drugset / Trial / NCT02452372
A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
RandomizedParallel-groupSingle-blindTreatment
Summary
The purpose of this study is to evaluate the safety and tolerability of givosiran (ALN-AS1) in AIP patients as well as to characterize pharmacokinetics (PK) and pharmacodynamics (PD) of ALN-AS1 in AIP patients.
Timeline
- Start
- 2015-05-06
- Primary completion
- 2017-09-06
- Completion
- 2017-09-06
Publications
- Sardh E, Balwani M, Rees DC, Anderson KE, Jia G, Sweetser MT, Wang B. Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study. Orphanet J Rare Dis. 2024 Oct 3;19(1):365. doi: 10.1186/s13023-024-03284-w.
- Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE. Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria. N Engl J Med. 2019 Feb 7;380(6):549-558. doi: 10.1056/NEJMoa1807838.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Givosiran | Small interfering RNA (siRNA) | 0.035 mg/kg | Subcutaneous |
| Comparator | Givosiran | Small interfering RNA (siRNA) | 0.1 mg/kg | Subcutaneous |
| Comparator | Givosiran | Small interfering RNA (siRNA) | 0.35 mg/kg | Subcutaneous |
| Comparator | Givosiran | Small interfering RNA (siRNA) | 1 mg/kg | Subcutaneous |
| Comparator | Givosiran | Small interfering RNA (siRNA) | 2.5 mg/kg | Subcutaneous |
| Comparator | Givosiran | Small interfering RNA (siRNA) | 5 mg/kg | Subcutaneous |