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

Completed · Phase 2

Conditions studied: Alport Syndrome, Focal Segmental Glomerulosclerosis

In brief

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

Key facts

Study ID: NCT05267262
Run by: River 3 Renal Corp.
People needed: 43
Starts: 2022-06-15
Expected to finish: 2025-08-19
Last updated by the study team: 2025-09-29

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

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.
Alport Syndrome Patients Inclusion Criteria (in addition):
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.
Confirmed diagnosis of AS by genetic testing and /or kidney biopsy. For patients enrolled in the US who meet all inclusion and exclusion criteria but have not had their diagnosis confirmed by genetic testing or kidney biopsy, the Sponsor will provide for patient's genetic testing.
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.
Focal Segmental Glomerulosclerosis Patients Inclusion Criteria (in addition):
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.

You may not qualify if…

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).
Children: ≥ 95th percentile or ≥ 130/80 mm Hg, whichever is lower
Moderate or severe hepatic impairment (Child-Pugh B or C), except if (a) decreased serum albumin is directly related to the renal disease (resulting in a Child Pugh score of 7), and (b) no other Child-Pugh Score parameters are increased and (c) patient has no liver pathology in medical history.
Presence of any active (i.e., with symptoms) and/or uncontrolled infection (including COVID).
Presence of Human immunodeficiency virus (HIV).
BMI > 40. Note - For Germany: BMI > 35 (Obesity Class II).
History of malignancy other than treated basal cell or squamous cell skin cancer within the past 5 years.
History of alcohol abuse in the last 5 years or currently drinks in excess of 21 and 14 units per week for males and females, respectively.
Received an investigational agent within 30 days or 5 half-lives prior to screening (whichever is longer).
History of non-compliance such that patient is unlikely to be compliant with study visits, procedures or drug administration.
Patient has had an organ transplant, is currently on an organ transplant waiting list or there is a reasonable possibility that the patient will have an organ transplant in the 6 months after screening.
Participation in an interventional trial within the previous 3 months prior to screening or concurrent participation in a research trial.
Patient is not suitable to participate in the study for any reason (including, but not limited to co-morbidities, history of non-compliance with study visits, procedures, or drug administration) in the opinion of the investigator.
Females of childbearing potential (those who are not surgically sterilized or post-menopausal for at least 1 year) are excluded from participation in the study unless they agree to use highly effective contraception.
Females that are lactating.
History of hypersensitivity to study drug and/or any of its excipients.
Patients with hereditary galactose intolerance, total lactase deficiency or glucose-galactose malabsorption.
Required concomitant use of bardoxolone, rituximab, cyclo-phosphamide, abatacept, or sparsentan
Alport Syndrome Patients Exclusion Criteria (in addition):
Kidney disease apart from AS, e.g. diabetic nephropathy or lupus nephritis.
Use of Bardoxolone or sparsentan treatment in the 30 days prior to screening. SGLT2 inhibitors are allowed if the patient is on a stable dose for at least 3 months prior to enrollment and during the study.
Focal Segmental Glomerulosclerosis Patients Exclusion Criteria (in addition):

Where it is running

Investigative Site — Los Angeles, California, United States
Investigative site — Boca Raton, Florida, United States
Investigative site — Miami, Florida, United States
Investigative site — Riverview, Florida, United States
Investigative site — Atlanta, Georgia, United States
Investigative site — Boston, Massachusetts, United States
Investigative site — Ann Arbor, Michigan, United States
Investigative site — Minneapolis, Minnesota, United States
Investigative Site — Cary, North Carolina, United States
Investigative site — Cleveland, Ohio, United States
Investigative site — Columbus, Ohio, United States
Investigative site — East Providence, Rhode Island, United States
Investigative Site — Dallas, Texas, United States
Investigative site — Houston, Texas, United States
Investigative site — Brussels, Belgium
Investigative site — Liège, Belgium
Investigative site — Paris, France
Investigative site — Göttingen, Germany
Investigative site — Amsterdam, Netherlands
Investigative site — Nijmegen, Netherlands
Investigative site — Leicester, United Kingdom
Investigative site — Nottingham, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.