A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on subjects with Ataxia Telangiectasia.
- Conditions
- Ataxia TelangiectasiaMedDRA version: 21.0Level: PTClassification code: 10003594Term: Ataxia telangiectasia Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- CTIS2023-509077-23-00
- Lead Sponsor
- Quince Therapeutics S.p.A.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 105
Subject meets clinical criteria for diagnosis of A-T. The neurological signs of A-T (incoordination of the head and eyes in lateral gaze deflection, gait ataxia associated with an inappropriately narrow base) must be documented. Such signs of A-T illustrate the body systems in which changes shall be confirmed, but the listed signs are examples and other changes in those systems may be observed and documented to confirm the diagnosis of A-T., Subject is in autonomous gait or is helped by periodic use of a support (i.e., ICARS score for Item 1 – Walking Capacities between 0 and 4 included)., Subject is at least 6 years of age (Day 0/Dose 1 must be on or after date of 6th birthday), of either sex., Genetic confirmation of A-T., Body weight =15 kg., The subject and parent/caregiver (if below the age of consent), or a legal representative, has provided written informed consent to participate. If consent is provided solely by the caregiver in accordance with local regulations, the subject must provide assent to participate in the study, to the extent possible.
General - A disability that may prevent the subject from completing all study requirements., Medical History and Current Status - Any clinically significant abnormality on standard laboratory examinations (hematology, biochemistry, urinalysis) at screening that remains abnormal on repeat testing, if considered as a possible sign of a clinical condition putting the subject at risk if enrolled. Eligibility of subjects with abnormal laboratory test values will be determined by the Investigator in consultation with the Medical Monitor., Medical History and Current Status - Subject with an early morning plasma cortisol level below 3-5 µg/dL (depending on assay) or subject exhibits signs or symptoms of adrenal insufficiency with an early morning plasma cortisol level below 10 µg/dL, and fails the ACTH stimulation test at screening., Medical History and Current Status - Confirmed hemoglobinopathies, e.g., hemoglobin C disease, sickle cell anemia, hereditary spherocytosis, or thalassemia., Medical History and Current Status - Current chronic or acute significant renal and/or hepatic impairment that in Investigator’s opinion will impact subject participation in the study., Medical History and Current Status - Subjects with suicidal ideation, Medical History and Current Status - Females that are pregnant or breast feeding. Females of childbearing potential using an adequate birth control method, as determined by their Health Care Provider, will be eligible. For further details on the highly effective contraceptive measures, Prior/Concomitant Medication - 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., Prior/Concomitant Medication - Chronic condition or prior allergic reaction representing a contraindication to the use of dexamethasone or other steroid drugs., Prior/Concomitant Medication - Has participated in any other trial with an investigational drug and received a dose within 30 days or at least 5 half-lives (whichever is greater) prior to the Screening visit., Prior/Concomitant Medication - Has participated in a previous trial with EryDex treatment, General - Current participation in another clinical study. Participation in observational, non-interventional studies is allowed with approval by the Medical Monitor as long as study investigational endpoint raters can remain blinded to the assessments from other studies, and as long as the other study participation does not interfere with participation in this study., Prior/Concomitant Medication - Requires any concomitant medication prohibited by the protocol, Medical History and Current Status - Immune impairment that includes CD4+ lymphocytes count <400/mm3 (for subjects less than 7 years old) or <150/mm3 (for subjects =7 years old). In presence of oral infections, like oral candidiasis, documented at screening or recurrent as per medical history documentation, the limit increases to <200/mm3 (for subjects =7 years old)., Medical History and Current Status - History of severe impairment of the immunological system such that steroid treatment would be contraindicated., Medical History and Current Status - Loss/removal of 250 mL or more of blood within the past 4 weeks prior to screening., Medical History and Current Status - Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years., Medical History and C
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Primary Efficacy Objective: To evaluate the effect of EryDex on CNS symptoms, as measured by the change of the RmICARS from baseline to Visit 9 (Day 168) compared to placebo in A-T (6- to 9-year-old subjects primary analysis population).;Secondary Objective: Secondary Efficacy Objective (key secondary) = To evaluate the overall clinical effect of EryDex, compared to placebo, in A-T (6- to 9-year-old subjects primary analysis population), based on CGI-S change from baseline to Visit 9 (Day 168), CGI-C at Visit 9 (Day 168).;Primary end point(s): Primary Efficacy Endpoint= To evaluate the effect of EryDex on CNS symptoms, as measured by the change of the RmICARS from baseline to Visit 9 (Day 168) compared to placebo in A-T (6- to 9-year-old subjects primary analysis population).
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Key Secondary Efficacy Endpoint = To evaluate the overall clinical effect of EryDex, compared to placebo, in A-T (6- to 9-year-old subjects primary analysis population), based on CGI-S from baseline to Visit 9 (Day 168);Secondary end point(s):Other Secondary Efficacy Endpoint = CGI-C from baseline to Visit 9 (Day 168)