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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04152200
Ethics application status
Date submitted
31/10/2019
Date registered
5/11/2019
Date last updated
19/04/2021

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 participants 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
Percent Change in Plasma Oxalate from Baseline to Month 6
Timepoint [1] 0 0
Baseline, Month 6
Primary outcome [2] 0 0
Percent Change in Pre-dialysis Plasma Oxalate from Baseline to Month 6
Timepoint [2] 0 0
Baseline, Month 6
Secondary outcome [1] 0 0
Percent Change in Plasma Oxalate Area Under the Curve (AUC) Between Dialysis Sessions from Baseline to Month 6 and Month 60
Timepoint [1] 0 0
Baseline, Month 6; Baseline, Month 60
Secondary outcome [2] 0 0
Absolute Change in Plasma Oxalate from Baseline to Month 6 and Month 60
Timepoint [2] 0 0
Baseline, Month 6; Baseline, Month 60
Secondary outcome [3] 0 0
Change in Urinary Oxalate from Baseline to Month 6 and Month 60
Timepoint [3] 0 0
Baseline, Month 6; Baseline, Month 60
Secondary outcome [4] 0 0
Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score from Baseline to Month 6 and Month 60 - Measures core dimensions of health plus role (school) functioning in children. Total score range: 0 - 100; higher scores indicate better health-related quality of life (HRQoL).
Timepoint [4] 0 0
Baseline, Month 6; Baseline, Month 60
Secondary outcome [5] 0 0
Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) from Baseline to Month 6 and Month 60 - KDQOL-36 is a kidney disease-specific measure of HRQoL. Score range (per domain): 0 - 100; higher scores indicate better HRQoL.
Timepoint [5] 0 0
Baseline, Month 6; Baseline, Month 60
Secondary outcome [6] 0 0
Maximum Plasma Concentration (Cmax) of Lumasiran
Timepoint [6] 0 0
Baseline, Month 6
Secondary outcome [7] 0 0
Time to Maximum Plasma Concentration (tmax) of Lumasiran
Timepoint [7] 0 0
Baseline, Month 6
Secondary outcome [8] 0 0
Elimination Half-life (t½ß) of Lumasiran
Timepoint [8] 0 0
Baseline, Month 6
Secondary outcome [9] 0 0
Area Under the Concentration-time Curve (AUC) of Lumasiran
Timepoint [9] 0 0
Baseline, Month 6
Secondary outcome [10] 0 0
Apparent Clearance (CL/F) of Lumasiran
Timepoint [10] 0 0
Baseline, Month 6
Secondary outcome [11] 0 0
Apparent Volume of Distribution (V/F) of Lumasiran
Timepoint [11] 0 0
Baseline, Month 6
Secondary outcome [12] 0 0
Percent Change in Plasma Oxalate from Baseline to End of Study
Timepoint [12] 0 0
Baseline, Month 60
Secondary outcome [13] 0 0
Change in Nephrocalcinosis from Baseline to End of Study - Nephrocalcinosis will be assessed by renal ultrasound.
Timepoint [13] 0 0
Baseline, Month 60
Secondary outcome [14] 0 0
Change in Frequency of Dialysis from Baseline to End of Study
Timepoint [14] 0 0
Baseline, Month 60
Secondary outcome [15] 0 0
Change in Mode of Dialysis from Baseline to End of Study - Modes of dialysis are defined as hemodialysis and peritoneal dialysis.
Timepoint [15] 0 0
Baseline, Month 60
Secondary outcome [16] 0 0
Change in Frequency of Renal Stone Events from Baseline to End of Study
Timepoint [16] 0 0
Baseline, Month 60
Secondary outcome [17] 0 0
Change in Renal Function Assessed by Estimated Glomerular Filtration Rate (eGFR) from Baseline to End of Study
Timepoint [17] 0 0
Baseline, Month 60
Secondary outcome [18] 0 0
Change in Measures of Systemic Oxalosis from Baseline to End of Study - Systemic oxalosis will be assessed in cardiac, skeletal, ophthalmologic, and dermatologic systems.
Timepoint [18] 0 0
Baseline, 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
Gender
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
N/A
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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
New York
Country [3] 0 0
United States of America
State/province [3] 0 0
Texas
Country [4] 0 0
Belgium
State/province [4] 0 0
Brussels
Country [5] 0 0
France
State/province [5] 0 0
Lyon
Country [6] 0 0
France
State/province [6] 0 0
Paris
Country [7] 0 0
Israel
State/province [7] 0 0
Haifa
Country [8] 0 0
Israel
State/province [8] 0 0
Jerusalem
Country [9] 0 0
Israel
State/province [9] 0 0
Nahariya
Country [10] 0 0
Jordan
State/province [10] 0 0
Irbid
Country [11] 0 0
Lebanon
State/province [11] 0 0
Beirut
Country [12] 0 0
Netherlands
State/province [12] 0 0
Amsterdam
Country [13] 0 0
Switzerland
State/province [13] 0 0
Bern
Country [14] 0 0
Turkey
State/province [14] 0 0
Yenimahalle
Country [15] 0 0
United Arab Emirates
State/province [15] 0 0
Dubai
Country [16] 0 0
United Kingdom
State/province [16] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and
pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1
(PH1).
Trial website
https://clinicaltrials.gov/show/NCT04152200
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

Summary results
Other publications