Open-label, Long-term, Extension Treatment Using Intra-Erythrocyte Dexamethasone Sodium Phosphate (EryDex System) in Patients With Ataxia Telangiectasia Who Participated in the ATTeST-IEDAT-02-2015 Study
Who is this study for? Child to adult patients with Ataxia Telangiectasia
Status: Terminated
Location: See all (17) locations...
Intervention Type: Combination product
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
Objective: To evaluate the long-term effect of EDS-EP in treating central nervous system (CNS) symptoms, as measured by the Modified International Cooperative Ataxia Rating Scale (mICARS), and Clinical Global Impression of severity and change (CGI-S/C).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• Patient completed the double-blind period in the ATTeST study and completed the final (Visit 15 / Month 12) Efficacy Assessments of ATTeST or discontinued the study during the COVID-19 pandemic.
• Patient tolerated the study medication, without any evidence of steroid adverse events, or treatment-related severe events / serious adverse events.
• Body weight \> 15 kg.
• The patient and his / her parent / caregiver (if below the age of consent), or a legal representative, provided written informed consent to participate. If consent was provided solely by the caregiver in accordance with local regulations, the patient also was asked to provide their assent to participate in the study.
• Patient did not present safety contraindication for continuation of treatment, as determined by the Principal Investigator (PI) according to the procedures described below.
⁃ Moreover, patients who were discontinued from the ATTeST study during the COVID-19 pandemic were eligible to receive the EryDex treatment in the IEDAT-03-2018 study, in the absence of safety contraindications to continuation of the treatment, and after signing the informed consent.
⁃ There were no de novo enrolled patients.
Locations
United States
California
Ataxia Center and HD Center of Excellence - UCLA
Los Angeles
Maryland
Division of Pediatric Allergy and Immunology - Johns Hopkins Hospital
Baltimore
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
Texas
Department of Pediatrics Division of Child and Adolescent Neurology Mitochondrial Clinic - University of Texas Medical School
Houston
Other Locations
Australia
Department of Neurology Royal Children's Hospital
Parkville
Belgium
Laboratory of Pediatric Immunology UZ Leuven
Leuven
Germany
Klinik für Kinder- und Jugendmedizin, Allergologie, pneumologie und Mukoviszidose, Universitätsklinikum Frankfurt
Frankfurt
India
National Institute of Mental Health and Neurosciences (NIMHANS) Department of Neurology
Bangalore
Vijaya Health Centre, Department of Neurology
Chennai
Nizam's Institute of Medical Sciences Department of Neurology
Hyderabad
Jaslok Hospital and Research Center Department of Pediatric Neurology
Mumbai
Italy
Dipartimento Neuroscienze umane e salute mentale, Policlinico Umberto I Università La Sapienza
Roma
Norway
Norwegian National Unit for Newborn Screening, Division of Pediatric and Adolescent Medicine, Oslo University Hospital
Oslo
Poland
Department of Clinical Immunology - The Children's Memorial Health Institute
Warsaw
Spain
Servicio de Neurolgia Pediatrica, Hospital Materno-Infantil La Paz
Nottingham University Hospitals NHS Trust - Queen's Medical Centre
Nottingham
Time Frame
Start Date:2018-06-12
Completion Date:2022-09-02
Participants
Target number of participants:104
Treatments
Experimental: active drug
\~14-22 mg dexamethasone sodium phosphate (DSP) for 12 months. The duration of EryDex treatment could be extended further and continue until patients eventually withdrew consent, or the Investigator decided to discontinue treatment based on a risk / benefit assessment.