A Proof of Concept Clinical Trial to Investigate OTL-300 in Transfusion-Dependent Beta-Thalassemia Patients
Phase of Trial: Phase I
Latest Information Update: 22 Jan 2019
At a glance
- Drugs OTL 300 (Primary)
- Indications Beta-thalassaemia
- Focus Adverse reactions; Proof of concept; Therapeutic Use
- 22 Jan 2019 According to an Orchard Therapeutics media release, this trial with the clinical coordination of doctor Sarah Marktel of SR-Tiget, has involved other Italian centers specializing in thalassemia and the cooperation of patient organizations.
- 22 Jan 2019 According to an Orchard Therapeutics media release, the company anticipates reporting data from all nine patients in this proof of concept trial later this year.
- 22 Jan 2019 According to an Orchard Therapeutics media release, interim analysis of long-term efficacy outcomes in seven of the nine treated patients with more than one year of follow up from this ongoing trial were published in Nature Medicine Journal.