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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03563053
Registration number
NCT03563053
Ethics application status
Date submitted
23/05/2018
Date registered
20/06/2018
Date last updated
8/10/2024
Titles & IDs
Public title
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
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Scientific title
Open-label, Long-term, Extension Treatment Using Intra-Erythrocyte Dexamethasone Sodium Phosphate (EryDex System) in Patients With Ataxia Telangiectasia Who Participated in the ATTeST-IEDAT-02-2015 Study
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Secondary ID [1]
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2018-000338-36
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Secondary ID [2]
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IEDAT-03-2018
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Universal Trial Number (UTN)
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Trial acronym
OLE-IEDAT
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Ataxia Telangiectasia
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Genetic Syndrome
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Condition category
Condition code
Neurological
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Other neurological disorders
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Neurological
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Neurodegenerative diseases
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Cardiovascular
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Diseases of the vasculature and circulation including the lymphatic system
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - EryDex System
Experimental: active drug - \~14-22 mg dexamethasone sodium phosphate (DSP) for 12 months. The duration of EryDex treatment could be extended further and continue until patients eventually withdrew consent, or the Investigator decided to discontinue treatment based on a risk / benefit assessment.
Other interventions: EryDex System
EryDex System was a combination product that was used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EDS) creating the EDS-end product which was infused into the patients.
EryDex treatment consisted of a dose range correspondent to the ATTeST High Dose (obtained by loading 125 mg of DSP to the EryDex process and that, in the ATTeST, resulted in a mean of 17.4 ± 5.4 mg (mean ± standard deviation) of DSP infused to patients) administered via ex vivo encapsulation into EDS that were infused into the patient with A-T.
The dose of EryDex treatment was selected to allow collection of long-term safety data on the dose of erythrocyte encapsulated DSP that was considered more effective among the two different doses that were employed in the ATTeST study, based on the analysis of the ATTeST blinded, Phase 3 study data.
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
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Assessment method [1]
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Assessment of TEAEs, treatment-emergent serious adverse events (TESAE), and adverse events of special interest (AESI) were performed throughout the study, from the time of signing of the ICF at Baseline Visit through to the Final Study Visit (Month 12 or early discontinuation). All patients were to be followed up through 30 days after the Final Visit (Month 12 or early discontinuation) or at least 60 days after the final infusion, whichever was longer.
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Timepoint [1]
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From Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months)
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Secondary outcome [1]
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Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36
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Assessment method [1]
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The EQ-5D included single item measures of 5 health dimensions: * mobility, * self-care, * usual activities, * pain / discomfort, and * anxiety / depression. The EQ-5D-5L included five levels of severity (i.e., no problems, slight problems, moderate problems, severe problems, and extreme problems) for each of the five EQ-5D dimensions. These levels were scored from 1 = no problems to 5 = extreme problems: from 5, min/worst, to 25, best/max).The EQ-5D results were converted into a continuous index score: as for this index score, the closer the value is to 1, the better is the health status. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations.
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Timepoint [1]
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From Baseline (Visit 1- Day 0) to Month 36
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Secondary outcome [2]
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Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36
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Assessment method [2]
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The CGI-C scale assesses the change in the patient's clinical status from baseline using a 7-point scale, ranging from 1 (very much improved) to 7 (very much worse), with a score of 4 indicating no change. Clinicians were required to conduct a full clinical interview and examination of the patient. The interview and examination assessed various aspects of the patient's appearance (grooming, evidence of falls, etc.), ataxia, cognition (orientation, calculation ability, language, ability to follow commands, memory, etc.), apraxia, dysarthria, extrapyramidal motor symptoms, activities of daily living, and mood. The higher the score the worse the outcome. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations.
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Timepoint [2]
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From Baseline (Visit 1- Day 0) to Month 36
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Secondary outcome [3]
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Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT From Baseline (Visit 1 - Day 0) to Month 36
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Assessment method [3]
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The CGI-S scale measures global severity of illness at a given point in time, and is usually rated on a 7-point, Likert-type scale ranging from 1 (normal, not ill at all) to 7 (among the most extremely ill patients). No version of the CGI-S exists which has been specifically adapted for use in patients with A-T; therefore, a 5-point version was developed that considered the severity of the following symptoms of A-T: ataxia (walking), dysarthria, dysmetria, extrapyramidal symptoms (chorea, myoclonus, dystonia, and tremor), and eye movements. Ratings of none (0), mild (1), moderate (2), severe (3), and very severe (4) were selected based on the level of symptomatology. The higher the score the worse the outcome. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations.
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Timepoint [3]
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From Baseline (Visit 1- Day 0) to Month 36
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Secondary outcome [4]
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Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36
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Assessment method [4]
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The International Cooperative Ataxia Rating Scale (ICARS) was an assessment of the degree of impairment in patients with cerebellar ataxia and was administered in its entirety; however, the primary efficacy assessment was based on the modified (m)ICARS, which excluded the Oculomotor domain (items 17 to 19) and items 8 to 12 of the Kinetic Functions domain of the ICARS. The mICARS was a 54 points maximum score (min 0) questionnaire divided into 3 sections: * Posture and Gait Disturbance section-7 items (min score 0, max score 34) * Kinetic Function-2 items (min 0, max 12) * Speech Disorder- 2 items (min 0, max 8). An higher scores - both for total and subscores - indicate a higher level of disease impairment. The subscores are added to give the total score. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations.
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Timepoint [4]
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From Baseline (Visit 1- Day 0) to Month 36
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Eligibility
Key inclusion criteria
1. Patient completed the double-blind period in the ATTeST study and completed the final (Visit 15 / Month 12) Efficacy Assessments of ATTeST or discontinued the study during the COVID-19 pandemic.
2. Patient tolerated the study medication, without any evidence of steroid adverse events, or treatment-related severe events / 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, provided written informed consent to participate. If consent was provided solely by the caregiver in accordance with local regulations, the patient also was asked to provide their assent to participate in the study.
5. Patient did not present safety contraindication for continuation of treatment, as determined by the Principal Investigator (PI) according to the procedures described below.
Moreover, patients who were discontinued from the ATTeST study during the COVID-19 pandemic were eligible to receive the EryDex treatment in the IEDAT-03-2018 study, in the absence of safety contraindications to continuation of the treatment, and after signing the informed consent.
There were no de novo enrolled patients.
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Minimum age
6
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
Patients who met one or more of the following criteria were not considered to be eligible to participate in the study:
General
1. Females that were:
1. Pregnant, or were breast-feeding (for EU countries only)
2. Of childbearing potential, pregnant, or were breast-feeding (for US and Rest of World countries).
Females of childbearing potential using adequate birth control, as determined by their Health Care Provider, were eligible.
2. A disability that may prevent the patient from completing all study requirements.
3. Current participation in another clinical study with another investigational drug.
Medical History and Current Status
4. Cluster differential 4 positive (CD4+) lymphocytes count < 400 / mm3 (for patients 6 years of age) or < 150 / mm3 (for patients > 6 years). In presence of oral infections, like oral candidiasis, documented at the screening or recurrent as per medical history documentation, the limit increases to < 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 had been stabilized (i.e., no hypoglycemic or hyperglycemic episodes in the past 3 months) were 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 were determined by the Investigator.
11. Confirmed haemoglobinopathies, e.g., haemoglobin C disease, sickle cell anaemia, 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 EryDex treatment administered in the ATTeST 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 were permitted.
15. Requires any other concomitant medication prohibited by the protocol.
16. Use of any drug that is a strong inducer / inhibitor of Cytochrome P450 3A4 (CYP3A4).
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Not applicable
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
12/06/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
2/09/2022
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Sample size
Target
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Accrual to date
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Final
104
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Department of Neurology Royal Children's Hospital - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment outside Australia
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United States of America
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State/province [1]
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California
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United States of America
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Maryland
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United States of America
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Ohio
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United States of America
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Texas
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Belgium
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Leuven
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Germany
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State/province [6]
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Frankfurt
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Country [7]
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India
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State/province [7]
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Tamil Nadu
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India
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Bangalore
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India
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Hyderabad
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India
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Mumbai
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Italy
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Roma
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Norway
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Oslo
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Poland
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Warsaw
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Spain
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State/province [14]
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Madrid
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Country [15]
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Tunisia
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State/province [15]
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Tunis
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Country [16]
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United Kingdom
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State/province [16]
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Nottinghamshire
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Quince Therapeutics S.p.A.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Primary Objective To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients. Secondary Objective To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale). Exploratory Objective: To evaluate the long-term effect of EDS-EP in treating central nervous system (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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Trial website
https://clinicaltrials.gov/study/NCT03563053
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Guenter R Janhofer, MD, PhD
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Address
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EryDel S.p.A.
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Irene Maccabruni, BS
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Address
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Country
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Phone
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+393458415028
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/53/NCT03563053/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/53/NCT03563053/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT03563053
Download to PDF