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Completed

NCT Number: NCT03681184

A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).

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Key information

Conditions

Primary Hyperoxaluria Type 1 (PH1) Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Hyperoxaluria Hyperoxaluria, Primary Kidney Diseases Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Primary hyperoxaluria type 1 Urogenital Diseases Urologic Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Clinical Trial Site, Lyon, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Willing to provide written informed consent or assent and to comply with study requirements
  • Confirmation of PH1 disease
  • Meet the 24 hour urine oxalate excretion requirements
  • If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days

Exclusion criteria

  • Clinically significant health concerns (with the exception of PH1) or clinical evidence of extrarenal systemic oxalosis
  • Clinically significant abnormal laboratory results
  • Known active or evidence of HIV or hepatitis B or C infection
  • An estimated GFR of < 30 mL/min/1.73m^2 at screening
  • Received an investigational agent within 30 days or 5 half-lives before the first dose of study drug or are in follow-up of another clinical study
  • History of kidney or liver transplant
  • Known history of multiple drug allergies or allergic reaction to an oligonucleotide or GalNAc
  • History of intolerance to subcutaneous injection
  • Women who are pregnant, planning a pregnancy, or breast-feeding or those of child bearing potential and not willing to use contraception
  • History of alcohol abuse within the last 12 months, or unable or unwilling to limit alcohol consumption throughout the study

Treatment and study plan

Placebo

Drug

Placebo by SC injection

Lumasiran

Drug

Lumasiran by SC injection

Other names: ALN-GO1

Primary outcomes

  1. Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6

    Time frame: Baseline to Month 6

    Percent change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average percent change from baseline across Months 3 through 6. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.

Secondary outcomes

  1. Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6

    Time frame: Baseline to Month 6

    Absolute change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average absolute change from baseline across Months 3 through 6. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.

  2. Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6

    Time frame: Baseline to Month 6

    Percent change in 24-hour urinary oxalate:creatine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.

  3. Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6

    Time frame: Month 6

    The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m^2 for 24-hour urinary oxalate excretion corrected for BSA.

  4. Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6

    Time frame: Month 6

    The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m^2 for 24-hour urinary oxalate excretion corrected for BSA.

  5. Percentage Change in Plasma Oxalate From Baseline to Month 6

    Time frame: 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.

  6. Absolute Change in Plasma Oxalate From Baseline to Month 6

    Time frame: Baseline to Month 6

    Absolute 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.

  7. Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Week 2 and Months 1, 2, 3, 4, 5 and 6

    Time frame: Baseline, Week 2, Months 1, 2, 3, 4, 5 and 6

    eGFR is calculated from serum creatinine based on the Modification of Diet in Renal Disease formula for patients ≥18 years of age and the Schwartz Bedside Formula for patients >1 year to <18 years of age at screening. Change from baseline to Month 6 is reported.

  8. Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for BSA From Baseline in the Extension Period

    Time frame: From Baseline to Month 54 and Month 60

    Absolute change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average absolute change from baseline in the extension periods. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.

  9. Percentage Change in 24-hour Urinary Oxalate Excretion Corrected by BSA From Baseline in the Extension Period

    Time frame: From Baseline to Month 54 and Month 60

    Percent change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average percent change from baseline in the extension periods. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.

  10. Percentage of Time That 24-hour Urinary Oxalate is at or Below 1.5 × ULN During Lumasiran Treatment

    Time frame: Up to Month 60

    The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m^2 for 24-hour urinary oxalate excretion corrected for BSA.

  11. Absolute Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline in the Extension Period

    Time frame: From Baseline to Month 54 and Month 60

    Absolute change in 24-hour urinary oxalate:creatinine ratio was estimated by an average absolute change from baseline to the end of the OLE period at Month 54 and Month 60. A negative change from Baseline indicates a favorable outcome.

  12. Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline in the Extension Period

    Time frame: From Baseline to Month 54 and Month 60

    eGFR is calculated from serum creatinine based on the Modification of Diet in Renal Disease formula for patients ≥18 years of age and the Schwartz Bedside Formula for patients >1 year to <18 years of age at screening.

  13. Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: DB Period (Placebo): From first dose of study drug (Day 1) up to Month 6; Placebo/Lumasiran: From first dose of lumasiran (Month 6) up to end of study (Month 60); Lumasiran/Lumasiran: From first dose of lumasiran (Day 1) up to end of study (Month 60).

    An AE is any untoward medical occurrence in a clinical investigational participant administered a medicinal product & which does not necessarily have a causal relationship with this treatment. SAE is any untoward medical occurrence that at any dose results in death, is life-threatening, requires in-patient hospitalization/prolongation of existing hospitalization, results in persistent/significant disability or incapacity, is a congenital anomaly or birth defect, is an important medical event that may not be immediately life-threatening/result in death/hospitalization but may jeopardize the participant & may require intervention to prevent one of the other outcomes listed above. All Lumasiran Treated Set: All participants who received any amount of lumasiran including participants who took lumasiran during 6-month double-blinded period & participants who initially took placebo during the 6-month double-blinded period and then switched to lumasiran during the extension period.

Other outcomes

  1. Rate of Renal Stone Events

    Time frame: 12-Month Period prior to Informed Consent, 6-Month DB Period

    A renal stone event is defined as a patient-reported event that includes ≥1 of the following: visit to healthcare provider because of a renal stone; medication for renal colic; stone passage; macroscopic hematuria due to a renal stone. Lower rates indicate a favorable outcome.

  2. Change From Baseline in Nephrocalcinosis as Assessed by Renal Ultrasound

    Time frame: Baseline, Month 6

    Renal ultrasound data were used to grade medullary nephrocalcinosis findings (range: 0 to 3), where a higher grade indicates greater severity. Improving=if both sides improve, or one side improves and the other side has no change; No change=if both sides have no change; Worsening=if both sides worsen, or one side worsens and the other side has no change; Indeterminate=if one side improves and the other side worsens.

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

Acronym: ILLUMINATE-A

Important dates

Study start
2018
Primary completion
2019
Study completion
2024
First posted
Sep 21, 2018
Registry last updated
Aug 12, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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