A Study of RG-012 in Subjects With Alport Syndrome

Alport Syndrome Clinical Trial

Official title:

A Phase 1, Open-Label Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of RG-012 for Injection, Including Its Effect on Renal microRNA-21, in Subjects With Alport Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03373786
• Other study ID #: PDY16327
• Secondary ID: RG012-06
• Status: Completed
• Phase: Phase 1
• Start date: December 22, 2017
• Est. completion date: May 20, 2019

Study information

• Verified date: April 2022
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: May 20, 2019
• Est. primary completion date: May 20, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Alport Syndrome
• Nephritis, Hereditary
• Syndrome

Intervention

Drug:
• RG012
RG012 in 0.3% sodium chloride

Locations

Country	Name	City	State
United States	Houston Nephrology Research	Cypress	Texas
United States	eStudySite	La Mesa	California
United States	Academic Medical Research Institute	Los Angeles	California
United States	Apex Research of Riverside	Riverside	California
United States	Utah Kidney Research Institute	Salt Lake City	Utah
United States	Eminence Medical & Clinical Research	Tampa	Florida
United States	Allegiance Research Specialists, LLC	Wauwatosa	Wisconsin

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety - Adverse Events	Incidence and severity of adverse events	8 weeks
Primary	Effect of RG-012 on renal microRNA-21 (miR-21)	Change in miR-21 expression in renal tissue	8 weeks
Secondary	Pharmacokinetic (PK) parameter - Cmax	Maximum observed plasma concentration	8 weeks
Secondary	Pharmacokinetic (PK) parameter - Tmax	Time to maximum observed plasma concentration	8 weeks
Secondary	Pharmacokinetic (PK) parameter - AUC	Area under the plasma concentration vs. time curve	8 weeks