Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1

Phase 1

• Conditions: Primary Hyperoxaluria Type 1 (PH1); MedDRA version: 20.1Level: PTClassification code 10020703Term: HyperoxaluriaSystem Organ Class: 10038359 - Renal and urinary disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2016-003134-24-DE
• Lead Sponsor: Alnylam Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-07-11
• Last Update Posted: 21 August 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1. Enrollment within 12 months of completion of Study ALN-GO1-001 and in the opinion of the investigator, tolerated the study drug
2. If taking vitamin B6 (pyridoxine), willing to remain on a stable regimen for the study duration
3. Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception. Males with partners of child-bearing potential must agree to use an appropriate method of contraception
4. Willing to provide written informed consent and to comply with study requirements.

Are the trial subjects under 18? yes
Number of subjects for this age range: 16
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Any uncontrolled or serious disease, or any medical or surgical condition (with the exception of PH1) that may either interfere with participation in the clinical study, and/or put the patient significant risk (according to the Investigator’s judgment) if he/she participates in the clinical study.
2. Requirement for chronic dialysis
3. Echo assessment of abnormal left ventricular systolic function, defined as left ventricular ejection fraction <55% at Screening

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Evaluate the long-term safety of multiple doses of ALN-GO1 in patients with PH1 ;Secondary Objective: - Evaluate the pharmacodynamic (PD) effect of ALN-GO1 on urinary oxalate excretion<br>- Characterize the effect of ALN-GO1 on markers of renal function ;Primary end point(s): Incidence of adverse events (AEs);Timepoint(s) of evaluation of this end point: Patients will be assessed for safety at each study visit

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Change in 24-hour urinary oxalate corrected for body surface area (BSA) over time<br>- Change in 24-hour urinary oxalate:creatinine ratio over time<br>- Change in estimated glomerular filtration rate (eGFR) over time<br>;Timepoint(s) of evaluation of this end point: Pharmacodynamics assessments will be made quarterly over the first 12 months of the study, and approximately every 6 months for the remaining duration of the study.