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

NCT03350451 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: ALN-GO1-0022016-003134-24
• Study Type: interventional
• Target: 20
• Locations: 5 countries
• Sites: 9

Brief Summary

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

Conditions

PH1; Primary Hyperoxaluria; RNAi Therapeutic; siRNA; AGT

Keywords

Hyperoxaluria, Primary; Hyperoxaluria; Kidney Diseases; Urologic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases

Outcome Measures

Primary Outcomes (1)

• Number of Participants With at Least One Adverse Event (AE)

  AE is any untoward medical occurrence in a participant or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Safety analysis set included all participants who received any amount of study drug.

  Baseline (Day -1) up to 54 months

Secondary Outcomes (3)

• Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 54 Months

  Baseline (Day -1) up to 54 months

• Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 54 Months

  Baseline (Day -1) up to 54 months

• Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 54 Months

  Baseline (Day -1) up to 54 months

Study Arms (2)

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

EXPERIMENTAL

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).

Drug: Lumasiran

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

EXPERIMENTAL

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.

Drug: Lumasiran

Interventions

Lumasiran DRUG

Multiple doses of lumasiran by SC injection

Also known as: ALN-GO1

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

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alnylam Pharmaceuticals: lead

Study Sites (9)

Clinical Trial Site

Bordeaux, France

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

Amsterdam, Netherlands

Location

Clinical Trial Site

Birmingham, United Kingdom

Location

Clinical Trial Site

London, United Kingdom

Location

MeSH Terms

Conditions

Hyperoxaluria, Primary; Hyperoxaluria; Kidney Diseases; Urologic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases

Interventions

lumasiran

Condition Hierarchy (Ancestors)

Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Carbohydrate Metabolism, Inborn Errors; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; 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 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2017
• First Posted: November 22, 2017
• Study Start: April 4, 2018
• Primary Completion: February 7, 2023
• Study Completion: February 7, 2023
• Last Updated: April 25, 2024
• Results First Posted: April 25, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will share

Locations

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