Chronic Kidney Disease, Congenital Anomalies of Kidney and Urinary Tract
Conditions
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.
Interventions
BIOLOGICALRenal Autologous Cell Therapy (REACT)
Autologous selected renal cells (SRC)
Sponsors
CTI Clinical Trial and Consulting Services
Prokidney
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-label
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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]. | 18 months following last REACT injection | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of subjects with renal-specific adverse events over a 18-month period following injection of Renal Autologous Cell Therapy (REACT). | 18 months following last REACT injection | 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. |
Countries
United States
Outcome results
None listed