PHYOS: An observational study of patients with Primary Hyperoxaluria type1

Completed

• Conditions: autosomal recessive disorders; PH1

• Registration Number: NL-OMON42592
• Lead Sponsor: Dicerna Pharmaceuticals, Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 7

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

- 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.

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>N.A.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>N.A.</p><br>