OLE-IEDAT, version 5.0
Research type
Research Study
Full title
Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study
IRAS ID
277790
Contact name
William Whitehouse
Contact email
Sponsor organisation
EryDel S.p.A.
Eudract number
2018-000338-36
Clinicaltrials.gov Identifier
115929, US IND
Duration of Study in the UK
1 years, 11 months, 4 days
Research summary
Ataxia telangiectasia (AT) is considered a rare disease. There is no marketed drug approved to treat AT, and the progression of the neurologic problems is currently unstoppable. Neurological degeneration is the major contributor to the severe outcome of the disease. The onset of symptoms becomes evident in early childhood when children demonstrate unsteadiness while walking, or swaying when standing still or sitting.
Early findings with administration of betamethasone demonstrated the benefit of a short-term treatment on the neurological impairment of AT patients. However, treatment discontinuation was accompanied by the reappearance of symptoms. These previous results prompted the search for steroids that could be given at low plasma concentrations for an extended period of time without the associated steroid-like effects. The trial treatment, consisting of dexamethasone sodium phosphate encapsulated in autologous erythrocytes, allows the administration of dexamethasone at low plasma concentrations without the associated steroid side effects, despite long-term use in the pediatric population.
This open-label study allows patients who completed the full treatment period in the IEDAT-02-2015, to continue treatment as long as their physician determines they are not experiencing any adverse effects that would necessitate discontinuation of treatment and do not present safety contraindications to continuation of treatment. This study also gives patients who were receiving placebo treatment in the prior study the opportunity to start treatment with the medication. The study aims to collect information on the long-term safety and efficacy of the trial treatment.
Patients meeting all selection criteria will receive monthly infusions. The open-label treatment period is planned for 12 months, but may be extended further and continue until patients eventually withdraw consent or the Investigator decides to discontinue treatment based on a risk/ benefit assessment.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
20/EM/0104
Date of REC Opinion
27 May 2020
REC opinion
Further Information Favourable Opinion