Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study.

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 155

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 does not present safety contraindications for continuation of treatment, as determined by the Principal Investigator (PI) according to
the procedures described below.
Procedure for selecting patients for further treatment:
The Principal Investigator will ask all patients who meet the aboverequirements, and determine their interest in continuing to receive treatment with the study medication in a new protocol. The Principal Investigator will then determine the eligibility of the patients on the basis of his/her clinical judgement of patients' status and their safety.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 16
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

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 will be eligible. A woman is considered of childbearing potential (WOCBP), i.e.
fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include
hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an
alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a postmenopausal
state in women not using hormonal contraception or hormonal replacement therapy. However in the absence of 12 months of
amenorrhea, a single FSH measurement is insufficient. See Section 11.7
Concomitant Medications for adequate methods of birth control.
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 lifethreatening 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

Primary Outcome Measures

Name	Time	Method
Main Objective: -To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.<br>-To evaluate the long term effect of EDS-EP in treating CNS symptoms as measured by the Modified International Cooperative Ataxia Rating Scale (mICARS), and Clinical Global Impression of severity and change (CGI-S/C);Secondary Objective: To evaluate the long-term effect of EDS-EP on health related Quality of Life (QoL; EQ-5D-5L scale).;Primary end point(s): To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients;Timepoint(s) of evaluation of this end point: During the study, long-term efficacy assessments will be performed every 6 months, while safety parameters will be assessed at each monthly visit.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): To evaluate the long-term effect of EDS-EP on health related Quality of Life (QoL; EQ-5D-5L scale).;Timepoint(s) of evaluation of this end point: Durante lo studio, verranno eseguite valutazioni di efficacia a lungo termine ogni 6 mesi, mentre i parametri di sicurezza saranno valutati a ciascuno<br>visita mensile.

Related Trials

ot Applicable

Phase 1

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Updated 5/13/2024