Clinical trial NCT03373786: A Study of RG-012 in Subjects With Alport Syndrome
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Alport Syndrome
Keywords: Kidney disease
Nephritis
Hereditary kidney disease
Hereditary nephritis
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2017-12-14
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Safety - Adverse Events
timeFrame: 8 weeks
measure: Effect of RG-012 on renal microRNA-21 (miR-21)
timeFrame: 8 weeks
Secondary outcomes:
measure: Pharmacokinetic (PK) parameter - Cmax
timeFrame: 8 weeks
measure: Pharmacokinetic (PK) parameter - Tmax
timeFrame: 8 weeks
measure: Pharmacokinetic (PK) parameter - AUC
timeFrame: 8 weeks
Other outcomes:
Locations:
city: La Mesa
country: United States
facility: eStudySite
state: California
city: Los Angeles
country: United States
facility: Academic Medical Research Institute
state: California
city: Riverside
country: United States
facility: Apex Research of Riverside
state: California
city: Tampa
country: United States
facility: Eminence Medical & Clinical Research
state: Florida
city: Cypress
country: United States
facility: Houston Nephrology Research
state: Texas
city: Salt Lake City
country: United States
facility: Utah Kidney Research Institute
state: Utah
city: Wauwatosa
country: United States
facility: Allegiance Research Specialists, LLC
state: Wisconsin
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations, M.D.
role: STUDY_DIRECTOR
References: