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Clinical trial updates

Rapamycin

  Update: 31 January 2020 No update available   Update: 25 November 2019 No update available.   Update: 27 September 2019 Rapamycin, a drug widely used for almost 20 years to protect organ transplant patients, has been found to reduce, in mice, the build up of toxic proteins that destroy ...

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Mitapivat (AG-348)

  Update: 31 January 2020 The clinical proof-of-concept has been established based on a preliminary analysis of the Phase 2 trial of Mitapivat (AG-348) in patients with non-transfusion-dependent thalassaemia announced at the 61st Annual Meeting of the American Society of Haematology. The Phase 2 study has enrolled 12 of the ...

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Concluded/Terminated Studies-Trials

Assessment of long-term safety of deferiprone in children (DEEP-3 Study)     Update: 20 December 2018 The study has concluded.   Update: 02 October 2018 The safety of deferiprone, either alone or in combination with deferoxamine, was evaluated in 297 patients from 16 hospitals in 6 Mediterranean countries, who had ...

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Sickle Cell Disease

ARU-1801 Gene Therapy (SCD)   Update: 31 January 2020 The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to ARU-1801, an experimental gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassaemia. This designation means eligibility for a  priority review voucher once the FDA approves a biologics ...

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Apotransferrin

  Update: 31 January 2020 No update available   Update: 25 November 2019 No update available.   Update: 27 September 2019 No update available   Update: 30 July 2019 No update available.   Update: 30 May 2019  Apotransferrinhas a physiological role in the transportation and distribution of iron among the ...

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Ferroportin inhibitors

VIT-2763   Update: 25 November 2019 No update available.   Update: 27 September 2019 No update available   Update: 30 July 2019 Results announced during 24th Annual Congress of the European Hematology Association show that treatment with single and multiple oral doses of VIT-2763 were well tolerated in healthy subjects, ...

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SLN-124

  Update: 31 January 2020 No update available   Update: 25 November 2019 No update available.   Update: 27 September 2019 No update available   Update: 30 July 2019 No update available.   Update: 30 May 2019 No update available.   Update: 29 March 2019 SLN124 has been submitted to ...

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Denosumab

  Update: 31 January 2020 No update available   Update: 25 November 2019 No update available.   Update: 27 September 2019 No update available   Update: 30 July 2019 Sandoz, a Novartis division focusing on biosimilars have enrolled the first patient in an intergrated Phase I/III study for the development ...

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Reblozyl

  ‘BELIEVE’ Study in Adults with TDT β-thalassaemia   Update: 31 January 2020 Data evaluating Reblozyl® (ex-Luspatercept-aamt) were presented at the 61stAmerican Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida. 1% of patients (101/224 patients receiving Luspatercept-aamt in the phase 3 BELIEVE study) achieved at least a 33% reduction ...

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Gene Therapy

ZYNTEGLO (ex-Lentiglobin) gene therapy for patients with TDT   Update: 31 January 2020 New data from ongoing studies of LentiGlobin™ gene therapy for β-thalassaemia (betibeglogene autotemcel) in pediatric, adolescent and adult patients who have transfusion-dependent β-thalassaemia (TDT), including results from the Phase 3 Northstar-3 (HGB-212) study in patients with a ...

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