Portal de investigación
Clinical trials in pediatric ALS: a TRICALS feasibility study.
Fecha de publicación:
Fecha Ahead of Print:
Autores de IIS La Fe
Participantes ajenos a IIS La Fe
- Kliest T
- Van Eijk RPA
- Al-Chalabi A
- Albanese A
- Andersen PM
- Amador MDM
- BrÅthen G
- Brunaud-Danel V
- Brylev L
- Camu W
- De Carvalho M
- Cereda C
- Cetin H
- Chaverri D
- Chiò A
- Corcia P
- Couratier P
- De Marchi F
- Desnuelle C
- Van Es MA
- Esteban J
- Filosto M
- GarcÍa Redondo A
- Grosskreutz J
- Hanemann CO
- HolmØy T
- HØyer H
- Ingre C
- Koritnik B
- Kuzma-Kozakiewicz M
- Lambert T
- Leigh PN
- Lunetta C
- Mandrioli J
- Mcdermott CJ
- Meyer T
- Mora JS
- Petri S
- Povedano M
- Reviers E
- Riva N
- Roes KCB
- Rubio MÁ
- Salachas F
- Sarafov S
- SorarÙ G
- Stevic Z
- Svenstrup K
- MØller AT
- Turner MR
- Van Damme P
- Van Leeuwen LAG
- Varona L
- Weber M
- Hardiman O
- Van Den Berg LH
Grupos
Abstract
Background: Pediatric investigation plans (PIPs) describe how adult drugs can be studied in children. In 2015, PIPs for Amyotrophic Lateral Sclerosis (ALS) became mandatory for European marketing-authorization of adult treatments, unless a waiver is granted by the European Medicines Agency (EMA).Objective: To assess the feasibility of clinical studies on the effect of therapy in children (<18 years) with ALS in Europe.Methods: The EMA database was searched for submitted PIPs in ALS. A questionnaire was sent to 58 European ALS centers to collect the prevalence of pediatric ALS during the past ten years, the recruitment potential for future pediatric trials, and opinions of ALS experts concerning a waiver for ALS.Results: Four PIPs were identified; two were waived and two are planned for the future. In total, 49 (84.5%) centers responded to the questionnaire. The diagnosis of 44,858 patients with ALS was reported by 46 sites; 39 of the patients had an onset < 18 years (prevalence of 0.008 cases per 100,000 or 0.087% of all diagnosed patients). The estimated recruitment potential (47 sites) was 26 pediatric patients within five years. A majority of ALS experts (75.5%) recommend a waiver should apply for ALS due to the low prevalence of pediatric ALS.Conclusions: ALS with an onset before 18 years is extremely rare and may be a distinct entity from adult ALS. Conducting studies on the effect of disease-modifying therapy in pediatric ALS may involve lengthy recruitment periods, high costs, ethical/legal implications, challenges in trial design and limited information.
Datos de la publicación
- ISSN/ISSNe:
- 2167-8421, 2167-9223
- Tipo:
- Article
- Páginas:
- 1-8
- PubMed:
- 35172656
- Factor de Impacto:
- 0,854 SCImago ℠
- Cuartil:
- Q2 SCImago ℠
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration TAYLOR & FRANCIS LTD
Citas Recibidas en Web of Science: 11
Documentos
- No hay documentos
Filiaciones
Keywords
- Pediatric amyotrophic lateral sclerosis; clinical trial; clinical trials; ethics; pediatric investigation plan; therapy
Campos de Estudio
Cita
Kliest T,Van R,Al A,Albanese A,Andersen PM,Amador MDM,BrÅthen G,Brunaud V,Brylev L,Camu W,De Carvalho M,Cereda C,Cetin H,Chaverri D,Chiò A,Corcia P,Couratier P,De Marchi F,Desnuelle C,Van MA,Esteban J,Filosto M,GarcÍa A,Grosskreutz J,Hanemann CO,HolmØy T,HØyer H,Ingre C,Koritnik B,Kuzma M,Lambert T,Leigh PN,Lunetta C,Mandrioli J,Mcdermott CJ,Meyer T,Mora JS,Petri S,Povedano M,Reviers E,Riva N,Roes KCB,Rubio MÁ,Salachas F,Sarafov S,SorarÙ G,Stevic Z,Svenstrup K,MØller AT,Turner MR,Van P,Van L,Varona L,VÁZQUEZ JF,Weber M,Hardiman O,Van Den Berg LH. Clinical trials in pediatric ALS: a TRICALS feasibility study. Amyotroph. Lateral Scher. Frontotemp. Degenerat. 2022. 23. (7-8):p. 1-8. IF:2,800. (3).