Clinical trial NCT04115345: 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).
Official title: 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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Chronic Kidney Disease
Congenital Anomalies of Kidney and Urinary Tract
Lead sponsor: Prokidney
Sponsor class: INDUSTRY
Collaborators: name: CTI Clinical Trial and Consulting Services
First posted: 2019-10-04
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open-label
Primary purpose: TREATMENT

Brief summary: 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)

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.

Eligibility criteria: 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.

Primary outcomes:
measure: 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].
timeFrame: 18 months following last REACT injection
Secondary outcomes:
measure: Number of subjects with renal-specific adverse events over a 18-month period following injection of Renal Autologous Cell Therapy (REACT).
timeFrame: 18 months following last REACT injection
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University Hospital
state: Georgia
city: Meridian
country: United States
facility: Boise Kidney & Hypertension Institute
state: Idaho
city: Neptune
country: United States
facility: Hackensack Meridian Jersey Shore University Medical Center
state: New Jersey
city: New York
country: United States
facility: Mt. Sinai Hospital
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina- Chapel Hill
state: North Carolina
Study officials:
affiliation: Prokidney
name: Study Director
role: STUDY_DIRECTOR
References:
citation: 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.