A Phase 1/2, Single-Blind, Placebo-Controlled, Single- and Multiple-Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered<br>ALN-GO1 in Healthy Adult Subjects, and Patients with Patients with Primary Hyperoxaluria Type 1
- Conditions
- Primary Hyperoxaluria Type 110021605
- Registration Number
- NL-OMON47017
- Lead Sponsor
- Alnylam Pharmaceuticals, Inc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
1. Male and female subjects aged 18-64 years (or age of legal consent,
whichever is older), inclusive (Part A) and 6-64 years, inclusive (Part B).
2. Women of child bearing potential must have a negative pregnancy
test, cannot be breastfeeding, and must be willing to use contraception.
3. Willing to provide written informed consent and to comply with study
requirements.
Additional Inclusion Criteria for Part B:
4. confirmation of PH1 disease.
5. 24-hour urinary oxalate excretion of >0.7 mmol/1.73m2/day.
6.Estimated GFR of >45 mL/min/1.73m2.
7. If taking Vitamin B6 (pyridoxine), must have been on stable regimen
for at least 90 days .
1. Any uncontrolled or serious disease, or any medical or surgical
condition (with the exception of PH1 for patients in Part B) that may
either interfere with participation in the clinical study, and/or put the
subject at significant risk (according to the Investigator's judgment) if
he/she participates in the clinical study.
2. Mental illness, alcoholism, drug abuse, or heavy smokers and users of
nicotine
3. History of multiple drug allergies or intolerance to subcutaneous
injection
4. Received an investigational agent within 3 months before the first
dose of study drug or are in follow-up of another clinical study
5. Known history of allergic reaction to an oligonucleotide or GalNAc
6. History of intolerance to SC injection or relevant abdominal scarring
7. Women who are pregnant or breast feeding
Part B only
8. Echocardiography (ECHO) assessment of normal left ventricular systolic function, defined as left ventricular ejection fraction <55% at screening
9. Troponin I > the upper limit of normal (ULN) at screening
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary<br /><br>* The primary endpoint is the incidence of adverse events. Safety will also be<br /><br>evaluated through vital signs, electrocardiograms, clinical laboratory<br /><br>assessments, and physical examinations.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Secondary Endpoints for Part B<br /><br>o PK parameters including, but not limited to, Cmax, tmax, AUC, t*, fe/F, and<br /><br>CLR<br /><br>o Urinary oxalate excretion (oxalate concentration in 24-hour urine collection)<br /><br>o Urinary glycolate excretion (glycolate concentration in 24-hour urine<br /><br>collection)<br /><br>o Plasma glycolate concentration<br /><br>o Calculated creatinine clearance</p><br>