The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 Disease

Phase 1

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

• Registration Number: EUCTR2015-004407-23-DE
• Lead Sponsor: Alnylam Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-03-07
• Study Completion: 2019-01-23
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 52

Inclusion Criteria

Inclusion Criteria for Parts A and B:
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
Are the trial subjects under 18? yes
Number of subjects for this age range: 16
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 36
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Exclusion Criteria for Parts A and B:
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 clinical trial of medicinal product
• Study Design: Not specified