Clinical Trials Logo
  1. Home
  2. Hemoglobinopathies
  3. NCT04853576

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

Sickle Cell Disease Clinical Trial

Official title:
A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease

Clinical Trial Summary

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).

Clinical Trial Description

This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant (HSCT) in subjects with severe SCD. Planned study subjects will be comprised of male and female adult and adolescent subjects with severe SCD, from 12 to 50 years of age, inclusive. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04853576
Study type Interventional
Source Editas Medicine, Inc.
Contact Editas Medicine's Clinical Trial Team
Phone 617-401-9007
Email Patients@editasmed.com
Status Recruiting
Phase Phase 1/Phase 2
Start date May 4, 2021
Completion date August 2025
View Details
See also
  Status Clinical Trial Phase
Completed NCT02227472 - Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease
Recruiting NCT06301893 - Uganda Sickle Surveillance Study (US-3)
Recruiting NCT04398628 - ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Completed NCT02522104 - Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH) Phase 4
Recruiting NCT04688411 - An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease N/A
Terminated NCT03615924 - Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease Phase 3
Not yet recruiting NCT06300723 - Clinical Study of BRL-101 in Severe SCD N/A
Recruiting NCT03937817 - Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Completed NCT04917783 - Health Literacy - Neurocognitive Screening in Pediatric SCD N/A
Completed NCT04134299 - To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease N/A
Completed NCT02580565 - Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
Recruiting NCT04754711 - Interest of Nutritional Care of Children With Sickle Cell Disease on Bone Mineral Density and Body Composition N/A
Completed NCT04388241 - Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD N/A
Recruiting NCT05431088 - A Phase 2/3 Study in Adult and Pediatric Participants With SCD Phase 2/Phase 3
Completed NCT01158794 - Genes Influencing Iron Overload State
Recruiting NCT03027258 - Point-of-Delivery Prenatal Test Results Through mHealth to Improve Birth Outcome N/A
Withdrawn NCT02960503 - Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease Phase 1/Phase 2
Withdrawn NCT02630394 - A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease Phase 1
Completed NCT02567682 - Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects Phase 1
Completed NCT02567695 - A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects Phase 1