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
Zusammenfassung der Studie
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)
Untersuchte Intervention
EryDex
(BASEC)
Untersuchte Krankheit(en)
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)
Ausschlusskriterien
- 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)
Studienstandort
Zürich
(BASEC)
Sponsor
Quince Therapeutics S.p.A.
(BASEC)
Kontakt für weitere Auskünfte zur Studie
Kontaktperson Schweiz
Pamela Raggi
00393473962536
clinops@clutterquincetx.comQuince Therapeutics S.p.A.
(BASEC)
Wissenschaftliche Auskünfte
Quince Therapeutics S.p.A.,
415-533-3236
clinops@clutterquincetx.com(ICTRP)
Name der bewilligenden Ethikkommission (bei multizentrischen Studien nur die Leitkommission)
Ethikkommission Zürich
(BASEC)
Datum der Bewilligung durch die Ethikkommission
17.01.2025
(BASEC)
ICTRP Studien-ID
NCT06193200 (ICTRP)
Offizieller Titel (Genehmigt von der Ethikkommission)
A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on subjects with Ataxia Telangiectasia (BASEC)
Wissenschaftlicher Titel
A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on Subjects With Ataxia Telangiectasia (NEAT) (ICTRP)
Öffentlicher Titel
Evaluate the Neurological Effects of EryDex on Subjects With A-T (ICTRP)
Untersuchte Krankheit(en)
Ataxia Telangiectasia (ICTRP)
Untersuchte Intervention
Drug: Dexamethasone sodium phosphateOther: Placebo (ICTRP)
Studientyp
Interventional (ICTRP)
Studiendesign
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor). (ICTRP)
Ein-/Ausschlusskriterien
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)
nicht verfügbar
Primäre und sekundäre Endpunkte
Rescored modified International Cooperative Ataxia Rating Scale (RmICARS) (ICTRP)
Clinical Global Impression of Severity (CGI-S);Clinical Global Impression of Change (CGI-C) (ICTRP)
Registrierungsdatum
11.12.2023 (ICTRP)
Einschluss des ersten Teilnehmers
nicht verfügbar
Sekundäre Sponsoren
Biotrial (ICTRP)
Weitere Kontakte
Dirk Thye, MD;Dirk Thye, MD, clinops@quincetx.com, 415-533-3236, Quince Therapeutics S.p.A., (ICTRP)
Sekundäre IDs
IEDAT-04-2022 (ICTRP)
Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten
nicht verfügbar
Weitere Informationen zur Studie
https://clinicaltrials.gov/ct2/show/NCT06193200 (ICTRP)
Ergebnisse der Studie
Zusammenfassung der Ergebnisse
nicht verfügbar
Link zu den Ergebnissen im Primärregister
nicht verfügbar