NCT04152200

Brief Summary

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).

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jan 2020

Longer than P75 for phase_3

Geographic Reach
11 countries

14 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 31, 2019

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 5, 2019

Completed
3 months until next milestone

Study Start

First participant enrolled

January 21, 2020

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 18, 2021

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

July 12, 2022

Completed
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 23, 2025

Completed
Last Updated

July 18, 2025

Status Verified

July 1, 2025

Enrollment Period

1.3 years

First QC Date

October 31, 2019

Results QC Date

May 18, 2022

Last Update Submit

July 8, 2025

Conditions

Primary Hyperoxaluria Type 1Primary Hyperoxaluria

Keywords

PH1Primary HyperoxaluriaHyperoxaluriaRNAi TherapeuticsiRNAAGTSystemic Oxalosis

Outcome Measures

Primary Outcomes (2)

  • Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6

    Percent change in plasma oxalate (umol/L) was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. For Cohort A, the baseline was defined as the mean of all plasma oxalate level values collected prior to the first dose of lumasiran.

    Baseline to Month 6

  • Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6

    Percent change in plasma oxalate (umol/L) was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. For Cohort B, the baseline is defined as the mean of the last four pre-dialysis plasma oxalate samples collected prior to the first dose of lumasiran. In Cohort B, only pre-dialysis samples are utilized.

    Baseline to Month 6

Secondary Outcomes (29)

  • Cohort B: Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 6

    Baseline to Month 6

  • Absolute Change in Plasma Oxalate From Baseline to Month 6

    Baseline to Month 6

  • Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6

    Baseline to Month 6

  • Cohort A: Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6

    Baseline to Month 6

  • Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6

    Baseline to Month 6

  • +24 more secondary outcomes

Study Arms (1)

Lumasiran

EXPERIMENTAL

All patients will receive open-label lumasiran.

Drug: Lumasiran

Interventions

LumasiranDRUG

Lumasiran will be administered by subcutaneous (SC) injection.

Also known as: ALN-GO1, OXLUMO
Lumasiran

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

Clinical Trial Site

Rochester, Minnesota, 55902, United States

Location

Clinical Trial Site

Houston, Texas, 77030, United States

Location

Clinical Trial Site

Garran, Australia

Location

Clinical Trial Site

Brussels, Belgium

Location

Clinical Trial Site

Bron, France

Location

Clinical Trial Site

Lyon, France

Location

Clinical Trial Site

Haifa, Israel

Location

Clinical Trial Site

Nahariya, Israel

Location

Clinical Trial Site

Rome, Italy

Location

Clinical Trial Site

Irbid, Jordan

Location

Clinical Trial Site

Beirut, Lebanon

Location

Clinical Trial Site

Amsterdam, Netherlands

Location

Clinical Trial Site

Yenimahalle, Turkey (Türkiye)

Location

Clinical Trial Site

Dubai, United Arab Emirates

Location

Related Publications (1)

  • 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.

    PMID: 35843439DERIVED

MeSH Terms

Conditions

Primary hyperoxaluria type 1Hyperoxaluria, PrimaryHyperoxaluria

Interventions

lumasiran

Condition Hierarchy (Ancestors)

Kidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Results Point of Contact

Title
Chief Medical Officer
Organization
Alnylam Pharmaceuticals Inc.
clinicaltrials@alnylam.com
866-330-0326

Study Officials

  • Medical Director

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR
Expanded Access
Yes

Study Record Dates

First Submitted

October 31, 2019

First Posted

November 5, 2019

Study Start

January 21, 2020

Primary Completion

May 18, 2021

Study Completion

June 23, 2025

Last Updated

July 18, 2025

Results First Posted

July 12, 2022

Record last verified: 2025-07

Data Sharing

IPD Sharing
Will share

Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.

Locations

JO(1)US(2)BE(1)IL(2)NL(1)TU(1)AE(1)LE(1)FR(2)IT(1)AU(1)