Clinical trial

ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

Name

ALN-GO1-004

Description

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).

Trial arms

Trial start

2019-04-22

Estimated PCD

2020-06-29

Trial end

2024-08-19

Status

Active (not recruiting)

Phase

Early phase I

Treatment

Lumasiran

Lumasiran will be administered by subcutaneous (SC) injection.

Arms:

Lumasiran

Other names:

ALN-GO1

Size

Primary endpoint

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

Baseline to Month 6

Eligibility criteria

Inclusion Criteria: * 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 Exclusion Criteria: * 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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 18, 'type': 'ACTUAL'}}

Updated at

2024-04-22

1 organization

1 product

2 indications

Product

Lumasiran

Indication

Primary Hyperoxaluria

Indication

Primary Hyperoxaluria Type 1

Organization

Alnylam Pharmaceuticals