Phase 2
N=20
An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
PH1 · Primary Hyperoxaluria · RNAi Therapeutic · siRNA · AGT
Bottom Line
View on ClinicalTrials.gov: NCT03350451 ↗Enrolled (actual)
20
Serious AEs
35.0%
Results posted
Apr 2024
Primary outcome: Primary: Number of Participants With at Least One Adverse Event (AE) — 13; 7 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Lumasiran (Drug)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- All
- Sponsor
- Alnylam Pharmaceuticals
- Primary completion
- Feb 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With at Least One Adverse Event (AE) |
13; 7 | — |
| SECONDARY Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 54 Months |
-1.425; -2.126 | — |
| SECONDARY Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 54 Months |
-0.2175; -0.2480 | — |
| SECONDARY Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 54 Months |
0.674; -12.026 | — |
Summary
The purpose of this study is to evaluate the long-term safety and tolerability of lumasiran in participants with Primary Hyperoxaluria Type 1.
Eligibility Criteria
Inclusion Criteria
- Enrollment within 12 months of completion of Study ALN-GO1-001
- In the opinion of the investigator tolerated the study drug
- If taking Vitamin B6 (pyridoxine), willing to remain on a stable regimen for the study duration
- Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception
- Willing to provide written informed consent and to comply with study requirements
Exclusion Criteria
- Clinically significant health concerns (with the exception of PH1)
- Clinically significant cardiovascular abnormality
- Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant
- Requirement for chronic dialysis
Data sourced from ClinicalTrials.gov (NCT03350451). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.