A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Assess the Efficacy, Safety, Tolerability, PK, and Biomarker Effects of PTC857 in Adult Subjects With Amyotrophic Lateral Sclerosis (CARDINALS)

TerminadoFase 2ClinicalTrials.gov

ID: NCT05349721Tipo: INTERVENTIONALInicio: 15 de may de 2022Fin estimado: 30 de ene de 2025

Neurología

Traducción no disponible, mostrando original

Resumen

This study will assess the efficacy and safety of PTC857 treatment in participants diagnosed with ALS.

Descripción detallada

Participants will be randomized to 1 of the 2 treatment groups: PTC857 or matching placebo. Following successful completion of the Treatment Period, participants who enter the LTE Period, will receive open-label PTC857 for 28 weeks. Following completion of the LTE period, participants who enter the Continued LTE Period will receive open-label PTC857 for an additional 108 weeks.

Elegibilidad

Edad mínima: 18 YearsEdad máxima: 80 YearsSexo: ALL

Criterios de inclusión

ALS with preserved function, defined as:
Onset of the first symptom leading to the diagnosis of ALS ≤24 months at the time of the initial Screening Visit
Revised EL Escorial criteria of either:
(i) Clinically definite ALS (ii) Clinically probable ALS
A total ALSFRS-R score of at least 34 at the start of the Screening Period
No significant respiratory compromise as evidenced by slow vital capacity ≥60% at the start of the Screening Period
All chronic concomitant medications (both prescription and over the counter), and non-pharmacologic therapy regimens, excluding standard-of-care therapy riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, should be stable and unchanged from 14 days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study
Female participants must have a negative breast cancer imaging screening status (not considered clinically abnormal and/or requiring further evaluation/treatment) within 6 months prior to the Screening Visit, or during the Screening Period.
Standard-of-care therapy for the treatment of ALS (riluzole, edaravone, or sodium phenylbutyrate/taurursodiol) should be stable and unchanged from 30 (-3) days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study.

Criterios de exclusión

Females who are pregnant or nursing or plan to become pregnant during the study
Participants with clinically significant gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, psychiatric, or cardiovascular/ischemic disease or any other condition that, in the opinion of the investigator would jeopardize the safety of the participant or impact the validity of the study results
Any clinically significant medical or psychiatric condition or medical history that, in the opinion of the investigator or the medical monitor, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant
Current participation in any other investigational study with an investigational product or participation within 30 days prior to the start of the Screening Period or 5 half-lives of the previously taken investigational drug, whichever is longer
Participant has previously received PTC857
Participant is receiving a combination of edaravone and sodium phenylbutyrate/taurursodiol treatment, where applicable, within 30 days prior to the start of the Screening Period
For female participants, any past medical history of breast cancer, regardless of remission status, or any first degree relative with history of breast cancer

Intervenciones

drug

PTC857

PTC8657 will be administered as an oral solution twice a day.

drug

Placebo

Matching placebo will be administered as an oral solution twice a day.

Ubicaciones

STAT Research S.A.

Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina

Iadin Srl.

Buenos Aires, Argentina

Hospital Ramos Mejía

Buenos Aires, Argentina

Patrocinadores

PrincipalPTC Therapeutics

Aviso: La información de este ensayo proviene de fuentes públicas y tiene carácter exclusivamente informativo. No constituye asesoramiento médico, regulatorio ni de ningún otro tipo. Las traducciones son automatizadas y no poseen validez oficial. Consultá siempre con un profesional de la salud. Ver Términos de Uso.

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, ... | EligiMed