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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04152200




Registration number
NCT04152200
Ethics application status
Date submitted
31/10/2019
Date registered
5/11/2019

Titles & IDs
Public title
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
Scientific title
ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1)
Secondary ID [1] 0 0
2019-001346-17
Secondary ID [2] 0 0
ALN-GO1-005
Universal Trial Number (UTN)
Trial acronym
ILLUMINATE-C
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Hyperoxaluria Type 1 0 0
Primary Hyperoxaluria 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - Lumasiran

Experimental: Lumasiran - All patients will receive open-label lumasiran.


Treatment: Drugs: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6
Timepoint [1] 0 0
Baseline to Month 6
Primary outcome [2] 0 0
Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6
Timepoint [2] 0 0
Baseline to Month 6
Secondary outcome [1] 0 0
Cohort B: Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 6
Timepoint [1] 0 0
Baseline to Month 6
Secondary outcome [2] 0 0
Absolute Change in Plasma Oxalate From Baseline to Month 6
Timepoint [2] 0 0
Baseline to Month 6
Secondary outcome [3] 0 0
Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6
Timepoint [3] 0 0
Baseline to Month 6
Secondary outcome [4] 0 0
Cohort A: Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6
Timepoint [4] 0 0
Baseline to Month 6
Secondary outcome [5] 0 0
Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
Timepoint [5] 0 0
Baseline to Month 6
Secondary outcome [6] 0 0
Cohort A: Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
Timepoint [6] 0 0
Baseline to Month 6
Secondary outcome [7] 0 0
Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 6 in Patients >=2 to 18 Years of Age at Time of Informed Consent
Timepoint [7] 0 0
Baseline to Month 6
Secondary outcome [8] 0 0
Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 6 in Patients =18 Years of Age at Time of Informed Consent
Timepoint [8] 0 0
Baseline to Month 6
Secondary outcome [9] 0 0
Maximum Plasma Concentration (Cmax) of Lumasiran
Timepoint [9] 0 0
Day 1; Month 6
Secondary outcome [10] 0 0
Time to Maximum Plasma Concentration (Tmax) of Lumasiran
Timepoint [10] 0 0
Day 1; Month 6
Secondary outcome [11] 0 0
Elimination Half-life (t½ß) of Lumasiran
Timepoint [11] 0 0
Day 1; Month 6
Secondary outcome [12] 0 0
Area Under the Concentration-time Curve From 0 to 24 Hours [AUC(0-24)] for Lumasiran
Timepoint [12] 0 0
Day 1; Month 6
Secondary outcome [13] 0 0
Apparent Clearance (CL/F) of Lumasiran
Timepoint [13] 0 0
Day 1; Month 6
Secondary outcome [14] 0 0
Apparent Volume of Distribution (V/F) of Lumasiran
Timepoint [14] 0 0
Day 1; Month 6
Secondary outcome [15] 0 0
Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 60
Timepoint [15] 0 0
Baseline to Month 60
Secondary outcome [16] 0 0
Absolute Change in Plasma Oxalate From Baseline to Month 60
Timepoint [16] 0 0
Baseline to Month 60
Secondary outcome [17] 0 0
Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 60
Timepoint [17] 0 0
Baseline to Month 60
Secondary outcome [18] 0 0
Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 60
Timepoint [18] 0 0
Baseline to Month 60
Secondary outcome [19] 0 0
Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60
Timepoint [19] 0 0
Baseline to Month 60
Secondary outcome [20] 0 0
Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60
Timepoint [20] 0 0
Baseline to Month 60
Secondary outcome [21] 0 0
Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 60 in Patients >=2 to 18 Years of Age at Time of Informed Consent
Timepoint [21] 0 0
Baseline to Month 60
Secondary outcome [22] 0 0
Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 60 in Patients =18 Years of Age at Time of Informed Consent
Timepoint [22] 0 0
Baseline to Month 60
Secondary outcome [23] 0 0
Percent Change in Plasma Oxalate From Baseline to End of Study
Timepoint [23] 0 0
Baseline to Month 60
Secondary outcome [24] 0 0
Change in Nephrocalcinosis From Baseline to End of Study
Timepoint [24] 0 0
Baseline to Month 60
Secondary outcome [25] 0 0
Change in Frequency of Dialysis From Baseline to End of Study
Timepoint [25] 0 0
Baseline to Month 60
Secondary outcome [26] 0 0
Change in Mode of Dialysis From Baseline to End of Study
Timepoint [26] 0 0
Baseline to Month 60
Secondary outcome [27] 0 0
Change in Frequency of Renal Stone Events From Baseline to End of Study
Timepoint [27] 0 0
Baseline to Month 60
Secondary outcome [28] 0 0
Change in Renal Function Assessed by Estimated Glomerular Filtration Rate (eGFR) From Baseline to End of Study
Timepoint [28] 0 0
Baseline to Month 60
Secondary outcome [29] 0 0
Change in Measures of Systemic Oxalosis From Baseline to End of Study
Timepoint [29] 0 0
Baseline to Month 60

Eligibility
Key inclusion criteria
* Has documented diagnosis of primary hyperoxaluria type 1 (PH1)
* Estimated glomerular filtration rate (eGFR) =45 mL/min/1.73 m^2 for patients =12 months of age (<12 months of age, must have serum creatinine considered elevated for age)
* Meets plasma oxalate level requirements
* If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
* If on dialysis, may be on hemodialysis therapy only and must have been on a stable regimen for at least 4 weeks
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Hemodialysis/peritoneal dialysis combination therapy or peritoneal dialysis alone
* Diagnosis of conditions other than PH1 contributing to renal insufficiency
* History of liver transplant
* History of kidney transplant and currently receiving immunosuppressants

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/01/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2025
Actual
Sample size
Target
Accrual to date
Final
21
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Garran
Recruitment postcode(s) [1] 0 0
- Garran
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Minnesota
Country [2] 0 0
United States of America
State/province [2] 0 0
Texas
Country [3] 0 0
Belgium
State/province [3] 0 0
Brussels
Country [4] 0 0
France
State/province [4] 0 0
Bron
Country [5] 0 0
France
State/province [5] 0 0
Lyon
Country [6] 0 0
Israel
State/province [6] 0 0
Haifa
Country [7] 0 0
Israel
State/province [7] 0 0
Nahariya
Country [8] 0 0
Italy
State/province [8] 0 0
Rome
Country [9] 0 0
Jordan
State/province [9] 0 0
Irbid
Country [10] 0 0
Lebanon
State/province [10] 0 0
Beirut
Country [11] 0 0
Netherlands
State/province [11] 0 0
Amsterdam
Country [12] 0 0
Turkey
State/province [12] 0 0
Yenimahalle
Country [13] 0 0
United Arab Emirates
State/province [13] 0 0
Dubai

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alnylam Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Alnylam Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

TypeOther DetailsAttachment
Study protocol
Statistical analysis plan



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04152200