Phase 2
Completed N=16
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
Acute Intermittent Porphyria
Source: ClinicalTrials.gov NCT02949830 ↗
Enrolled (actual)
16
Serious AEs
43.8%
Results posted
Mar 2024
Primary outcomePrimary: Percentage of Participants With Adverse Events (AEs) — 100 percentage of participants
Summary
The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients who completed study ALN-AS1-001 (NCT02452372).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Adverse Events (AEs) |
100 | — |
| SECONDARY The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) as Measured by Percent Decrease From Baseline |
92.531 | — |
| SECONDARY The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) as Measured by Percent Decrease From Baseline |
94.194 | — |
| SECONDARY Annualized Rate of Composite Porphyria Attacks |
0.4 | — |
| SECONDARY Annualized Rate of Hemin Administration |
0.9 | — |
Eligibility Criteria
Inclusion Criteria
- Completed participation in Part C of study ALN-AS1-001 (NCT02452372)
- Not on a scheduled regimen of hemin
- Women of child bearing potential must have a negative serum pregnancy test, not be nursing, and use acceptable contraception
- Willing and able to comply with the study requirements and to provide written informed consent
Exclusion Criteria
- Clinically significant abnormal laboratory results
- Received an investigational agent (other than ALN-AS1) within 90 days before the first dose of study drug or are in follow-up of another clinical study
- History of multiple drug allergies or intolerance to subcutaneous injection
Data sourced from ClinicalTrials.gov (NCT02949830). 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. Informational only — not medical advice.