Registrationsdatum der Studie
07.11.2017
Einschluss der ersten teilnehmenden Person
16.11.2017
Rekrutierungsstatus
Active, not recruiting
Wissenschaftlicher Titel
(Datenquelle: WHO)
ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias
Studientyp
(Datenquelle: WHO)
Interventional
Design der Studie
(Datenquelle: WHO)
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
Phase
(Datenquelle: WHO)
Phase 3
Primäre Endpunkte
(Datenquelle: WHO)
Annualized Rate of Porphyria Attacks in Participants With Acute Intermittent Porphyria (AIP)
Sekundäre Endpunkte
(Datenquelle: WHO)
The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) in Participants With AIP;The PD Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) in Participants With AIP;Annualized Rate of Hemin Administration in Participants With AIP;Annualized Rate of Porphyria Attacks in Participants With AHP;Area Under the Curve (AUC) of the Change From Baseline in Weekly Mean Score of Daily Worst Pain as Measured by the Brief Pain Inventory-Short Form (BPI-SF) Numeric Rating Scale (NRS) in Participants With AIP;Average Change From Baseline in Weekly Mean Score of Daily Worst Fatigue Score as Measured by the Brief Fatigue Inventory-Short Form (BFI-SF) NRS in Participants With AIP;AUC of the Change From Baseline in Weekly Mean Score Daily Worst Nausea Score as Measured by NRS in Participants With AIP;Average Change From Baseline in Weekly Mean Score Daily Worst Nausea Score as Measured by NRS in Participants With AIP;Change From Baseline in the Physical Component Summary (PCS) of the 12-Item Short Form Survey (SF-12) in Participants With AIP;AUC of the Change From Baseline in Weekly Mean Score of Daily Worst Fatigue Score as Measured by the Brief Fatigue Inventory-Short Form (BFI-SF) NRS in Participants With AIP;Average Change From Baseline in Weekly Mean Score of Daily Worst Pain as Measured by the Brief Pain Inventory-Short Form (BPI-SF) Numeric Rating Scale (NRS) in Participants With AIP
Kontakt für Auskünfte
(Datenquelle: WHO)
Please refer to primary and secondary sponsors