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EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to < 48 months. The study follows and open-label, dose-escalation design.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort A | Experimental | Cohort A will evaluate ETX101 dose level 1. |
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| Cohort B | Experimental | Cohort B will evaluate ETX101 dose level 2. |
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| Cohort C | Experimental | Cohort C will evaluate ETX101 dose level 3. |
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| Cohort D | Experimental | Cohort D will evaluate ETX101 dose level 4. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ETX101 | Drug | ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A) |
| Measure | Description | Time Frame |
|---|---|---|
| Percent change in monthly countable seizure frequency (MCSF) between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period. | Between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period (defined as Week 5 to Week 52) |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Bayley-4 cognitive subdomain raw score at Week 52 | Baseline to Week 52 | |
| Change from Baseline in Vineland-3 subdomain GSVs at Week 52. | Baseline to Week 52. | |
| Measure | Description | Time Frame |
|---|---|---|
| Safety Endpoint: Proportions of participants experiencing any treatment-emergent DLTs, AEs, serious adverse events (SAEs), related AEs, AEs with severity Grade ≥ 3, AEs resulting in study discontinuation, and AEs resulting in death. | From Day 1 through Study Completion. | |
| Safety Endpoint: Proportion of participants experiencing SAEs leading to hospitalization. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Salvador Rico, M.D., Ph. D | Encoded Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Queen Elizabeth Hospital | Glasgow | G51 4TF | United Kingdom | |||
| Great Ormond Street Hospital |
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| Change from Baseline in Bayley-4 subdomain GSVs at Week 52. |
| Baseline to Week 52. |
| Proportion of participants achieving ≥ 75% reduction in MCSF between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period. | Between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period (defined as Week 5 to Week 52). |
| Proportion of participants achieving ≥ 50% reduction in MCSF between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period. | Between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period (defined as Week 5 to Week 52). |
| Change from Baseline in the Vineland-3 Adaptive Behavior Composite standard score at Week 52. | Baseline to Week 52. |
| Change from Baseline in Vineland-3 subdomain raw scores at Week 52. | Baseline to Week 52. |
| Change from Baseline in Bayley-4 subdomain raw scores (excluding cognitive subdomain) at Week 52. | Baseline to Week 52. |
| Proportion of CGI-I responders, defined as participants with a CGI-I score of 1 (Very much improved) or 2 (Much improved), at Week 52. | Baseline to Week 52. |
| Proportion of CGI-S responders, defined as participants who either have a CGI-S score of 1 (Normal, not at all ill) or demonstrate a ≥2 point improvement from Baseline, at Week 52. | Baseline to Week 52. |
| From Day 1 through Study Completion. |
| Safety Endpoint: Overall survival. | From Day 1 through Study Completion. |
| London |
| WC1N3JH |
| United Kingdom |
| Sheffield Children's Hospital | Sheffield | S10 2TH | United Kingdom |
| ID | Term |
|---|---|
| D004831 | Epilepsies, Myoclonic |
| ID | Term |
|---|---|
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D000073376 | Epileptic Syndromes |
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