Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-… (NCT03563053) | Clinical Trial Compass
TerminatedPhase 3
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
Stopped: terminated by sponsor
United States, Australia104 participantsStarted 2018-06-12
Plain-language 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).
Who can participate
Age range6 Years
SexALL
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Inclusion criteria
✓. 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.
✓. Patient tolerated the study medication, without any evidence of steroid adverse events, or treatment-related severe events / serious adverse events.
✓. Body weight \> 15 kg.
✓. 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.
✓. Patient did not present safety contraindication for continuation of treatment, as determined by the Principal Investigator (PI) according to the procedures described below.
Exclusion criteria
✕. Females that were:
✕. Pregnant, or were breast-feeding (for EU countries only)
✕
What they're measuring
1
Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study
Timeframe: From Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months)
. Of childbearing potential, pregnant, or were breast-feeding (for US and Rest of World countries).
✕. A disability that may prevent the patient from completing all study requirements.
✕. Current participation in another clinical study with another investigational drug.
✕. 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).
✕. Current neoplastic disease.
✕. Severe impairment of the immunological system.