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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

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).

Condition or disease Intervention/treatment Phase
Primary Hyperoxaluria Type 1 Primary Hyperoxaluria Drug: Lumasiran Phase 3

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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)
Estimated Study Start Date : November 2019
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : August 2025

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Lumasiran
All participants will receive open-label lumasiran.
Drug: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
Other Name: ALN-GO1




Primary Outcome Measures :
  1. Percent Change in Plasma Oxalate from Baseline to Month 6 [ Time Frame: Baseline, Month 6 ]
  2. Percent Change in Pre-dialysis Plasma Oxalate from Baseline to Month 6 [ Time Frame: Baseline, Month 6 ]

Secondary Outcome Measures :
  1. 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 ]
  2. Absolute Change in Plasma Oxalate from Baseline to Month 6 and Month 60 [ Time Frame: Baseline, Month 6; Baseline, Month 60 ]
  3. Change in Urinary Oxalate from Baseline to Month 6 and Month 60 [ Time Frame: Baseline, Month 6; Baseline, Month 60 ]
  4. 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).

  5. 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.

  6. Maximum Plasma Concentration (Cmax) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  7. Time to Maximum Plasma Concentration (tmax) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  8. Elimination Half-life (t½β) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  9. Area Under the Concentration-time Curve (AUC) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  10. Apparent Clearance (CL/F) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  11. Apparent Volume of Distribution (V/F) of Lumasiran [ Time Frame: Baseline, Month 6 ]
  12. Percent Change in Plasma Oxalate from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
  13. Change in Nephrocalcinosis from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
    Nephrocalcinosis will be assessed by renal ultrasound.

  14. Change in Frequency of Dialysis from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
  15. 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.

  16. Change in Frequency of Renal Stone Events from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
  17. Change in Renal Function Assessed by Estimated Glomerular Filtration Rate (eGFR) from Baseline to End of Study [ Time Frame: Baseline, Month 60 ]
  18. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04152200


Contacts
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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

Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Tracy McGregor, MD Alnylam Pharmaceuticals

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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04152200     History of Changes
Other Study ID Numbers: ALN-GO1-005
2019-001346-17 ( EudraCT Number )
First Posted: November 5, 2019    Key Record Dates
Last Update Posted: November 5, 2019
Last Verified: November 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alnylam Pharmaceuticals:
PH1
Primary Hyperoxaluria
Hyperoxaluria
RNAi Therapeutic
siRNA
AGT
Systemic Oxalosis
Additional relevant MeSH terms:
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Hyperoxaluria, Primary
Hyperoxaluria
Kidney Diseases
Urologic Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases