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
Descrizione riassuntiva dello studio
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)
Intervento studiato
EryDex
(BASEC)
Malattie studiate
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)
Criteri di esclusione
- 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)
Luogo dello studio
Zurigo
(BASEC)
Sponsor
Quince Therapeutics S.p.A.
(BASEC)
Contatto per ulteriori informazioni sullo studio
Persona di contatto in Svizzera
Pamela Raggi
00393473962536
clinops@clutterquincetx.comQuince Therapeutics S.p.A.
(BASEC)
Informazioni scientifiche
Quince Therapeutics S.p.A.,
415-533-3236
clinops@clutterquincetx.com(ICTRP)
Nome del comitato etico approvante (per studi multicentrici solo il comitato principale)
Commissione etica Zurigo
(BASEC)
Data di approvazione del comitato etico
17.01.2025
(BASEC)
ID di studio ICTRP
NCT06193200 (ICTRP)
Titolo ufficiale (approvato dal comitato etico)
A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on subjects with Ataxia Telangiectasia (BASEC)
Titolo accademico
A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on Subjects With Ataxia Telangiectasia (NEAT) (ICTRP)
Titolo pubblico
Evaluate the Neurological Effects of EryDex on Subjects With A-T (ICTRP)
Malattie studiate
Ataxia Telangiectasia (ICTRP)
Intervento studiato
Drug: Dexamethasone sodium phosphateOther: Placebo (ICTRP)
Tipo di studio
Interventional (ICTRP)
Disegno dello studio
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor). (ICTRP)
Criteri di inclusione/esclusione
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 disponibile
Endpoint primari e secondari
Rescored modified International Cooperative Ataxia Rating Scale (RmICARS) (ICTRP)
Clinical Global Impression of Severity (CGI-S);Clinical Global Impression of Change (CGI-C) (ICTRP)
Data di registrazione
11.12.2023 (ICTRP)
Inclusione del primo partecipante
non disponibile
Sponsor secondari
Biotrial (ICTRP)
Contatti aggiuntivi
Dirk Thye, MD;Dirk Thye, MD, clinops@quincetx.com, 415-533-3236, Quince Therapeutics S.p.A., (ICTRP)
ID secondari
IEDAT-04-2022 (ICTRP)
Risultati-Dati individuali dei partecipanti
non disponibile
Ulteriori informazioni sullo studio
https://clinicaltrials.gov/ct2/show/NCT06193200 (ICTRP)
Risultati dello studio
Riepilogo dei risultati
non disponibile
Link ai risultati nel registro primario
non disponibile