Clinical trial

ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1)

Name

ALN-GO1-005

Description

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 arms

Trial start

2020-01-21

Estimated PCD

2021-05-18

Trial end

2025-07-01

Status

Active (not recruiting)

Phase

Early phase I

Treatment

Lumasiran

Lumasiran will be administered by subcutaneous (SC) injection.

Arms:

Lumasiran

Other names:

ALN-GO1, OXLUMO

Size

Primary endpoint

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

Baseline to Month 6

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

Baseline to Month 6

Eligibility criteria

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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 21, 'type': 'ACTUAL'}}

Updated at

2024-04-22

1 organization

1 product

2 indications

Product

Lumasiran

Indication

Primary Hyperoxaluria Type 1

Indication

Primary Hyperoxaluria

Organization

Alnylam Pharmaceuticals