Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1 Level: PT Classification code 10020703 Term: Hyperoxaluria System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Interventions
Product Name: lumasiran
Product Code: ALN-GO1
Pharmaceutical Form: Solution for injection
INN or Proposed INN: LUMASIRAN
Current Sponsor
Sponsors
Alnylam Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Have reached at least 37 weeks estimated gestational age (full-term infant) but less than 6 years of age at consent. 2.Documentation of PH1 as determined by genetic analysis prior to initial dosing. 3.Urinary oxalate:creatinine ratio greater than the upper limit of normal based on age on at least 2 of 3 single-void collections during screening. 4.If taking therapeutic vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days before screening, and is able to remain on this stable regimen until at least the Month 6 visit. Dose adjustments for interval weight gain are acceptable. 5.Legal guardian(s) is (are) willing and able to comply with the study requirements and to provide written informed consent; the patient should provide assent per local and national requirements. Are the trial subjects under 18? yes Number of subjects for this age range: 20 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1.Has any of the following laboratory parameter assessments: a.Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2× ULN for age b.Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert’s syndrome are eligible if the total bilirubin is <2× ULN 2.Has known active human immunodeficiency virus (HIV) infection; or evidence of current or chronic hepatitis C virus (HCV) or hepatitis B virus (HBV) infection. 3.If =12 months old at screening, has an estimated glomerular filtration (GFR) of =45 mL/min/1.73m2 (calculation will be based on the Schwartz Bedside Formula); if <12 months old at screening, has serum creatinine value per the central laboratory above the ULN for age. 4.Received an investigational agent within the last 30 days or 5 half-lives, whichever is longer, prior to the first dose of study drug, or are in follow-up of another clinical study prior to randomization. Consultation with Medical Monitor required if treated with unapproved products prior to consent. 5.Medical history includes clinical evidence of extrarenal systemic oxalosis as determined by the Investigator. 6.Has undergone renal or liver transplantation or a liver transplant is anticipated in the 6 months after the initial dose of lumasiran. 7.Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation. 8.History of allergic reaction to an oligonucleotide or GalNAc. 9.History of intolerance to subcutaneous (SC) injection(s). 10.For female patients who may achieve menarche during the study, is not willing to comply with the contraceptive requirements during the study period
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluate the effect of lumasiran on urinary oxalate excretion;Primary end point(s): Percent change in urinary oxalate excretion from baseline to Month 6;Timepoint(s) of evaluation of this end point: Urine samples will be collected from screening through Month 6 as specified in the protocol; Secondary Objective: Extension Phase (Month 6 to End of Study) •Evaluate the long-term effects of lumasiran on urinary oxalate Duration of Study •Evaluate the effects of lumasiran on additional measures of urinary oxalate •Evaluate the effects of lumasiran on plasma oxalate •Characterize the pharmacokinetics (PK) of lumasiran •Evaluate the effect of lumasiran on renal function | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Extension Phase (Month 6 to End of Study) •Percent change in urinary oxalate excretion from baseline •Percentage of time that spot urinary oxalate:creatinine ratio is at or below the near-normalization threshold (=1.5 x ULN) change Duration of Study •Absolute change in urinary oxalate excretion from baseline •Proportion of patients with urinary oxalate excretion = the upper limit of normal (ULN) and = 1.5 x ULN •Change (percent and absolute) in plasma oxalate from baseline •Plasma PK parameters of lumasiran •Change from baseline in estimated glomerular filtration rate (eGFR) ;Timepoint(s) of evaluation of this end point: Efficacy assessment will be conducted from screening through Month 60 as specified in the protocol | — |
Countries
France, Germany, Israel, United Kingdom, United States
Contacts
Public ContactClinical Trial Hotline
Alnylam Pharmaceuticals, Inc.
Outcome results
None listed