Alport Syndrome
Conditions
Keywords
Kidney disease, Nephritis, Hereditary kidney disease, Hereditary nephritis
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.
Interventions
DRUGRG012
RG012 in 0.3% sodium chloride
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
United States
Outcome results
None listed