An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Patients With Primary Hyperoxaluria Type 1

Sponsor

Alnylam Pharmaceuticals (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT03350451

Collaborator

(none)

Enrollment

Locations

Arm

56.9

Anticipated Duration (Months)

2.2

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to evaluate the long-term safety and tolerability of lumasiran in patients with Primary Hyperoxaluria Type 1.

Condition or Disease	Intervention/Treatment	Phase
PH1 Primary Hyperoxaluria RNAi Therapeutic siRNA AGT	Drug: Lumasiran	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

20 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1

Actual Study Start Date :

Apr 4, 2018

Anticipated Primary Completion Date :

Jan 1, 2023

Anticipated Study Completion Date :

Jan 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Lumasiran (ALN-GO1)	Drug: Lumasiran Multiple doses of lumasiran by subcutaneous (sc) injection Other Names: ALN-GO1

Arm

Intervention/Treatment

Experimental: Lumasiran (ALN-GO1)

Drug: Lumasiran

Multiple doses of lumasiran by subcutaneous (sc) injection

Other Names:

ALN-GO1

Outcome Measures

Primary Outcome Measures

Incidence of Adverse Events [Up to approximately 1600 days]

Secondary Outcome Measures

Change in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) Over Time [Up to approximately 1600 days]
Change in 24-hour Urinary Oxalate:Creatinine Ratio Over Time [Up to approximately 1600 days]
Change in Estimated Glomerular Filtration Rate (eGFR) Over Time [Up to approximately 1600 days]

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Enrollment within 12 months of completion of Study ALN-GO1-001
In the opinion of the investigator tolerated the study drug
If taking Vitamin B6 (pyridoxine), willing to remain on a stable regimen for the study duration
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
Willing to provide written informed consent and to comply with study requirements

Exclusion Criteria:

Clinically significant health concerns (with the exception of PH1)
Clinically significant cardiovascular abnormality
Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant
Requirement for chronic dialysis

Contacts and Locations

Locations

	Site	City	Country
1	Clinical Trial Site	Bordeaux	France
2	Clinical Trial Site	Lyon	France
3	Clinical Trial Site	Paris	France
4	Clinical Trial Site	Bonn	Germany
5	Clinical Trial Site	Haifa	Israel
6	Clinical Trial Site	Jerusalem	Israel
7	Clinical Trial Site	Amsterdam	Netherlands
8	Clinical Trial Site	Birmingham	United Kingdom
9	Clinical Trial Site	London	United Kingdom