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Trofinetide for the treatment of Rett syndrome: Long-term safety and efficacy results of the 32-month, open-label LILAC-2 study.
Percy, Alan K; Neul, Jeffrey L; Benke, Timothy A; Berry-Kravis, Elizabeth M; Glaze, Daniel G; Marsh, Eric D; Barrett, Amy M; An, Di; Bishop, Kathie M; Youakim, James M.
Afiliación
  • Percy AK; Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL, USA.
  • Neul JL; Department of Pediatrics, Vanderbilt Kennedy Center, Vanderbilt University Medical Center, Nashville, TN, USA.
  • Benke TA; Department of Pediatrics, Children's Hospital of Colorado/University of Colorado School of Medicine, Aurora, CO, USA.
  • Berry-Kravis EM; Departments of Pediatrics, Neurological Sciences and Biochemistry, Rush University Medical Center, Chicago, IL, USA.
  • Glaze DG; Department of Pediatrics and Neurology, Texas Children's Hospital/Baylor College of Medicine, Houston, TX, USA.
  • Marsh ED; Division of Child Neurology, Children's Hospital of Philadelphia, Departments of Neurology and Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Barrett AM; Patient-Centered Outcomes Assessment, RTI Health Solutions, Research Triangle Park, NC, USA.
  • An D; Acadia Pharmaceuticals, Inc., San Diego, CA, USA.
  • Bishop KM; Acadia Pharmaceuticals, Inc., San Diego, CA, USA.
  • Youakim JM; Acadia Pharmaceuticals, Inc., San Diego, CA, USA. Electronic address: jyouakim@acadia-pharm.com.
Med ; 5(10): 1275-1281.e2, 2024 Oct 11.
Article en En | MEDLINE | ID: mdl-39025065
ABSTRACT

BACKGROUND:

Trofinetide was approved for the treatment of Rett syndrome (RTT) in patients aged ≥2 years based on the results of the 12-week, randomized, phase 3 LAVENDER study. In LILAC, a 40-week, open-label extension study of LAVENDER, trofinetide continued to improve the symptoms of RTT, with a similar safety profile as LAVENDER. Here, we report long-term safety and efficacy results of LILAC-2, a 32-month, open-label extension study.

METHODS:

Females aged 5-22 years who completed LILAC were eligible to enter LILAC-2. Safety and tolerability were assessed with the incidence of adverse events (AEs). Efficacy was assessed with Rett Syndrome Behaviour Questionnaire (RSBQ) and Clinical Global Impression-Improvement (CGI-I) scores. Caregiver interviews explored the patient's experience with RTT and the efficacy of trofinetide during study participation.

FINDINGS:

In total, 77 participants were enrolled in LILAC-2. The most common AEs were diarrhea (53.2%), COVID-19 (27.3%), and vomiting (19.5%). The mean (standard error [SE]) change in RSBQ score from LAVENDER baseline to week 104 of LILAC-2 was -11.8 (2.45). The mean (SE) CGI-I score from LILAC baseline to week 12 of LILAC-2 was 3.1 (0.10). Most caregivers (96%; n = 24/25) were satisfied or very satisfied with the benefits of trofinetide.

CONCLUSIONS:

Long-term treatment with trofinetide continued to improve RTT symptoms, without new safety concerns. Caregivers reported satisfaction with trofinetide related to improvements that were meaningful for their child and themselves.

FUNDING:

The study was supported by Acadia Pharmaceuticals (San Diego, CA, USA). This study was registered at ClinicalTrials.gov NCT04776746.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Síndrome de Rett Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans Idioma: En Revista: Med Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Síndrome de Rett Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans Idioma: En Revista: Med Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos