autosomal recessive disorders PH1
Conditions
Interventions
None listed
Sponsors
Dicerna Pharmaceuticals, Inc
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - Diagnosis of PH1, confirmed by genotyping for mutations in the AGXT gene. - Urine oxalate excretion >=0.7 mmol per 1.73 m2 body surface area (BSA) in 24 hours. - Estimated glomerular filtration rate (eGFR) >=40 mL/min per 1.73 m2 BSA.
Exclusion criteria
Exclusion criteria: - Prior renal and/or hepatic transplantation, or patients undergoing dialysis. - Pregnancy or lactation at the time of screening or enrollment. - Any significant illness, organ system dysfunction, or other condition that, in the opinion of the Investigator, would interfere with the subject*s ability to comply with the protocol requirements, including the ability to attend all visits and undergo all assessments.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| N.A. | — |
Secondary
| Measure | Time frame |
|---|---|
| N.A. | — |
Countries
Netherlands
Outcome results
None listed