A multicenter, randomized, double-blind, placebo-controlled study to assess the neurological effects of EryDex in patients with ataxia telangiectatica (NEAT study) – IEDAT-04-2022
Résumé de l'étude
The study IEDAT-04-2022 is an international, multicenter, randomized, prospective, double-blind, placebo-controlled phase 3 clinical trial evaluating the effect of EryDex, administered as an intravenous (i.v.) infusion once every 28 days (time window +2 days, -7 days), on neurological symptoms of patients with A-T. Evaluations will be conducted for the overall population, for patients aged 6 to 9 years, and for patients aged ≥ 10 years. A total of 11 study visits are planned, from the screening visit to the safety observation. Patients will be invited to at least 8 visits at the study centers and will receive 2 remote visits (phone calls). The safety follow-up may be conducted either by phone or on-site. After a screening period of up to 30 days, patients who meet all selection criteria will be randomized at baseline in a 1:1 ratio to receive EryDex or placebo and will receive the first treatment. Patients will then have five additional treatment appointments. The study is considered complete for a patient when visit 10 has been conducted. Therefore, the duration of the study for an individual patient, including the safety observation phase, is expected to be approximately 8 months.
(BASEC)
Intervention étudiée
EryDex
(BASEC)
Maladie en cours d'investigation
Ataxia telangiectatica
(BASEC)
- The patient meets the clinical criteria for the diagnosis of A-T. - Clinical diagnosis of A-T - The patient is independently mobile or regularly uses a walking aid - Genetic confirmation of A-T - Body weight ≥ 15 kg (BASEC)
Critères d'exclusion
- Current participation in another clinical study - Immunodeficiency - History of severe immune system impairment - Current neoplastic disease or previous neoplastic disease that has not been in remission for at least 2 years - Severe or unstable lung disease - Uncontrolled diabetes - Current significant chronic or acute renal and/or liver dysfunction - Previous oral or parenteral steroid use within 6 weeks prior to study initiation. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as the use of topical steroids, is permitted - A disability that may prevent the patient from meeting all study requirements (BASEC)
Lieu de l’étude
Zurich
(BASEC)
Sponsor
Quince Therapeutics S.p.A.
(BASEC)
Contact pour plus d'informations sur l'étude
Personne de contact en Suisse
Pamela Raggi
00393473962536
clinops@clutterquincetx.comQuince Therapeutics S.p.A.
(BASEC)
Informations scientifiques
Quince Therapeutics S.p.A.,
415-533-3236
clinops@clutterquincetx.com(ICTRP)
Nom du comité d'éthique approbateur (pour les études multicentriques, uniquement le comité principal)
Commission cantonale de Zurich
(BASEC)
Date d'approbation du comité d'éthique
17.01.2025
(BASEC)
Identifiant de l'essai ICTRP
NCT06193200 (ICTRP)
Titre officiel (approuvé par le comité d'éthique)
A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on subjects with Ataxia Telangiectasia (BASEC)
Titre académique
A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on Subjects With Ataxia Telangiectasia (NEAT) (ICTRP)
Titre public
Evaluate the Neurological Effects of EryDex on Subjects With A-T (ICTRP)
Maladie en cours d'investigation
Ataxia Telangiectasia (ICTRP)
Intervention étudiée
Drug: Dexamethasone sodium phosphateOther: Placebo (ICTRP)
Type d'essai
Interventional (ICTRP)
Plan de l'étude
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor). (ICTRP)
Critères d'inclusion/exclusion
Inclusion Criteria:
- Clinical diagnosis of A-T
- In autonomous gait or is helped by periodic use of a support
- Genetic confirmation of A-T
- Body weight =15 kg
Exclusion Criteria:
- Participation in another clinical study
- Immune impairment
- History of severe impairment of the immunological system
- Current neoplastic disease or previous neoplastic disease not in remission for at
least 2 years
- Severe or unstable pulmonary disease
- Uncontrolled diabetes
- Current chronic or acute significant renal and/or hepatic impairment
- Any previous oral or parenteral steroid use within 6 weeks before Baseline.
Treatment with inhaled or intranasal steroids for asthma or allergies, as well as
use of topical steroids will be permitted
- A disability that may prevent the subject from completing all study requirements (ICTRP)
non disponible
Critères d'évaluation principaux et secondaires
Rescored modified International Cooperative Ataxia Rating Scale (RmICARS) (ICTRP)
Clinical Global Impression of Severity (CGI-S);Clinical Global Impression of Change (CGI-C) (ICTRP)
Date d'enregistrement
11.12.2023 (ICTRP)
Inclusion du premier participant
non disponible
Sponsors secondaires
Biotrial (ICTRP)
Contacts supplémentaires
Dirk Thye, MD;Dirk Thye, MD, clinops@quincetx.com, 415-533-3236, Quince Therapeutics S.p.A., (ICTRP)
ID secondaires
IEDAT-04-2022 (ICTRP)
Résultats-Données individuelles des participants
non disponible
Informations complémentaires sur l'essai
https://clinicaltrials.gov/ct2/show/NCT06193200 (ICTRP)
Résultats de l'essai
Résumé des résultats
non disponible
Lien vers les résultats dans le registre primaire
non disponible