Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02770807 | Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients | PHASE3 | COMPLETED | 176 | — | — | Mar 2, 2017 | May 13, 2021 | May 10, 2024 | 22 | United States, Australia +10 |
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 score 0, max score 12) * Speech Disorder- 2 items (min score 0, max score 8). The assessment was designed to be completed within 30 minutes, and higher scores - both for total and subscores - indicate a higher level of disease impairment. The subscores are added to give the total score.
| Arm | Type | Description |
|---|---|---|
| EryDex Low Dose DSP | EXPERIMENTAL | EDS-EP dose range of \~5-10 mg DSP/infusion. This study treatment was to be administered by IV infusion once per month for 12 consecutive months (6 months Initial Treatment Period + 6 months Extension Treatment Period). Each patient underwent a 30-day Screening Period, during which any previous treatments with other corticosteroid compounds were withdrawn (washout from previous treatment). Other Names: EryDex System end product Low dose DSP |
| EryDex High Dose DSP | EXPERIMENTAL | EDS-EP dose range of \~14-22 mg DSP/infusion. This study treatment was to be administered by IV infusion once per month for 12 consecutive months (6 months Initial Treatment Period + 6 months Extension Treatment Period). Each patient underwent a 30-day Screening Period, during which any previous treatments with other corticosteroid compounds were withdrawn (washout from previous treatment). Other Names: EryDex System end product High dose DSP |
| Placebo | PLACEBO_COMPARATOR | Patients were treated with autologous erythrocytes prepared with the EDS process using a placebo solution. Placebo was to be administered by IV infusion once per month for 12 consecutive months (6 months Initial Treatment Period + 6 months Extension Treatment Period). Each patient underwent a 30-day Screening Period, during which any previous treatments with other corticosteroid compounds were withdrawn (washout from previous treatment). |
| Name | Type | Description |
|---|---|---|
| EryDex Low dose DSP | DRUG | EDS-EP dose range of \~5-10 mg DSP/infusion |
| EryDex High dose DSP | DRUG | EDS-EP dose range of \~14-22 mg DSP/infusion |
| Pooled Placebo | DRUG | EDS processed autologous erythrocytes using a sodium chloride \[NaCl\] solution. |
Inclusion Criteria: 1. 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 sys...