A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT03745287

Recruitment Status : Active, not recruiting

First Posted : November 19, 2018

Last Update Posted : May 16, 2024

View this study on the modernized ClinicalTrials.gov

Sponsor:

Collaborator:

CRISPR Therapeutics

Information provided by (Responsible Party):

Vertex Pharmaceuticals Incorporated

No Study Results Posted on ClinicalTrials.gov for this Study

About Study Results Reporting on ClinicalTrials.gov

Recruitment Status :	Active, not recruiting
Estimated Primary Completion Date :	October 2024
Estimated Study Completion Date :	October 2024

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.

Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and beta-Thalassemia. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5.

Modarai SR, Kanda S, Bloh K, Opdenaker LM, Kmiec EB. Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples. Gene Ther. 2021 Feb;28(1-2):105-113. doi: 10.1038/s41434-020-00192-z. Epub 2020 Sep 1.

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