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RP-L201

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

Ensayo de ROCKET PHARMACEUTICALS, INC. en Leukocyte Adhesion Defect - Type I (del registro, en inglés).

Fase

Fase 1/2

Estado

Completado

Participantes

9

previstos

Centros

3

Fin del objetivo primario

sep 2023

fecha real

Qué significa cada fase y cada estado.

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

Qué mide

Phase I: Number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v.5.0 (del registro, en inglés) (2 años)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe primary purpose of the Phase I portion of the study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with the therapeutic lentiviral vector, Chim-CD18-WPRE, RP-L201. The primary objectives of the Phase II portion of the study are evaluation of survival, as determined by the proportion of subjects alive at age 2 (24 months) and at least 1-year post-infusion without allogeneic hematopoietic stem cell transplant (HSCT) and characterization of the safety and toxicity associated with the infusion.

Más ensayos de RCKT

Todos los de RCKT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT03812263), actualizada en nov 2023.