Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study

Stopped early · Phase 3

Conditions studied: Ataxia Telangiectasia, Genetic Syndrome

In brief

Primary Objective To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients. Secondary Objective To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale). Exploratory 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).

Key facts

Study ID

NCT03563053

Run by

Quince Therapeutics S.p.A.

People needed

104

Starts

2018-06-12

Expected to finish

2022-09-02

Last updated by the study team

2024-10-08

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Patients who met one or more of the following criteria were not considered to be eligible to participate in the study:
• General
• Females that were:
• Pregnant, or were breast-feeding (for EU countries only)
• Of childbearing potential, pregnant, or were breast-feeding (for US and Rest of World countries).
• Females of childbearing potential using adequate birth control, as determined by their Health Care Provider, were eligible.
• A disability that may prevent the patient from completing all study requirements.
• Current participation in another clinical study with another investigational drug.
• Medical History and Current Status
• Cluster differential 4 positive (CD4+) lymphocytes count < 400 / mm3 (for patients 6 years of age) or < 150 / mm3 (for patients > 6 years). In presence of oral infections, like oral candidiasis, documented at the screening or recurrent as per medical history documentation, the limit increases to < 200 / mm3 (for patients > 6 years).
• Current neoplastic disease.
• Severe impairment of the immunological system.
• Severe or unstable pulmonary disease.
• Uncontrolled diabetes. Patients with diabetes that had been stabilized (i.e., no hypoglycemic or hyperglycemic episodes in the past 3 months) were eligible.
• Any other severe, unstable, or serious disease or condition that in the Investigator's opinion would put the patient at risk for imminent life-threatening morbidity, need for hospitalization, or mortality.
• Eligibility of patients with abnormal laboratory test values were determined by the Investigator.
• Confirmed haemoglobinopathies, e.g., haemoglobin C disease, sickle cell anaemia, or thalassemia.
• Moderate or severe renal and / or hepatic impairment.
• Patients who experienced moderate / severe steroid side effects, or moderate / severe adverse events associated with the EryDex treatment administered in the ATTeST study.
• Prior / Concomitant Medication
• Requires treatment with an oral or parenteral steroid. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids were permitted.
• Requires any other concomitant medication prohibited by the protocol.
• Use of any drug that is a strong inducer / inhibitor of Cytochrome P450 3A4 (CYP3A4).

Where it is running

• Ataxia Center and HD Center of Excellence - UCLA — Los Angeles, California, United States
• Division of Pediatric Allergy and Immunology - Johns Hopkins Hospital — Baltimore, Maryland, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Department of Pediatrics Division of Child and Adolescent Neurology Mitochondrial Clinic - University of Texas Medical School — Houston, Texas, United States
• Department of Neurology Royal Children's Hospital — Parkville, Victoria, Australia
• Laboratory of Pediatric Immunology UZ Leuven — Leuven, Belgium
• Klinik für Kinder- und Jugendmedizin, Allergologie, pneumologie und Mukoviszidose, Universitätsklinikum Frankfurt — Frankfurt, Germany
• Vijaya Health Centre, Department of Neurology — Chennai, Tamil Nadu, India
• National Institute of Mental Health and Neurosciences (NIMHANS) Department of Neurology — Bangalore, India
• Nizam's Institute of Medical Sciences Department of Neurology — Hyderabad, India
• Jaslok Hospital and Research Center Department of Pediatric Neurology — Mumbai, India
• Dipartimento Neuroscienze umane e salute mentale, Policlinico Umberto I Università La Sapienza — Roma, Italy
• Norwegian National Unit for Newborn Screening, Division of Pediatric and Adolescent Medicine, Oslo University Hospital — Oslo, Norway
• Department of Clinical Immunology - The Children's Memorial Health Institute — Warsaw, Poland
• Servicio de Neurolgia Pediatrica, Hospital Materno-Infantil La Paz — Madrid, Spain
• Razi Hospital, Clinical Investigation Center-Neuroscience — Tunis, Tunisia
• Nottingham University Hospitals NHS Trust - Queen's Medical Centre — Nottingham, Nottinghamshire, United Kingdom

Full record on ClinicalTrials.gov

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