Calendario›QNCX›EryDex Low dose DSP · ATTeST
EryDex Low dose DSP · ATTeST
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
Ensayo de Quince Therapeutics, Inc. en Nervous System Disease · Genetic Syndrome (del registro, en inglés).
- Fase
- Fase 3
- Estado
- Completado
- Participantes
- 176
- Centros
- 22
- Fin del objetivo primario
- may 2021
previstos
fecha real
Qué significa cada fase y cada estado.
Estudio de intervención, aleatorizado, triple ciego. Comenzó en mar 2017.
Qué mide
Change From Baseline in Modified International Cooperative Ataxia Rating Scale (mICARS) (to Month 6 (Visit 9)) (del registro, en inglés)
Cómo lo describe el promotor
En el documentoEn inglés, del registroObjectives: The objective of study was to evaluate the safety and the efficacy of EryDex (Dexamethasone sodium phosphate encapsulated in autologous erythrocytes, using the EryDex System - EDS) at two dose levels (low dose and high dose DSP/infusion), compared to placebo, on Neurological Symptoms in Patients With Ataxia Telangiectasia. Initial Double-Blind Treatment Period (0 to 6 Months) Primary Efficacy Objective: • Evaluate the effect of EryDex at two dose levels (low dose and high dose DSP/infusion), compared to placebo, on central nervous system (CNS) symptoms measured by the change in the Modified International Cooperative Ataxia Rating Scale (mICARS) from baseline to Month 6 (Visit 9) in patients with ataxia telangiectasia (A-T). Secondary Efficacy Objectives: * Evaluate the effect of EryDex, compared to placebo, on the Clinical Global Impression of Change (CGI-C) in patients with A-T from baseline to Month 6 (Visit 9). * Evaluate the effect of EryDex, compared to placebo, on measures of Clinical Global Impression of Severity (CGI-S; structured) in patients with A-T from baseline to Month 6 (Visit 9) * Evaluate the effect of EryDex, compared to placebo, on measures of Adaptive behavior measures in patients with A-T by the Vineland Adaptive Behavior Scales (VABS) from baseline to Month 6 (Visit 9). Safety Objectives: • Evaluate the safety and tolerability of two non-overlapping doses of EryDex, compared to placebo, in patients with A-T over the 12-month double-blind study duration. Extension Treatment Period (6-12 Months): Primary Objective: • Evaluate the efficacy of EryDex at two dose levels (low dose and high dose DSP/infusion) compared to placebo, in treating CNS symptoms in A-T patients during longer-term treatment (up to 12 months), as measured by the mICARS. Secondary Objectives: * Evaluate the longer-term (up to 12 months) safety and tolerability of EryDex in A-T patients. * Compare the effects of EryDex on the CGI-C and CGI-S (structured), VABS, and QoL using the EQ-5D-5L scale.
Más ensayos de QNCX
| Ensayo | Fase | Estado | Lectura de resultados |
|---|---|---|---|
| Dexamethasone sodium phosphate · OLE_NEAT | Fase 3 | Interrumpido | ene 2026 (real) |
| Dexamethasone sodium phosphate · NEAT | Fase 3 | Completado | dic 2025 (real) |
| EryDex System · OLE-IEDAT | Fase 3 | Interrumpido | sep 2022 (real) |
| DSP loaded RBC using EryDex… | Fase 1 | Completado | jun 2015 (real) |
| International Ataxia Rating Scale in Younger Patients | no aplica | Completado | may 2014 (real) |
| EryDex | Fase 1 | Completado | oct 2013 (real) |
Todos los de QNCX, en su ficha.
Ficha completa en ClinicalTrials.gov (NCT02770807), actualizada en may 2024.