A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1.
- Conditions
- Primary Hyperoxaluria Type 1metabolic disorders10038430
- Registration Number
- NL-OMON50391
- Lead Sponsor
- Alnylam Pharmaceuticals, Inc
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
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 from 14 days before first dose and throughout study participation
until the completion of the follow-up period.
4. Willing and able to comply with the study requirements and to provide
written informed consent and assent in the case of patients under the age of
legal consent, per local and national requirements.
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. An underlying known disease or surgical or medical condition (with the
exception of PH1) that in the opinion of the investigator might interfere with
the interpretation of the clinical study results
3. Requirement for chronic dialysis
4. Triplicate 12-lead ECG with clinically significant abnormalities at Baseline
visit, at the discretion of the investigator
5. Echo assessment of abnormal left ventricular systolic function, defined as
left ventricular ejection fraction <55% at Screening
6. Abnormal for aspartate aminotransferase (AST)/alanine aminotransferase (ALT)
and any other clinical safety laboratory result considered clinically
significant and unacceptable by the Investigator at Baseline, at discretion of
investigator
7. Legal incapacity or limited legal capacity at Screening of patient, parent,
or legal guardian
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary study outcome is the incidence of adverse events (AEs)</p><br>
- Secondary Outcome Measures
Name Time Method