Recurrent Calcium Oxalate Kidney Stone Disease, Elevated Urinary Oxalate Levels
Conditions
Keywords
Calcium oxalate, Kidney stones, Elevated urinary oxalate, Urinary oxalate
Brief summary
The primary purpose of this study is to evaluate the effect of lumasiran on the percent change in urinary oxalate excretion in patients with recurrent calcium oxalate kidney stone disease.
Interventions
DRUGLumasiran
Lumasiran will be administered by SC injection.
DRUGPlacebo
Placebo will be administered by SC injection.
Sponsors
Alnylam Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Documented diagnosis of recurrent kidney stone disease based on ≥2 stone events with a minimum of 1 stone event within 5 years prior to screening * Meets the 24 hour urine oxalate excretion requirements * The 2 most recently analyzed kidney stones prior to randomization contained 50% or more of calcium oxalate; if only one stone analysis is available, then it must have contained 50% or more of calcium oxalate
Exclusion criteria
* Known history of secondary causes of elevated urinary oxalate and/or recurrent kidney stones * Primary hyperoxaluria * Estimated glomerular filtration rate (eGFR) of \<30 mL/min/1.73m\^2 at screening * Received an investigational agent within the last 30 days or 5 half-lives, whichever is longer
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent Change in 24-hour Urinary Oxalate From Baseline to Month 6 | Baseline to Month 6 |
Secondary
| Measure | Time frame |
|---|---|
| Percentage of Patients Who Achieve a ≥20% Reduction in 24-hour Urinary Oxalate From Baseline to Month 6 | Baseline to Month 6 |
| Percent Change in Urinary Calcium Oxalate Supersaturation From Baseline to Month 6 | Baseline to Month 6 |
Countries
Belgium, Italy, Spain, Switzerland, United Kingdom, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Lumasiran Dose 1 Participants will be administered lumasiran by subcutaneous (SC) injection.
Lumasiran: Lumasiran will be administered by SC injection. | 1 |
| Lumasiran Dose 2 Participants will be administered lumasiran by SC injection.
Lumasiran: Lumasiran will be administered by SC injection. | 0 |
| Placebo Participants will be administered placebo by SC injection.
Placebo: Placebo will be administered by SC injection. | 1 |
| Total | 2 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Study terminated by Sponsor | 1 | 0 | 0 |
| Overall Study | Withdrawal by Subject | 0 | 0 | 1 |
Baseline characteristics
| Characteristic | Lumasiran Dose 1 | Lumasiran Dose 2 | Placebo | Total |
|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Age, Continuous | 52 years | — | 65 years | 58.5 years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | — | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | — | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | — | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | — | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | — | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | — | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 1 Participants | — | 1 Participants | 2 Participants |
| Region of Enrollment Belgium | 1 participants | — | 0 participants | 1 participants |
| Region of Enrollment Italy | 0 participants | — | 0 participants | 0 participants |
| Region of Enrollment Spain | 0 participants | — | 0 participants | 0 participants |
| Region of Enrollment Switzerland | 0 participants | — | 0 participants | 0 participants |
| Region of Enrollment United Kingdom | 0 participants | — | 0 participants | 0 participants |
| Region of Enrollment United States | 0 participants | — | 1 participants | 1 participants |
| Sex: Female, Male Female | 1 Participants | — | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 0 Participants | — | 1 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 1 | 0 / 0 | 0 / 1 |
| other Total, other adverse events | 1 / 1 | 0 / 0 | 1 / 1 |
| serious Total, serious adverse events | 0 / 1 | 0 / 0 | 0 / 1 |
Outcome results
Primary
Percent Change in 24-hour Urinary Oxalate From Baseline to Month 6
Time frame: Baseline to Month 6
Population: No participant data was collected for this outcome measure due to early study termination.
Secondary
Percentage of Patients Who Achieve a ≥20% Reduction in 24-hour Urinary Oxalate From Baseline to Month 6
Time frame: Baseline to Month 6
Population: No participant data was collected for this outcome measure due to early study termination.
Secondary
Percent Change in Urinary Calcium Oxalate Supersaturation From Baseline to Month 6
Time frame: Baseline to Month 6
Population: No participant data was collected for this outcome measure due to early study termination.