Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
United States, Australia176 participantsStarted 2017-03-02
Plain-language summary
Objectives:
The objective of study was to evaluate the safety and the efficacy of EryDex (Dexamethasone sodium phosphate encapsulated in autologous erythrocytes, using the EryDex System - EDS) at two dose levels (low dose and high dose DSP/infusion), compared to placebo, on Neurological Symptoms in Patients With Ataxia Telangiectasia.
Initial Double-Blind Treatment Period (0 to 6 Months)
Primary Efficacy Objective:
• Evaluate the effect of EryDex at two dose levels (low dose and high dose DSP/infusion), compared to placebo, on central nervous system (CNS) symptoms measured by the change in the Modified International Cooperative Ataxia Rating Scale (mICARS) from baseline to Month 6 (Visit 9) in patients with ataxia telangiectasia (A-T).
Secondary Efficacy Objectives:
* Evaluate the effect of EryDex, compared to placebo, on the Clinical Global Impression of Change (CGI-C) in patients with A-T from baseline to Month 6 (Visit 9).
* Evaluate the effect of EryDex, compared to placebo, on measures of Clinical Global Impression of Severity (CGI-S; structured) in patients with A-T from baseline to Month 6 (Visit 9)
* Evaluate the effect of EryDex, compared to placebo, on measures of Adaptive behavior measures in patients with A-T by the Vineland Adaptive Behavior Scales (VABS) from baseline to Month 6 (Visit 9).
Safety Objectives:
• Evaluate the safety and tolerability of two non-overlapping doses of EryDex, compared to placebo, in patients with A-T over the 12-month double-blind study duration.
Extension Treatment Period (6-12 Months):
Primary Objective:
• Evaluate the efficacy of EryDex at two dose levels (low dose and high dose DSP/infusion) compared to placebo, in treating CNS symptoms in A-T patients during longer-term treatment (up to 12 months), as measured by the mICARS.
Secondary Objectives:
* Evaluate the longer-term (up to 12 months) safety and tolerability of EryDex in A-T patients.
* Compare the effects of EryDex on the CGI-C and CGI-S (structured), VABS, and QoL using the EQ-5D-5L scale.
Who can participate
Age range6 Years
SexALL
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Inclusion criteria
✓. Patient met clinical criteria for diagnosis of A-T. The neurological signs of A-T (incoordination of the head and eyes in lateral gaze deflection, gait ataxia associated with an inappropriately narrow base) must have been documented. Such signs of A-T illustrated the body systems in which changes were confirmed, but the listed changes were examples and other changes in those systems may have been observed and documented to confirm the diagnosis of A-T.
✓. Patient was in autonomous gait or was helped by periodic use of a support (i.e., score for Item 1 of the full ICARS - Walking Capacities between 0 and 4, included).
✓. Patient was investigated for the proven genetic diagnosis of A-T (prior documentation or by central laboratory test report).
✓. Patient was at least 6 years of age.
✓. Body weight was \>15 kg.
✓. The patient and 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 must have provided assent to participate in the study.
Exclusion criteria
What they're measuring
1
Change From Baseline in Modified International Cooperative Ataxia Rating Scale (mICARS)
✕. Pregnant or breast-feeding (for European Union \[EU\] countries only).
✕. Of childbearing potential, pregnant, or breast-feeding (for US and Rest of World countries) not using adequate birth control, as determined by their Healthcare Provider.
✕. A disability that may have prevented the patient from completing all study requirements.
✕. Current participation in another clinical study.
✕. 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 increased to \<200/mm3 (for patients \>6 years).
✕. Loss/removal of 250 mL or more of blood within the past 4 weeks prior to screening.
✕. Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years.