A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

NCT03905694 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: ALN-GO1-0042018-004014-17
• Study Type: interventional
• Target: 18
• Locations: 5 countries
• Sites: 9

Brief Summary

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).

Conditions

Primary Hyperoxaluria; Primary Hyperoxaluria Type 1 (PH1)

Keywords

PH1; Primary Hyperoxaluria; Hyperoxaluria; RNAi Therapeutic; siRNA; AGT

Outcome Measures

Primary Outcomes (1)

• Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6

  Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.

  Baseline to Month 6

Secondary Outcomes (17)

• Percentage Change in Spot Urinary Oxalate: Creatinine Ratio in the Extension Period (Month 6 to End of Study [Month 60])

  From Month 6 to Month 60

• Percentage of Time That Spot Urinary Oxalate: Creatinine Ratio is at or Below the Near-normalization Threshold (≤1.5 × Upper Limit of Normal (ULN) for Age)

  Up to 60 months

• Absolute Change in Spot Urinary Oxalate: Creatinine Ratio From Baseline

  From Baseline to Month 6 and Month 60

• Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ the ULN for Age

  Up to 60 months

• Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ 1.5xULN for Age

  Up to 60 months

Study Arms (1)

Lumasiran

EXPERIMENTAL

Lumasiran will be administered by subcutaneous (SC) injection.

Drug: Lumasiran

Interventions

Lumasiran DRUG

Lumasiran will be administered by subcutaneous (SC) injection.

Also known as: ALN-GO1

Lumasiran

Eligibility Criteria

• Age: 0 Years - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Has genetic confirmation of primary hyperoxaluria type 1 (PH1)
• Meets urinary oxalate excretion requirements
• If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days

You may not qualify if:

• If \<12 months old at screening, has an abnormally high serum creatinine
• If ≥12 months old at screening, has an estimated glomerular filtration rate (GFR) of ≤45 mL/min/1.73m\^2
• Clinical evidence of systemic oxalosis
• History of kidney or liver transplant

Sponsors & Collaborators

• Alnylam Pharmaceuticals: lead

Study Sites (9)

Clinical Trial Site

Rochester, Minnesota, 55905, United States

Location

Clinical Trial Site

Houston, Texas, 77030, United States

Location

Clinical Trial Site

Lyon, France

Location

Clinical Trial Site

Paris, France

Location

Clinical Trial Site

Bonn, Germany

Location

Clinical Trial Site

Haifa, Israel

Location

Clinical Trial Site

Jerusalem, Israel

Location

Clinical Trial Site

Nahariya, Israel

Location

Clinical Trial Site

London, United Kingdom

Location

Related Publications (3)

• Frishberg Y, Hayes W, Shasha-Lavsky H, Sas DJ, Michael M, Sellier-Leclerc AL, Hogan J, Willey R, Gansner JM, Magen D. Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial. Front Pediatr. 2024 Sep 16;12:1392644. doi: 10.3389/fped.2024.1392644. eCollection 2024.

  PMID: 39355649 DERIVED

• Hayes W, Sas DJ, Magen D, Shasha-Lavsky H, Michael M, Sellier-Leclerc AL, Hogan J, Ngo T, Sweetser MT, Gansner JM, McGregor TL, Frishberg Y. Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial. Pediatr Nephrol. 2023 Apr;38(4):1075-1086. doi: 10.1007/s00467-022-05684-1. Epub 2022 Aug 1.

  PMID: 35913563 DERIVED

• Sas DJ, Magen D, Hayes W, Shasha-Lavsky H, Michael M, Schulte I, Sellier-Leclerc AL, Lu J, Seddighzadeh A, Habtemariam B, McGregor TL, Fujita KP, Frishberg Y; ILLUMINATE-B Workgroup. Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children. Genet Med. 2022 Mar;24(3):654-662. doi: 10.1016/j.gim.2021.10.024. Epub 2021 Dec 8.

  PMID: 34906487 DERIVED

MeSH Terms

Conditions

Hyperoxaluria, Primary; Primary hyperoxaluria type 1; Hyperoxaluria

Interventions

lumasiran

Condition Hierarchy (Ancestors)

Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional 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

Study Record Dates

• First Submitted: April 1, 2019
• First Posted: April 5, 2019
• Study Start: April 22, 2019
• Primary Completion: June 29, 2020
• Study Completion: July 26, 2024
• Last Updated: February 14, 2025
• Results First Posted: July 19, 2021

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share

Locations

US (2); DE (1); GB (1); FR (2); IL (3)