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
Zusammenfassung der Studie
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)
Untersuchte Intervention
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)
Untersuchte Krankheit(en)
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)
Ausschlusskriterien
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)
Studienstandort
Basel
(BASEC)
Sponsor
nicht verfügbar
Kontakt für weitere Auskünfte zur Studie
Kontaktperson Schweiz
Prof. Dr. med. Daniel Fuster
+41-31-632-3144
Daniel.Fuster@clutterinsel.ch(BASEC)
Allgemeine Auskünfte
Alnylam Pharmaceuticals
(ICTRP)
Wissenschaftliche Auskünfte
Alnylam Pharmaceuticals
(ICTRP)
Name der bewilligenden Ethikkommission (bei multizentrischen Studien nur die Leitkommission)
Ethikkommission Bern
(BASEC)
Datum der Bewilligung durch die Ethikkommission
01.04.2019
(BASEC)
ICTRP Studien-ID
NCT03681184 (ICTRP)
Offizieller Titel (Genehmigt von der Ethikkommission)
nicht verfügbar
Wissenschaftlicher Titel
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)
Öffentlicher Titel
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 (ICTRP)
Untersuchte Krankheit(en)
Primary Hyperoxaluria Type 1 (PH1) (ICTRP)
Untersuchte Intervention
Drug: Placebo;Drug: Lumasiran (ICTRP)
Studientyp
Interventional (ICTRP)
Studiendesign
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor). (ICTRP)
Ein-/Ausschlusskriterien
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)
nicht verfügbar
Primäre und sekundäre Endpunkte
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)
Registrierungsdatum
nicht verfügbar
Einschluss des ersten Teilnehmers
nicht verfügbar
Sekundäre Sponsoren
nicht verfügbar
Weitere Kontakte
Medical Director, Alnylam Pharmaceuticals (ICTRP)
Sekundäre IDs
2018-001981-40, ALN-GO1-003 (ICTRP)
Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten
nicht verfügbar
Weitere Informationen zur Studie
https://clinicaltrials.gov/ct2/show/NCT03681184 (ICTRP)
Ergebnisse der Studie
Zusammenfassung der Ergebnisse
nicht verfügbar
Link zu den Ergebnissen im Primärregister
nicht verfügbar