A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 (ILLUMINATE-B)

• ClinicalTrials.gov Identifier: NCT03905694
• Recruitment Status: Active, not recruiting
• First Posted: April 5, 2019
• Last Update Posted: February 5, 2021
• Sponsor: Alnylam Pharmaceuticals
• Information provided by (Responsible Party): Alnylam Pharmaceuticals

Tracking Information

• First Submitted Date: April 1, 2019
• First Posted Date: April 5, 2019
• Last Update Posted Date: February 5, 2021
• Actual Study Start Date: April 22, 2019
• Actual Primary Completion Date: June 29, 2020 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: April 4, 2019): Percentage Change in Urinary Oxalate Excretion from Baseline to Month 6 [ Time Frame: Up to 6 months ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: October 7, 2019)

• Percentage Change in Urinary Oxalate Excretion from Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
• Absolute Change in Urinary Oxalate Excretion from Baseline [ Time Frame: Up to 60 months ]
• Percentage of Time that Spot Urinary Oxalate:Creatinine Ratio ≤ Near-normalization Threshold (≤1.5 × ULN) [ Time Frame: Up to 60 months ]
• Percentage of Participants with Urinary Oxalate Excretion ≤ the Upper Limit of Normal (ULN) and ≤ 1.5 x ULN [ Time Frame: Up to 60 months ]
• Percentage Change in Plasma Oxalate from Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
• Absolute Change in Plasma Oxalate from Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
• Maximum Observed Plasma Concentration (Cmax) of Lumasiran [ Time Frame: Up to 24 months ]
• Time to Maximum Observed Plasma Concentration (tmax) of Lumasiran [ Time Frame: Up to 24 months ]
• Elimination Half-life (t1/2beta) of Lumasiran [ Time Frame: Up to 24 months ]
• Area Under the Concentration-time Curve (AUC) of Lumasiran [ Time Frame: Up to 24 months ]
• Apparent Clearance (CL/F) of Lumasiran [ Time Frame: Up to 24 months ]
• Apparent Volume of Distribution (V/F) of Lumasiran [ Time Frame: Up to 24 months ]
• Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline [ Time Frame: Up to 60 months ]
• Frequency of Adverse Events (AEs) [ Time Frame: Up to 60 months ]

Original Secondary Outcome Measures (submitted: April 4, 2019)

• Percentage Change in Urinary Oxalate Excretion from Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
• Absolute Change in Urinary Oxalate Excretion from Baseline [ Time Frame: Up to 60 months ]
• Percentage of Participants with Urinary Oxalate Excretion ≤ the Upper Limit of Normal (ULN) and ≤ 1.5 x ULN [ Time Frame: Up to 60 months ]
• Maximum Observed Plasma Concentration (Cmax) of Lumasiran [ Time Frame: Up to 24 months ]
• Time to Maximum Observed Plasma Concentration (tmax) of Lumasiran [ Time Frame: Up to 24 months ]
• Elimination Half-life (t1/2beta) of Lumasiran [ Time Frame: Up to 24 months ]
• Area Under the Concentration-time Curve (AUC) of Lumasiran [ Time Frame: Up to 24 months ]
• Apparent Clearance (CL/F) of Lumasiran [ Time Frame: Up to 24 months ]
• Apparent Volume of Distribution (V/F) of Lumasiran [ Time Frame: Up to 24 months ]
• Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline [ Time Frame: Up to 60 months ]
• Frequency of Adverse Events (AEs) [ Time Frame: Up to 60 months ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
• Official Title: ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
• Brief Summary: The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Primary Hyperoxaluria
• Primary Hyperoxaluria Type 1 (PH1)

Intervention

Drug: Lumasiran

Lumasiran will be administered by subcutaneous (SC) injection.

Other Name: ALN-GO1

Study Arms

Experimental: Lumasiran

Participants will receive lumasiran during the study.

Intervention: Drug: Lumasiran

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: February 14, 2020): 18
• Original Estimated Enrollment (submitted: April 4, 2019): 8
• Estimated Study Completion Date: August 19, 2024
• Actual Primary Completion Date: June 29, 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Has confirmation of primary hyperoxaluria type 1 (PH1)
• Meets urinary oxalate excretion requirements
• If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days

Exclusion Criteria:

• Abnormal serum creatinine levels at screening for infants who are less than 1 year old
• Does not have relatively preserved kidney function
• Clinical evidence of systemic oxalosis
• History of kidney or liver transplant

Sex/Gender

• Sexes Eligible for Study: All

• Ages: up to 5 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: France, Germany, Israel, United Kingdom, United States

Administrative Information

• NCT Number: NCT03905694
• Other Study ID Numbers: ALN-GO1-004; 2018-004014-17 ( EudraCT Number )
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Alnylam Pharmaceuticals
• Study Sponsor: Alnylam Pharmaceuticals
• Collaborators: Not Provided

Investigators

• Study Director: Medical Director; Alnylam Pharmaceuticals

• PRS Account: Alnylam Pharmaceuticals
• Verification Date: February 2021