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).

Study Overview
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Brief Summary
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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
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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.

Official Title
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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)

Conditions
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Chronic Kidney Disease, Congenital Anomalies of Kidney and Urinary Tract

Intervention / Treatment
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* Biological: Renal Autologous Cell Therapy (REACT)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The patient is male or female, 18 to 65 years of age on the date of informed consent. The patient has a documented history of a corrected abnormality of the kidney and/or urinary tract (CAKUT) leading to renal dysfunction. The patient has an established diagnosis of Stage III/IV CKD not requiring renal dialysis. 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: The patient has a history of renal transplantation. The patient has a diagnosis of hydronephrosis, SFU Grade 4 or 5. The patient has an uncorrected VUR Grade 5. The patient has a diffuse cortical thickness throughout the kidney measuring < 5 mm on MRI that prevents safe cortical biopsy.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Open-label
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Renal Autologous Cell Therapy (REACT)<br>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)<br>* Autologous selected renal cells (SRC)<br>|

What is the study measuring?
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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 |