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).

Prokidney

Status and phase: Completed; Phase 1

Conditions: Chronic Kidney Disease; Congenital Anomalies of Kidney and Urinary Tract

Treatments: Biological: Renal Autologous Cell Therapy (REACT)

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT04115345

REGEN-004

Details and patient eligibility

About

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)

Full 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.

Enrollment: 6 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 1 patient group

Renal Autologous Cell Therapy (REACT)

Experimental group

Description:

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.

Treatment:

Biological: Renal Autologous Cell Therapy (REACT)