A Study of RG-012 in Subjects With Alport Syndrome

Phase 1

Completed

• Conditions: Alport Syndrome
• Interventions: Drug: RG012

• Registration Number: NCT03373786
• Lead Sponsor: Genzyme, a Sanofi Company

Brief 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.

Detailed 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.

Study Timeline

• Study First Submitted: 2017-10-22
• Study First Submitted QC: 2017-12-13
• Study First Posted: 2017-12-14
• Study Start: 2017-12-22
• Primary Completion: 2019-05-20
• Study Completion: 2019-05-20
• Status Verified: 2022-04
• Last Update Submitted: 2022-04-21
• Last Update Posted: 2022-04-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 4

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
RG012 Every Other Week	RG012	1.5 mg/kg RG012 subcutaneous injections every other week
RG-012 Single Dose	RG012	1.5 mg/kg RG012 subcutaneous injection

Primary Outcome Measures

Name	Time	Method
Safety - Adverse Events	8 weeks	Incidence and severity of adverse events
Effect of RG-012 on renal microRNA-21 (miR-21)	8 weeks	Change in miR-21 expression in renal tissue

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetic (PK) parameter - Cmax	8 weeks	Maximum observed plasma concentration
Pharmacokinetic (PK) parameter - Tmax	8 weeks	Time to maximum observed plasma concentration
Pharmacokinetic (PK) parameter - AUC	8 weeks	Area under the plasma concentration vs. time curve

Trial Locations

Locations (7)

Academic Medical Research Institute; 🇺🇸 Los Angeles, California, United States

eStudySite; 🇺🇸 La Mesa, California, United States

Eminence Medical & Clinical Research; 🇺🇸 Tampa, Florida, United States

Apex Research of Riverside; 🇺🇸 Riverside, California, United States

Utah Kidney Research Institute; 🇺🇸 Salt Lake City, Utah, United States

Houston Nephrology Research; 🇺🇸 Cypress, Texas, United States

Allegiance Research Specialists, LLC; 🇺🇸 Wauwatosa, Wisconsin, United States