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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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21 August 2023 |
Main ID: |
EUCTR2016-003134-24-DE |
Date of registration:
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11/07/2017 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1
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Scientific title:
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A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1
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Date of first enrolment:
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28/12/2017 |
Target sample size:
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20 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-003134-24 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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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
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): no
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Countries of recruitment
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France
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Germany
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Israel
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Jordan
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Netherlands
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United Arab Emirates
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United Kingdom
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United States
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Contacts
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Name:
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Clinical Trial Hotline
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Address:
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300 Third Street
02142
Cambridge
United States |
Telephone:
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001 866 330 0326 |
Email:
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clinicaltrials@alnylam.com |
Affiliation:
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Alnylam Pharmaceuticals Inc |
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Name:
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Clinical Trial Hotline
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Address:
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300 Third Street
02142
Cambridge
United States |
Telephone:
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001 866 330 0326 |
Email:
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clinicaltrials@alnylam.com |
Affiliation:
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Alnylam Pharmaceuticals Inc |
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Key inclusion & exclusion criteria
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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
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Health Condition(s) or Problem(s) studied
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Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1
Level: PT
Classification code 10020703
Term: Hyperoxaluria
System Organ Class: 10038359 - Renal and urinary disorders
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Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
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Intervention(s)
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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-
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Primary Outcome(s)
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Main Objective: Evaluate the long-term safety of multiple doses of ALN-GO1 in patients with PH1
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Timepoint(s) of evaluation of this end point: Patients will be assessed for safety at each study visit
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Primary end point(s): Incidence of adverse events (AEs)
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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
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Secondary Outcome(s)
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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
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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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Secondary ID(s)
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ALN-GO1-002
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Source(s) of Monetary Support
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Alnylam Pharmaceuticals Inc
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Ethics review
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Status: Approved
Approval date: 28/12/2017
Contact:
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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