A Phase 1 Study of DCR-PH1 in Patients with Primary Hyperoxaluria Type 1 (PH1)

Completed

• Conditions: Autosomal recessive disorder; PH1

• Registration Number: NL-OMON44062
• Lead Sponsor: Dicerna Pharmaceuticals, lnc

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 homozygosity or compound heterozygosity in
the AGXT gene (historically available genotype information is acceptable for study eligibility).
- 24-hour urine oxalate excretion * 0.7 mmol per 1.73 m2 body surface area (BSA).
- Estimated glomerular filtration rate * 40 mL/min normalized to 1.73 m2 BSA calculated using
the Modification of Diet in Renal Disease (MDRD) formula in adults (age * 18 years).

Exclusion Criteria

- Prior renal and/or hepatic transplantation.
- History of clinical signs and symptoms of systemic oxalosis other than nephrolithiasis or
nephrocalcinosis.
- Participation in any clinical study involving administration of any investigational drug within
the 30 days before enrollment

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Safety and tolerability as determined by number of subjects with adverse events</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>1) Determination of pharmacokinetics parameters<br /><br>2) Change in plasma levels from baseline (BL) to each time point of oxalate and<br /><br>glycolate<br /><br>3) Change in urine levels from baseline (BL) of oxalate, oxalate to creatinine<br /><br>ratio and glycolate</p><br>