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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-04-04

Study Completion Date

2023-02-07

Brief Summary

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

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Detailed Description

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Conditions

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PH1 Primary Hyperoxaluria RNAi Therapeutic siRNA AGT

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M

Participants enrolling from study 001B (NCT02706886), received lumasiran, subcutaneous (SC) injection, at a starting dose of 1.0 milligrams per kilograms (mg/kg) once monthly (QM) or 3.0 mg/kg once every 3 months \[Q3M\]) from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period.

All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the Safety Review Committee (SRC).

Group Type EXPERIMENTAL

Lumasiran

Intervention Type DRUG

Multiple doses of lumasiran by SC injection

Lumasiran (ALN-GO1): 3.0 mg/kg QM

Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period.

Group Type EXPERIMENTAL

Lumasiran

Intervention Type DRUG

Multiple doses of lumasiran by SC injection

Interventions

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Lumasiran

Multiple doses of lumasiran by SC injection

Intervention Type DRUG

Other Intervention Names

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ALN-GO1

Eligibility Criteria

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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

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No