Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
van Spronsen Francjan, Peters Heidi, Margvelashvili Lali, Agladze Dodo, Schwartz Ida Vanessa D, Giżewska Maria, Hamazaki Takashi, Guilder Laura, MacDonald Anita, Vijay Suresh, Inwood Anita, Minami Maria, Fjellbirkeland Olivia, Lund Allan, Lah Melissa, Thomas Janet A, Longo Nicola, Kiykim Ertuğrul, Ishige Mika, Burlina Alberto, Bélanger-Quintana Amaya, Rutsch Frank, Opladen Thomas, Mungan Halise, Ezgü Fatih, Bratkovic Drago, Chakrapani Anupam, Ito Tetsuya, Guimas Arlindo, Zori Roberto, Tchan Michel, Sacharow Stephanie, Oliveira Anabela, Janeiro Patricia, Guerra Ixiu-Cabrales, Plana Jaume Campistol, Yıldız Yılmaz, Canda Ebru, Khan Aneal, Vockley Jerry, Breilyn Margo Sheck, Manti Filippo, Larkin Alex, Hughes Catalina, Liu Emelline, Gao Lan, Ingalls Kimberly, Smith Neil, Muntau Ania C
Аннотация
METHODS: Participants received an age-based dose of oral sepiapterin daily; those with mean blood phenylalanine (Phe) levels <360 μmol/L (<5.95 mg/dL) after 2 weeks underwent a 26-week dietary Phe tolerance assessment, wherein dietary Phe intake was adjusted and blood Phe levels monitored. Other participants continued treatment with optional diet liberalization. Primary endpoints included change from baseline to week 26 in dietary Phe intake and treatment-emergent adverse events (TEAEs).
RESULTS: As of September 2, 2024, 169 participants received sepiapterin (median [minimum, maximum] age: 14.0 [0.2, 55.0] years, median exposure: 72.9 weeks); 102 participants underwent dietary Phe tolerance assessments. Mean (SD) dietary Phe intake increased from 27.6 (18.0) mg/kg/day at baseline to 62.5 (41.5) mg/kg/day at week 26 (least-squares mean change [SE]: 36.4 [2.8] mg/kg/day from baseline) (P < .0001 from post hoc analysis). The incidence of treatment-related TEAEs was 29.0%; 3 participants (1.8%) discontinued treatment owing to treatment-related TEAEs. There were no treatment-related serious TEAEs or deaths.
CONCLUSION: Interim results support the long-term safety of sepiapterin and demonstrate the potential for diet liberalization in adults and children with phenylketonuria.
GOV IDENTIFIER: NCT05166161 (https://www.
CLINICALTRIALS: gov/study/NCT05166161; date of registration, December 8, 2021).