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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.
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
Sotatercept
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
