This website uses cookies to enhance your browsing experience, improve site performance, and gather analytics. By selecting 'Accept,' you consent to these cookies as described in our Privacy Policy.

A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

Sponsored by Alnylam Pharmaceuticals

About this trial

Last updated a year ago

Study ID

ALN-GO1-003

Status

Completed

Type

Interventional

Phase

Phase 3

Placebo

Yes

Accepting

18-75 Years
6+ Years
All
All

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Ended 2 years ago

What is this trial about?

The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).

What are the participation requirements?

Yes

Inclusion Criteria

- Willing to provide written informed consent or assent and to comply with study requirements

- Confirmation of PH1 disease

- Meet the 24 hour urine oxalate excretion requirements

- If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days

No

Exclusion Criteria

- Clinically significant health concerns (with the exception of PH1) or clinical evidence of extrarenal systemic oxalosis

- Clinically significant abnormal laboratory results

- Known active or evidence of HIV or hepatitis B or C infection

- An estimated GFR of < 30 mL/min/1.73m^2 at screening

- Received an investigational agent within 30 days or 5 half-lives before the first dose of study drug or are in follow-up of another clinical study

- History of kidney or liver transplant

- Known history of multiple drug allergies or allergic reaction to an oligonucleotide or GalNAc

- History of intolerance to subcutaneous injection

- Women who are pregnant, planning a pregnancy, or breast-feeding or those of child bearing potential and not willing to use contraception

- History of alcohol abuse within the last 12 months, or unable or unwilling to limit alcohol consumption throughout the study

Trial Results Summary

Locations

Location

Status

For more information, view the full study details:

NCT036811842018-001981-40