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A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 (ILLUMINATE-A)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03681184
Recruitment Status : Active, not recruiting
First Posted : September 21, 2018
Last Update Posted : December 9, 2019
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
The purpose of this study is to evaluate the efficacy and safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 (PH1).

Condition or disease Intervention/treatment Phase
Primary Hyperoxaluria Type 1 (PH1) Drug: Lumasiran Drug: Sterile Normal Saline (0.9% NaCl) Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
Actual Study Start Date : November 27, 2018
Actual Primary Completion Date : November 5, 2019
Estimated Study Completion Date : May 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Lumasiran (ALN-GO1) Drug: Lumasiran
Multiple doses of lumasiran by subcutaneous (SC) injection

Placebo Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match experimental




Primary Outcome Measures :
  1. Percent Change in 24-hour Urinary Oxalate Excretion from Baseline to Month 6 [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Absolute Change in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) from Baseline to Month 6 [ Time Frame: 6 months ]
  2. Change in 24-hour Urinary Oxalate:Creatinine Ratio (Value/Upper Limit of Normal [ULN]) from Baseline to Month 6 [ Time Frame: 6 months ]
  3. Percentage of Participants with 24-hour Urinary Oxalate Level At or Below 1.5 x ULN at Month 6 [ Time Frame: 6 months ]
  4. Percentage of Participants with 24-hour Urinary Oxalate Level At or Below ULN at Month 6 [ Time Frame: 6 months ]
  5. Percentage Change in Plasma Oxalate from Baseline to Month 6 [ Time Frame: 6 months ]
  6. Absolute Change in Plasma Oxalate from Baseline to Month 6 [ Time Frame: 6 months ]
  7. Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline to Month 6 [ Time Frame: 6 months ]
  8. Absolute Change in 24-hour Urinary Oxalate Excretion from Baseline over Time During the Extension Phase [ Time Frame: Up to 60 months ]
  9. Percentage Change in 24-hour Urinary Oxalate Excretion from Baseline over Time During the Extension Phase [ Time Frame: Up to 60 months ]
  10. Percentage of Time That 24-hour Urinary Oxalate is At or Below 1.5 × ULN During the Extension Phase [ Time Frame: Up to 36 months ]
  11. Change in 24-hour Urinary Oxalate:Creatinine Ratio (Value/Upper Limit of Normal [ULN]) from Baseline over Time During the Extension Phase [ Time Frame: Up to 60 months ]
  12. Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline over Time During the Extension Phase [ Time Frame: Up to 60 months ]


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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Willing to provide written informed consent or assent and to comply with study requirements
  • Confirmation of PH1 disease
  • Meet the 24 hour urine oxalate excretion requirements
  • If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days

Exclusion Criteria:

  • Clinically significant health concerns (with the exception of PH1) or clinical evidence of extrarenal systemic oxalosis
  • Clinically significant abnormal laboratory results
  • Known active or evidence of HIV or hepatitis B or C infection
  • An estimated GFR of < 30 mL/min/1.73m2 at screening
  • Received an investigational agent within 30 days or 5 half-lives before the first dose of study drug or are in follow-up of another clinical study
  • History of kidney or liver transplant
  • Known history of multiple drug allergies or allergic reaction to an oligonucleotide or GalNAc
  • History of intolerance to subcutaneous injection
  • Women who are pregnant, planning a pregnancy, or breast-feeding or those of child bearing potential and not willing to use contraception
  • History of alcohol abuse within the last 12 months, or unable or unwilling to limit alcohol consumption throughout the study

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): NCT03681184


Locations
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United States, Alabama
Clinical Trial Site
Birmingham, Alabama, United States, 35294
United States, California
Clinical Trial Site
San Diego, California, United States, 92120
United States, Florida
Clinical Trial Site
Jacksonville, Florida, United States, 32216
United States, Minnesota
Clinical Trial Site
Rochester, Minnesota, United States, 55905
United States, New York
Clinical Trial Site
New York, New York, United States, 10029
United States, Texas
Clinical Trial Site
Houston, Texas, United States, 77030
France
Clinical Trial Site
Bordeaux, France
Clinical Trial Site
Lyon, France
Clinical Trial Site
Paris, France
Germany
Clinical Trial Site
Bonn, Germany
Israel
Clinical Trial Site
Haifa, Israel
Clinical Trial Site
Jerusalem, Israel
Clinical Trial Site
Nahariya, Israel
Netherlands
Clinical Trial Site
Amsterdam, Netherlands
Switzerland
Clinical Trial Site
Bern, Switzerland
United Arab Emirates
Clinical Trial Site
Dubai, United Arab Emirates
United Kingdom
Clinical Trial Site
Birmingham, United Kingdom
Clinical Trial Site
London, United Kingdom, NW3 2QG
Clinical Trial Site
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: John Gansner, MD Alnylam Pharmaceuticals

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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03681184     History of Changes
Other Study ID Numbers: ALN-GO1-003
2018-001981-40 ( EudraCT Number )
First Posted: September 21, 2018    Key Record Dates
Last Update Posted: December 9, 2019
Last Verified: December 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
RNAi therapeutic
siRNA
AGT
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