Sickle Cell Disease (SCD) and Nephropathy
Conditions
Brief summary
ACR response, defined as a ≥30% decrease in ACR from baseline to Month 6
Detailed description
1. Change from baseline in eGFRcr-cys 2. Change from baseline in ACR 3. Stable ACR, defined as Month 6 ACR within ±20% of baseline ACR 4. Annualized rate of visits to emergency room (ER) 5. Annualized rate of days of hospitalizations 6. Type, frequency and severity of AEs and SAEs and relationship to study drug
Interventions
DRUGMITAPIVAT
Sponsors
Agios Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| ACR response, defined as a ≥30% decrease in ACR from baseline to Month 6 | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Change from baseline in eGFRcr-cys 2. Change from baseline in ACR 3. Stable ACR, defined as Month 6 ACR within ±20% of baseline ACR 4. Annualized rate of visits to emergency room (ER) 5. Annualized rate of days of hospitalizations 6. Type, frequency and severity of AEs and SAEs and relationship to study drug | — |
Countries
France, Ireland
Outcome results
None listed