At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 6 enrolled
Drug / intervention
DREPAGLOBE drug productgenetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene (GLOBE1 βAS3 Modified Autologous CD34+ Cells) in Patients With Sickle Cell Disease (SCD)
In Brief
A Phase 2 clinical trial evaluating DREPAGLOBE drug product for Sickle Cell Disease. Completed, enrolled 6 participants across 1 site.
Detailed Summary
The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the Sickle Cell disease by Transplantation of an Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced ex vivo with the GLOBE1 lentiviral vector expressing the βAS3 globin gene (GLOBE1 βAS3 Modified Autologous CD34+ Cells) in Patients with Sickle Cell Disease (SCD)
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSickle Cell Disease
CountriesFrance
CollaboratorsURC-CIC Paris Descartes Necker Cochin
Timeline
Phase 2CompletedFinished
2020202120222023202420252026
First PostedMay 2019
Enrollment StartNov 2019
Primary CompletionJul 2022
Study CompletionJan 2024
TodayJul 2026
First PostedMay 28, 2019
Enrollment StartNov 12, 2019
Primary CompletionJul 28, 2022
Study CompletionJan 23, 2024
TodayJul 2, 2026
Enrollment to primary: 2.7 yearsPosted 7.1 years ago
Interventions
DREPAGLOBE drug productgenetic
Each patient will receive a single IV infusion of DREPAGLOBE drug product