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

• Register: EUCTR
• Last refreshed on: 21 August 2023
• Main ID: EUCTR2016-003134-24-DE
• Date of registration: 11/07/2017
• Prospective Registration: Yes
• Primary sponsor: Alnylam Pharmaceuticals, Inc.
• Public title: Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1
• Scientific title: A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1
• Date of first enrolment: 28/12/2017
• Target sample size: 20
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-003134-24
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

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

Contacts

• Name: Clinical Trial Hotline
• Affiliation: Alnylam Pharmaceuticals Inc

• Name: Clinical Trial Hotline
• Affiliation: Alnylam Pharmaceuticals Inc

Key inclusion & exclusion 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:
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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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

Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Intervention(s)

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: ALN-65585
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 189-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: Patients will be assessed for safety at each study visit

Main Objective: Evaluate the long-term safety of multiple doses of ALN-GO1 in patients with PH1

Primary end point(s): Incidence of adverse events (AEs)

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

Secondary Outcome(s)

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.

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

Secondary ID(s)

ALN-GO1-002

Source(s) of Monetary Support

Alnylam Pharmaceuticals Inc

Ethics review

Status: Approved
Approval date: 28/12/2017
Contact: