Back to resultsA Study of RG-012 in Subjects With Alport Syndrome
Primary Purpose
Alport Syndrome
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
RG012
Sponsored by
About this trial
This is an interventional treatment trial for Alport Syndrome focused on measuring Kidney disease, Nephritis, Hereditary kidney disease, Hereditary nephritis
Eligibility Criteria
Inclusion Criteria:
- Males or females, ages 18 to 65 years
- Confirmed diagnosis of Alport syndrome
- eGFR between 40 and 90 mL/min/1.73m2
- Proteinuria of at least 300 mg protein/g creatinine
- For subjects taking an ACE inhibitor or an ARB, the dosing regimen should be stable for at least 30 days prior to screening
- Willing to comply with contraception requirements
Exclusion Criteria:
- Causes of chronic kidney disease aside from Alport syndrome (such as diabetic nephropathy, hypertensive nephropathy, lupus nephritis, or IgA nephropathy)
- End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transportation
- Any other condition that may pose a risk to the subject's safety and well-being
- Female subjects who are pregnant or lactating
Sites / Locations
- eStudySite
- Academic Medical Research Institute
- Apex Research of Riverside
- Eminence Medical & Clinical Research
- Houston Nephrology Research
- Utah Kidney Research Institute
- Allegiance Research Specialists, LLC
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
RG-012 Single Dose
RG012 Every Other Week
Arm Description
1.5 mg/kg RG012 subcutaneous injection
1.5 mg/kg RG012 subcutaneous injections every other week
Outcomes
Primary Outcome Measures
Safety - Adverse Events
Incidence and severity of adverse events
Effect of RG-012 on renal microRNA-21 (miR-21)
Change in miR-21 expression in renal tissue
Secondary Outcome Measures
Pharmacokinetic (PK) parameter - Cmax
Maximum observed plasma concentration
Pharmacokinetic (PK) parameter - Tmax
Time to maximum observed plasma concentration
Pharmacokinetic (PK) parameter - AUC
Area under the plasma concentration vs. time curve
Full Information
NCT ID
NCT03373786
First Posted
October 22, 2017
Last Updated
April 21, 2022
Sponsor
Genzyme, a Sanofi Company
1. Study Identification
Unique Protocol Identification Number
NCT03373786
Brief Title
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
2. Study Status
Record Verification Date
April 2022
Overall Recruitment Status
Completed
Study Start Date
December 22, 2017 (Actual)
Primary Completion Date
May 20, 2019 (Actual)
Study Completion Date
May 20, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alport Syndrome
Keywords
Kidney disease, Nephritis, Hereditary kidney disease, Hereditary nephritis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
ParticipantInvestigator
Allocation
Non-Randomized
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
RG-012 Single Dose
Arm Type
Experimental
Arm Description
1.5 mg/kg RG012 subcutaneous injection
Arm Title
RG012 Every Other Week
Arm Type
Experimental
Arm Description
1.5 mg/kg RG012 subcutaneous injections every other week
Intervention Type
Drug
Intervention Name(s)
RG012
Intervention Description
RG012 in 0.3% sodium chloride
Primary Outcome Measure Information:
Title
Safety - Adverse Events
Description
Incidence and severity of adverse events
Time Frame
8 weeks
Title
Effect of RG-012 on renal microRNA-21 (miR-21)
Description
Change in miR-21 expression in renal tissue
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
Pharmacokinetic (PK) parameter - Cmax
Description
Maximum observed plasma concentration
Time Frame
8 weeks
Title
Pharmacokinetic (PK) parameter - Tmax
Description
Time to maximum observed plasma concentration
Time Frame
8 weeks
Title
Pharmacokinetic (PK) parameter - AUC
Description
Area under the plasma concentration vs. time curve
Time Frame
8 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Males or females, ages 18 to 65 years
Confirmed diagnosis of Alport syndrome
eGFR between 40 and 90 mL/min/1.73m2
Proteinuria of at least 300 mg protein/g creatinine
For subjects taking an ACE inhibitor or an ARB, the dosing regimen should be stable for at least 30 days prior to screening
Willing to comply with contraception requirements
Exclusion Criteria:
Causes of chronic kidney disease aside from Alport syndrome (such as diabetic nephropathy, hypertensive nephropathy, lupus nephritis, or IgA nephropathy)
End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transportation
Any other condition that may pose a risk to the subject's safety and well-being
Female subjects who are pregnant or lactating
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations, M.D.
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
eStudySite
City
La Mesa
State/Province
California
ZIP/Postal Code
91942
Country
United States
Facility Name
Academic Medical Research Institute
City
Los Angeles
State/Province
California
ZIP/Postal Code
90022
Country
United States
Facility Name
Apex Research of Riverside
City
Riverside
State/Province
California
ZIP/Postal Code
92505
Country
United States
Facility Name
Eminence Medical & Clinical Research
City
Tampa
State/Province
Florida
ZIP/Postal Code
33604
Country
United States
Facility Name
Houston Nephrology Research
City
Cypress
State/Province
Texas
ZIP/Postal Code
77429
Country
United States
Facility Name
Utah Kidney Research Institute
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84107
Country
United States
Facility Name
Allegiance Research Specialists, LLC
City
Wauwatosa
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Learn more about this trial
A Study of RG-012 in Subjects With Alport Syndrome
We'll reach out to this number within 24 hrs