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

A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-003134-24-DE

Enrollment

Registered

2017-07-11

Start date

2017-12-28

Completion date

Unknown

Last updated

2023-08-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1 Level: PT Classification code 10020703 Term: Hyperoxaluria System Organ Class: 10038359 - Renal and urinary disorders

Interventions

Product Name: lumasiran Product Code: ALN-GO1 Pharmaceutical Form: Solution for injection INN or Proposed INN: lumasiran CAS Number: 1834610-13-7 Current Sponsor code: ALN-GO1 Other descriptive name:

Eligibility

Sex/Gender

All

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
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 - 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	—

Measure

Time frame

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 - 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

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Secondary

Measure	Time frame
Secondary end point(s): - Change in 24-hour urinary oxalate corrected for body surface area (BSA) over time - Change in 24-hour urinary oxalate:creatinine ratio over time - Change in estimated glomerular filtration rate (eGFR) over time ;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.	—

Measure

Time frame

Secondary end point(s): - Change in 24-hour urinary oxalate corrected for body surface area (BSA) over time - Change in 24-hour urinary oxalate:creatinine ratio over time - Change in estimated glomerular filtration rate (eGFR) over time ;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.

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Countries

France, Germany, Israel, Jordan, Netherlands, United Arab Emirates, United Kingdom, United States

Contacts

Public ContactClinical Trial Hotline

Alnylam Pharmaceuticals Inc

clinicaltrials@alnylam.com001 866 330 0326

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026