Study to Evaluate R3R01 in Patients With Alport Syndrome and Patients With Focal Segmental Glomerulosclerosis

NCT05267262 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: R3R01-ASFSGS-201
• Study Type: interventional
• Target: 43
• Locations: 6 countries
• Sites: 22

Brief Summary

This is a Phase 2, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

Conditions

Alport Syndrome; Focal Segmental Glomerulosclerosis

Keywords

FSGS; Kidney Disease; glomeruli

Outcome Measures

Primary Outcomes (2)

• Incidence of adverse events (Safety and Tolerability)

  Safety and tolerability as determined by the incidence of adverse events (AEs)

  12 weeks

• Assess change in urine creatinine protein ratio

  Change from baseline in urine creatinine protein ratio for Cohort 1 (Alport Syndrome patient group) and Cohort 2(Focal Segmental Glomerulosclerosis patient group).

  12 weeks

Secondary Outcomes (2)

• Change in quality-of-life assessment from baseline to end of treatment and to the end of the follow-up period by cohort for adults

  24 weeks

• Change in quality-of-life assessment from baseline to end of treatment and to the end of the follow-up period by cohort for children

  24 weeks

Study Arms (2)

Cohort 2 (Alport Syndrome Patients)

EXPERIMENTAL

R3R01 administered orally as 200 mg tablets twice daily for 84 days.

Drug: R3R01

Cohort 3 (Focal Segmental Glomerulosclerosis Patients)

EXPERIMENTAL

R3R01 administered orally as 200 mg tablets twice daily for the 84 days.

Drug: R3R01

Interventions

R3R01 DRUG

R3R01 administered orally for 12 weeks

Cohort 2 (Alport Syndrome Patients); Cohort 3 (Focal Segmental Glomerulosclerosis Patients)

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• All Patients:
• Patient is able to communicate well with the investigator, understands and is willing to comply with all requirements of the study, and understands and signs the written informed consent form (ICF).
• For children to be eligible, one or both parents/legal guardians must sign a parental permission form which provides information contained in the ICF. Children capable of assent must express their willingness to participate by signing an assent form.
• If patient has received a COVID vaccination, the baseline visit must occur at least one week or more after the second/booster vaccination.
• Patients who have had active symptoms of COVID within 3 months prior to screening and are now asymptomatic for the last 2 weeks but have tested COVID PCR positive. If a patient is asymptomatic at screening but is COVID positive, then rescreening can occur after a minimum of two weeks.
• Both female patients, as well as, female partners of male patients who are of child-bearing potential must be willing to not become pregnant for the complete duration of the study (\>180 days) (90 days after the last dose of study medication).
• Males (including sterilized subjects) whose female partners have child-bearing potential, must agree to use male contraception (condoms) during the period from the time of signing the informed consent form (ICF) through 90 days after the last dose of study drug. They must agree to immediately inform the investigator if their partner becomes pregnant during the study.
• Males and females with X-Linked AS and males and females with autosomal inherited AS.
• For countries that are enrolling pediatric patients: patients from age 12 years and older.
• For countries that are not enrolling pediatric patients: patients from age 18 years and older.

You may not qualify if:

• UPCR ≥1.0 g/g.
• eGFR ≥ 30 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside Schwartz equation for children).
• ACEi/ARB therapy at maximum tolerated dose stable for at least 4 weeks prior to enrollment and during the study.
• Male or female patients,
• For countries that are enrolling pediatric patients: 12 to 75 years old at the time of signing the informed consent
• For countries that are not enrolling pediatric patients: 18 to 75 years old at the time of signing the informed consent
• Primary FSGS, (without any identifiable cause, and where the FSGS is confirmed by renal biopsy) or FSGS where there is documentation of a genetic mutation in a podocyte protein associated with FSGS.
• If on steroids, the dose should remain stable for at least 4 weeks prior to enrollment and during the study. Subjects who are steroid-resistant, defined as failure to achieve partial or complete remission, or subjects who experienced adverse events without acceptable clinical benefit after at least 8 weeks of adequate corticosteroid therapy for children and 12 weeks for adults are eligible.
• UPCR between 1.5g/g and 12.0g/g.
• eGFR \> 30 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside Schwartz equation for children).
• If taking concomitant ACEi and/or ARB treatment, it should remain at a stable dose for at least 4 weeks prior to enrollment and during the study.
• All Patients:
• Uncontrolled diabetes mellitus as evidenced by an HbA1c ≥ 11%. For Germany: HbA1c ≥ 8.5%.
• Uncontrolled hypertension
• Adults: (SBP ≥ 180mmHg and/or DBP ≥ 100mmHg). For Germany: (SBP ≥ 140mmHg and/or DBP ≥ 100mmHg).

Sponsors & Collaborators

• River 3 Renal Corp.: lead

Study Sites (22)

Investigative Site

Los Angeles, California, 90022, United States

Location

Investigative site

Boca Raton, Florida, 33431, United States

Location

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Miami, Florida, 33136, United States

Location

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Riverview, Florida, 33578, United States

Location

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Atlanta, Georgia, 30322, United States

Location

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Boston, Massachusetts, 02111, United States

Location

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Ann Arbor, Michigan, 48109-5718, United States

Location

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Minneapolis, Minnesota, 55454, United States

Location

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Cary, North Carolina, 27511, United States

Location

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Cleveland, Ohio, 44195, United States

Location

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Columbus, Ohio, 43235, United States

Location

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East Providence, Rhode Island, 02914, United States

Location

Investigative Site

Dallas, Texas, 75204, United States

Location

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Houston, Texas, 77054, United States

Location

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Brussels, 1200, Belgium

Location

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Liège, 4000, Belgium

Location

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Paris, 75015, France

Location

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Göttingen, 37075, Germany

Location

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Amsterdam, 1105AZ, Netherlands

Location

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Nijmegen, 6525 GA, Netherlands

Location

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Leicester, LE5 4PW, United Kingdom

Location

Investigative site

Nottingham, NG5 1PB, United Kingdom

Location

MeSH Terms

Conditions

Nephritis, Hereditary; Glomerulosclerosis, Focal Segmental; Kidney Diseases

Condition Hierarchy (Ancestors)

Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Nephritis; Urologic Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Glomerulonephritis

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 23, 2022
• First Posted: March 4, 2022
• Study Start: June 15, 2022
• Primary Completion: May 21, 2025
• Study Completion: August 19, 2025
• Last Updated: September 29, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

BE (2); US (14); FR (1); DE (1); GB (2); NL (2)