A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

NCT ID: NCT04152200

Last Updated: 2025-07-18

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-01-21

Study Completion Date

2025-06-23

Brief Summary

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The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1 (PH1).

Detailed Description

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Conditions

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Primary Hyperoxaluria Type 1 Primary Hyperoxaluria

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Lumasiran

All patients will receive open-label lumasiran.

Group Type EXPERIMENTAL

Lumasiran

Intervention Type DRUG

Lumasiran will be administered by subcutaneous (SC) injection.

Interventions

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Lumasiran

Lumasiran will be administered by subcutaneous (SC) injection.

Intervention Type DRUG

Other Intervention Names

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ALN-GO1 OXLUMO

Eligibility Criteria

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Inclusion Criteria

* Has documented diagnosis of primary hyperoxaluria type 1 (PH1)
* Estimated glomerular filtration rate (eGFR) ≤45 mL/min/1.73 m\^2 for patients ≥12 months of age (\<12 months of age, must have serum creatinine considered elevated for age)
* Meets plasma oxalate level requirements
* If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
* If on dialysis, may be on hemodialysis therapy only and must have been on a stable regimen for at least 4 weeks

Exclusion Criteria

* Hemodialysis/peritoneal dialysis combination therapy or peritoneal dialysis alone
* Diagnosis of conditions other than PH1 contributing to renal insufficiency
* History of liver transplant
* History of kidney transplant and currently receiving immunosuppressants
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals

Locations

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Clinical Trial Site

Rochester, Minnesota, United States

Site Status

Clinical Trial Site

Houston, Texas, United States

Site Status

Clinical Trial Site

Garran, , Australia

Site Status

Clinical Trial Site

Brussels, , Belgium

Site Status

Clinical Trial Site

Bron, , France

Site Status

Clinical Trial Site

Lyon, , France

Site Status

Clinical Trial Site

Haifa, , Israel

Site Status

Clinical Trial Site

Nahariya, , Israel

Site Status

Clinical Trial Site

Rome, , Italy

Site Status

Clinical Trial Site

Irbid, , Jordan

Site Status

Clinical Trial Site

Beirut, , Lebanon

Site Status

Clinical Trial Site

Amsterdam, , Netherlands

Site Status

Clinical Trial Site

Yenimahalle, , Turkey (Türkiye)

Site Status

Clinical Trial Site

Dubai, , United Arab Emirates

Site Status

Countries

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United States Australia Belgium France Israel Italy Jordan Lebanon Netherlands Turkey (Türkiye) United Arab Emirates

References

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Michael M, Groothoff JW, Shasha-Lavsky H, Lieske JC, Frishberg Y, Simkova E, Sellier-Leclerc AL, Devresse A, Guebre-Egziabher F, Bakkaloglu SA, Mourani C, Saqan R, Singer R, Willey R, Habtemariam B, Gansner JM, Bhan I, McGregor T, Magen D. Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial. Am J Kidney Dis. 2023 Feb;81(2):145-155.e1. doi: 10.1053/j.ajkd.2022.05.012. Epub 2022 Jul 14.

Reference Type DERIVED
PMID: 35843439 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2019-001346-17

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-503382-29-00

Identifier Type: CTIS

Identifier Source: secondary_id

ALN-GO1-005

Identifier Type: -

Identifier Source: org_study_id

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