Calendario›ELOX›ELX-02

ELX-02

A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis

Ensayo de Eloxx Pharmaceuticals, Inc. en Genetic Disease · Nonsense Mutation · Cystinosis (del registro, en inglés).

Fase

Fase 2

Estado

Interrumpido

Participantes

3

previstos

Centros

1

Fin del objetivo primario

dic 2019

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en ago 2019.

Este ensayo se detuvo. Motivo declarado por el promotor (del registro, en inglés): «Due to study design limitations, this study has been discontinued and will not proceed with the 2nd cohort as contemplated in the original protocol.».

Qué mide

AEs associated with different dose levels of ELX-02 (From the time of first dosing through the follow-up visit, an average of approximately 10 weeks) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis is a Phase 2 open label study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 in patients with cystinosis with nonsense mutation in at least one allele. Six patients will be enrolled in the trial. The study will comprise of the following periods for each patient: * A screening period of up to 6 weeks * A total treatment period of 4 weeks * A safety follow-up period of 4 weeks after the last treatment Each patient will receive three escalating doses as follows: * Treatment period 1: ELX-02 0.5 mg/kg SC daily for 7 days (total dose not to exceed 3.5 mg/kg for this week; the daily dose will be individualized to achieve the target weekly exposure of about 47.5 µg\*h/mL) * Treatment period 2: ELX-02 1.0 mg/kg SC daily for 7 days (total dose not to exceed 7.0 mg/kg for this week; the daily dose will be individualized to achieve the target weekly exposure of about 95 µg\*h/mL) * Treatment period 3: ELX-02 2.0 mg/kg SC daily for 14 days (total dose not to exceed 14 mg/kg for these two weeks; the daily dose will be individualized to achieve the target weekly exposure of about 190 µg\*h/mL)

Más ensayos de ELOX

Todos los de ELOX, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT04069260), actualizada en may 2020.