A Study of RG-012 in Subjects With Alport Syndrome

Genzyme, a Sanofi Company4 enrolled

Overview

This is a Phase 1, open-label, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome.

This is a Phase 1, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome. During this open-label study, all eligible subjects will receive RG-012. The study consists of two parts (Part A and Part B). During Part A, half of the participants will receive a single dose of RG-012 and half will receive 4 doses of RG-012 (one dose every other week for 6 weeks). All subjects will undergo two renal biopsies, one before and one after receiving RG-012, to assess the effect of RG-012 on the kidney. After completing Part A, subjects will be able to enter Part B of the study. During Part B, all subjects will receive RG-012 every other week for 48 weeks.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Alport Syndrome

Interventions

RG012DRUG

RG012 in 0.3% sodium chloride

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Males or females, ages 18 to 65 years 2. Confirmed diagnosis of Alport syndrome 3. eGFR between 40 and 90 mL/min/1.73m2 4. Proteinuria of at least 300 mg protein/g creatinine 5. For subjects taking an ACE inhibitor or an ARB, the dosing regimen should be stable for at least 30 days prior to screening 6. Willing to comply with contraception requirements Exclusion Criteria: 1. Causes of chronic kidney disease aside from Alport syndrome (such as diabetic nephropathy, hypertensive nephropathy, lupus nephritis, or IgA nephropathy) 2. End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transportation 3. Any other condition that may pose a risk to the subject's safety and well-being 4. Female subjects who are pregnant or lactating

Locations (7)

eStudySite

La Mesa, California, United States

Academic Medical Research Institute

Los Angeles, California, United States

Apex Research of Riverside

Riverside, California, United States

Eminence Medical & Clinical Research

Tampa, Florida, United States

Outcomes

Primary Outcomes

Safety - Adverse Events

Incidence and severity of adverse events

Time frame: 8 weeks

Effect of RG-012 on renal microRNA-21 (miR-21)

Change in miR-21 expression in renal tissue

Time frame: 8 weeks

Secondary Outcomes

Pharmacokinetic (PK) parameter - Cmax

Maximum observed plasma concentration

Time frame: 8 weeks

Pharmacokinetic (PK) parameter - Tmax

Time to maximum observed plasma concentration

Time frame: 8 weeks

Pharmacokinetic (PK) parameter - AUC

Area under the plasma concentration vs. time curve

Time frame: 8 weeks

Data from ClinicalTrials.gov

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