E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patient with neurological symptoms of Ataxia Telangiectasia |
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E.1.1.1 | Medical condition in easily understood language |
Patient with neurological symptoms of Ataxia Telangiectasia |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10003594 |
E.1.2 | Term | Ataxia telangiectasia |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
•To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.
•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) |
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E.2.2 | Secondary objectives of the trial |
To evaluate the long-term effect of EDS-EP on health related Quality of Life (QoL; EQ-5D-5L scale). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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 above requirements, 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.
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E.4 | Principal 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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients |
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E.5.1.1 | 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. |
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E.5.2 | Secondary end point(s) |
To evaluate the long-term effect of EDS-EP on health related Quality of Life (QoL; EQ-5D-5L scale). |
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E.5.2.1 | 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. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 4 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Belgium |
Germany |
India |
Israel |
Poland |
Spain |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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LPLV
The open-label treatment period is planned for 12 months, but may be extended further and continue until patients eventually withdraw consent or the Investigator decides to discontinue treatment based on a risk/ benefit assessment |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |