected at one site in a recipient kidney as measured by procedure- and/or product related adverse events (AEs) through 18 months post-treatment.	18 months following last REACT injection

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of subjects with renal-specific adverse events over a 18-month period following injection of Renal Autologous Cell Therapy (REACT).	The number of subjects with renal-specific adverse events over a 18-month period following injection of Renal Autologous Cell Therapy (REACT) will be observed utilizing renal-specific laboratory assessments.	18 months following last REACT injection

Collaborators and Investigators

• Sponsor: Prokidney
• Collaborators: CTI Clinical Trial and Consulting Services
• Investigators: Study Director: Study Director, Prokidney

Publications and helpful links

General Publications

• Stavas J, Diaz-Gonzalez de Ferris M, Johns A, Jain D, Bertram T. Protocol and Baseline Data on Renal Autologous Cell Therapy Injection in Adults with Chronic Kidney Disease Secondary to Congenital Anomalies of the Kidney and Urinary Tract. Blood Purif. 2021;50(4-5):678-683. doi: 10.1159/000512586. Epub 2021 Mar 1.

Study record dates

Study Major Dates

• Study Start (Actual): August 13, 2019
• Primary Completion (Actual): January 31, 2023
• Study Completion (Actual): January 31, 2023

Study Registration Dates

• First Submitted: October 2, 2019
• First Submitted That Met QC Criteria: October 2, 2019
• First Posted (Actual): October 4, 2019

Study Record Updates

• Last Update Posted (Estimated): December 21, 2023
• Last Update Submitted That Met QC Criteria: December 19, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Urologic Diseases; Disease Attributes; Renal Insufficiency; Chronic Disease; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Renal Insufficiency, Chronic; Congenital Abnormalities
• Other Study ID Numbers: REGEN-004

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No