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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 been started on deferiprone before the age of 18; 60% of patient aged <10 and 38% <6. Patients were followed for a median of 2 years and the incidence of adverse drug reactions were in line with available data in older populations.

 Source: https://www.deepproject.eu/deep-3-performing-a-long-term-safety-study-of-deferiprone-use-in-paediatric-patients/



Bitopertin molecule in NTDT patients


Update: 02 October 2018

The exploration of the use of bitopertin in NTDT patients has been undertaken in the βitoThal (BP39642) proof-of-mechanism, open-label study. The 22 week study will explore the safety, tolerability and efficacy of bitopertin, a molecule given by mouth. A total of 24 patients across 3 countries have been enrolled.

The safety and efficacy of the molecule of NTDT patients has not been established, hence the trial has terminated.

Source: https://clinicaltrials.gov/ct2/show/NCT03271541


New oral chelator SP-420


Update: 02 October 2018

A phase 1 study evaluated the safety and pharmacokinetics of a novel oral iron chelator of the desferrithiocin class with the code name SP-420 in 24 TDT patients.

The study was terminated early due to renal adverse events including proteinuria, increase in serum creatinine and one case of Fanconi syndrome


Source: Taher eta al, Am J Hematol, Dec. 2017 https://www.ncbi.nlm.nih.gov/pubmed/28940308




Update: 20 December 2018

The study in adults with β-thalassaemia has been side-lined in favour of Luspatercept.

Source: Email communication.


Update: 02 October 2018

A dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with β-thalassaemia is underway, with results expected in 2020.

The drug has also been retargeted to the treatment of pulmonary hypertension in non haemoglobinopathy patients.

Source: https://clinicaltrials.gov/ct2/show/NCT01571635