ILLUMINATE-A: a randomized, double-blind, placebo-controlled phase III study with extended treatment duration to assess the efficacy and safety of Lumasiran in children and adults with primary hyperoxaluria type 1
Résumé de l'étude
The aim of this study is to investigate whether the investigational drug Lumasiran can reduce oxalate production in the liver and to assess the safety of Lumasiran and the body's response to the treatment. Approximately 30 patients will participate in this study, which will be conducted at about 20 participating trial centers worldwide. For the study, patients will be randomly assigned to receive either the investigational drug Lumasiran or placebo during the first 6 months. The probability of receiving placebo in the first 6 months is 33%, and the probability of receiving Lumasiran during the first 6 months is 67%. After month 9 of the study, an open-label treatment phase begins, during which all participants will receive Lumasiran every 3 months until the end of the study.
(BASEC)
Intervention étudiée
Patients suffer from primary hyperoxaluria type 1. Primary hyperoxaluria type 1 (PH1) is a rare, inherited disorder that causes excessive production of a substance called oxalate in the liver. The excess oxalate combines with calcium to form a hard substance that can accumulate in the kidneys and cause kidney stones, and may eventually lead to kidney failure. In severe cases, it can also accumulate in other parts of the body and damage other organs.
(BASEC)
Maladie en cours d'investigation
primary hyperoxaluria type 1
(BASEC)
1. Age 18 years or older (in Switzerland). 2. Diagnosis of primary hyperoxaluria type 1 (PH1) confirmed by genetic testing. 3. Participants must have a certain amount of oxalate in the urine (≥0.7 mmol/24h/1.73m2), measured by collecting urine over a 24-hour period. 4. If vitamin B6 (pyridoxine) is taken for the treatment of PH1, participants must have taken a stable dose (in the last 90 days) and be able to continue taking it for the next year. 5. Participants must be able to read and understand the consent form and agree to participate in the study procedures. (BASEC)
Critères d'exclusion
1. Insufficient kidney function (GFR of ≤45 mL/min/1.73m2). 2. Patients with a kidney or liver transplant, 3. Oxalate deposits outside the kidneys, 4. Infection with HIV (human immunodeficiency virus), hepatitis C virus (HCV), or hepatitis B virus, 5. Patients who are not willing or able to limit their daily alcohol intake during the study, e.g., to one glass of wine [approximately 125 ml] or beer [approximately 284 ml], or who have had alcohol abuse in the twelve months prior to screening, 5. Women who are pregnant, wish to become pregnant, or are breastfeeding. (BASEC)
Lieu de l’étude
Bâle
(BASEC)
Sponsor
non disponible
Contact pour plus d'informations sur l'étude
Personne de contact en Suisse
Prof. Dr. med. Daniel Fuster
+41-31-632-3144
Daniel.Fuster@clutterinsel.ch(BASEC)
Informations générales
Alnylam Pharmaceuticals
(ICTRP)
Informations scientifiques
Alnylam Pharmaceuticals
(ICTRP)
Nom du comité d'éthique approbateur (pour les études multicentriques, uniquement le comité principal)
Commission cantonale d'éthique de Berne
(BASEC)
Date d'approbation du comité d'éthique
01.04.2019
(BASEC)
Identifiant de l'essai ICTRP
NCT03681184 (ICTRP)
Titre officiel (approuvé par le comité d'éthique)
non disponible
Titre académique
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 (ICTRP)
Titre public
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 (ICTRP)
Maladie en cours d'investigation
Primary Hyperoxaluria Type 1 (PH1) (ICTRP)
Intervention étudiée
Drug: Placebo;Drug: Lumasiran (ICTRP)
Type d'essai
Interventional (ICTRP)
Plan de l'étude
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor). (ICTRP)
Critères d'inclusion/exclusion
Gender: All
Maximum age: N/A
Minimum age: 6 Years
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 (ICTRP)
non disponible
Critères d'évaluation principaux et secondaires
Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 (ICTRP)
Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6;Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6;Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6;Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6;Percentage Change in Plasma Oxalate From Baseline to Month 6;Absolute Change in Plasma Oxalate From Baseline to Month 6;Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Week 2 and Months 1, 2, 3, 4, 5 and 6;Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for BSA From Baseline in the Extension Period;Percentage Change in 24-hour Urinary Oxalate Excretion Corrected by BSA From Baseline in the Extension Period;Percentage of Time That 24-hour Urinary Oxalate is at or Below 1.5 ? ULN During Lumasiran Treatment;Absolute Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline in the Extension Period;Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline in the Extension Period;Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) (ICTRP)
Date d'enregistrement
non disponible
Inclusion du premier participant
non disponible
Sponsors secondaires
non disponible
Contacts supplémentaires
Medical Director, Alnylam Pharmaceuticals (ICTRP)
ID secondaires
2018-001981-40, ALN-GO1-003 (ICTRP)
Résultats-Données individuelles des participants
non disponible
Informations complémentaires sur l'essai
https://clinicaltrials.gov/ct2/show/NCT03681184 (ICTRP)
Résultats de l'essai
Résumé des résultats
non disponible
Lien vers les résultats dans le registre primaire
non disponible