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A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (ILLUMINATE-C)

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ClinicalTrials.gov Identifier: NCT04152200
Recruitment Status : Not yet recruiting
First Posted : November 5, 2019
Last Update Posted : November 5, 2019
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE October 31, 2019
First Posted Date  ICMJE November 5, 2019
Last Update Posted Date November 5, 2019
Estimated Study Start Date  ICMJE November 2019
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 4, 2019)
  • Percent Change in Plasma Oxalate from Baseline to Month 6 [ Time Frame: Baseline, Month 6 ]
  • Percent Change in Pre-dialysis Plasma Oxalate from Baseline to Month 6 [ Time Frame: Baseline, Month 6 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: November 4, 2019)
  • Percent Change in Plasma Oxalate Area Under the Curve (AUC) Between Dialysis Sessions from Baseline to Month 6 and Month 60 [ Time Frame: Baseline, Month 6; Baseline, Month 60 ]
  • Absolute Change in Plasma Oxalate from Baseline to Month 6 and Month 60 [ Time Frame: Baseline, Month 6; Baseline, Month 60 ]
  • Change in Urinary Oxalate from Baseline to Month 6 and Month 60 [ Time Frame: Baseline, Month 6; Baseline, Month 60 ]
  • Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score from Baseline to Month 6 and Month 60 [ Time Frame: Baseline, Month 6; Baseline, 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).
  • Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) from Baseline to Month 6 and Month 60 [ Time Frame: Baseline, Month 6; Baseline, Month 60 ]
    KDQOL-36 is a kidney disease-specific measure of HRQoL. Score range (per domain): 0 - 100; higher scores indicate better HRQoL.
  • Maximum Plasma Concentration (Cmax) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  • Time to Maximum Plasma Concentration (tmax) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  • Elimination Half-life (t½β) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  • Area Under the Concentration-time Curve (AUC) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  • Apparent Clearance (CL/F) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  • Apparent Volume of Distribution (V/F) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  • Percent Change in Plasma Oxalate from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
  • Change in Nephrocalcinosis from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
    Nephrocalcinosis will be assessed by renal ultrasound.
  • Change in Frequency of Dialysis from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
  • Change in Mode of Dialysis from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
    Modes of dialysis are defined as hemodialysis and peritoneal dialysis.
  • Change in Frequency of Renal Stone Events from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
  • Change in Renal Function Assessed by Estimated Glomerular Filtration Rate (eGFR) from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
  • Change in Measures of Systemic Oxalosis from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
    Systemic oxalosis will be assessed in cardiac, skeletal, ophthalmologic, and dermatologic systems.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
Official Title  ICMJE ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1)
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Primary Hyperoxaluria Type 1
  • Primary Hyperoxaluria
Intervention  ICMJE Drug: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
Other Name: ALN-GO1
Study Arms  ICMJE Experimental: Lumasiran
All participants will receive open-label lumasiran.
Intervention: Drug: Lumasiran
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: November 4, 2019)
16
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2025
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Has documented diagnosis of primary hyperoxaluria type 1 (PH1)
  • Estimated glomerular filtration rate (eGRF) ≤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

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526 clinicaltrials@alnylam.com
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04152200
Other Study ID Numbers  ICMJE ALN-GO1-005
2019-001346-17 ( 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  ICMJE
Plan to Share IPD: No
Responsible Party Alnylam Pharmaceuticals
Study Sponsor  ICMJE Alnylam Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Tracy McGregor, MD Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date November 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP