ot Applicable

Phase 1

• Conditions: Patient with neurological symptoms of Ataxia Telangiectasia; MedDRA version: 20.1Level: PTClassification code 10003594Term: Ataxia telangiectasiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2018-000338-36-ES
• Lead Sponsor: EryDel S.p.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-04-05
• Last Update Posted: 26 September 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 61

Inclusion Criteria

1.Patient completed the double-blind period in the IEDAT-02-2015 trial and must have completed the final (Visit 15/Month 12) efficacy assessments of IEDAT-02-2015.
2.Patient tolerated the study medication, without any evidence of steroid adverse events, or treatment-related severe/ serious adverse events.
3.Body weight > 15 kg.
4.The patient and his/her 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 patient must provide assent to participate in the study.
5.Patient benefitted from treatment with the study medication, or did not worsen significantly, as determined by the Principal Investigator (PI) according to the procedures described below.

Procedure for selecting patients for further treatment in IEDAT-03-2018
•The Principal Investigator will ask all patients who meet the above requirements, and determine their interest in continuing to receive treatment with the study medication in a new protocol. This information will be submitted to the CRO, where an individual will access the study randomization and determine that patient shows evidence of disease stabilization or improvement, as determined by the ratings of the CGI-C.
The CRO will provide the decision on the patient’s eligibility to the PI, within 2 weeks of receipt of the request, without indicating the previous treatment, or the criteria used to determine eligibility.
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Females that are
a.pregnant, or are breast-feeding (for EU countries only);
b.of childbearing potential, pregnant, or are breast-feeding (for US and Rest of World countries).
Females of childbearing potential using adequate birth control, as determined by their Health Care Provider, will be eligible.
2.A disability that may prevent the patient from completing all study requirements.
3.Current participation in a clinical study with another investigational drug.
Medical History and Current Status
4.CD4+ lymphocytes count <400/mm3 (for patients 6 years of age) or <200/mm3 (for patients >6 years).
5.Current neoplastic disease.
6.Severe impairment of the immunological system.
7.Severe or unstable pulmonary disease.
8.Uncontrolled diabetes.
Patients with diabetes that has been stabilized (i.e. no hypoglycemic or hyperglycemic episodes in the past 3 months) will be eligible.
9.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.
10.Eligibility of patients with abnormal laboratory test values will be determined by the Investigator.
11.Confirmed hemoglobinopathies, e.g. hemoglobin C disease, sickle cell anemia, or thalassemia.
12.Moderate or severe renal and/or hepatic impairment.
13.Patients who experienced moderate/ severe steroid side effects, or moderate/ severe adverse events associated with the study medication administered in the IEDAT-02 study.
Prior/Concomitant Medication
14.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 will be permitted.
15.Requires any other concomitant medication prohibited by the protocol.
16.Use of any drug that is a strong inducer/inhibitor of CYP3A4.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified