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ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to <36 months (Part 1A), aged ≥48 months to <18 years (Part 1B), and aged ≥6 to <48 months (Part 2). Part 1A follows an open-label, dose-escalation design, Part 1B follows an open-label design, and Part 2 is a randomized, double-blind, sham delayed-treatment control study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part 1 (US Only) | Experimental | Part 1A will follow an open-label, rule-based, dose-escalation design and will evaluate up to 4 dose levels of ETX101. Part 1B will follow an open-label design and will evaluate 1 dose level of ETX101. |
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| Part 2 | Sham Comparator | Part 2 will follow a double-blind (up through Week 52), randomized, sham delayed-treatment control design. There will be 2 cohorts in Part 2. A single dose level of ETX101 will be evaluated in Part 2 and participants will be randomized 2:1 to study treatment or sham procedure with delayed treatment. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ETX101 | Drug | ETX101 is a non-replicating, recombinant adeno-associated viral vector serotype 9 (rAAV9) comprising a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A). ETX101 is intended as a one-time intracerebroventricular (ICV) administration. |
| 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 (Key Secondary Endpoint for Part 2). | From Baseline to Week 52. | |
| Change from Baseline in Vineland-3 subdomain GSVs at Week 52. | From Baseline to Week 52. |
| Measure | Description | Time Frame |
|---|---|---|
| Safety Endpoint: Proportions of participants experiencing any treatment-emergent DLTs (for Part 1A only), 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 | Role | Phone | Extension | |
|---|---|---|---|---|
| Encoded Patient Advocacy | Contact | +1 (650) 398-4301 | patientadvocacy@encoded.com |
| Name | Affiliation | Role |
|---|---|---|
| Salvador Rico, M.D., Ph.D | Encoded Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospitals | Recruiting | San Francisco | California | 94158 | United States |
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Part 1A and Part 1B are open-label with no blinding.
Part 2 will be conducted in a double-blinded manner whereby all Primary Site Staff (including clinicians, research coordinators, neuropsychologists, pharmacists, and physical therapists), study participants and caregivers, Sponsor and Sponsor-designees will be blinded through the end of Week 52 following Day 1 for each participant. The Surgical Site Staff will be unblinded to treatment assignment.
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| Change from Baseline in Bayley-4 subdomain GSVs at Week 52. | From 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. | From Baseline to Week 52. |
| Change from Baseline in Vineland-3 subdomain raw scores at Week 52. | From Baseline to Week 52. |
| Change from Baseline in Bayley-4 subdomain raw scores (excluding cognitive subdomain) at Week 52. | From 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. | From 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. | From Baseline to Week 52. |
| From Day 1 through Study Completion. |
| Safety Endpoint: Overall survival. | From Day 1 through Study Completion. |
| Colorado Children's Hospital | Recruiting | Aurora | Colorado | 80045 | United States |
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| Nicklaus Children's Hospital | Recruiting | Miami | Florida | 33155 | United States |
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| Ann & Robert H. Lurie Children's Hospital of Chicago | Recruiting | Chicago | Illinois | 60611 | United States |
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| Boston Children's Hospital | Not yet recruiting | Boston | Massachusetts | 02115 | United States |
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| Mott Children's Hospital | Recruiting | Ann Arbor | Michigan | 48109 | United States |
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| Mayo Clinic | Recruiting | Rochester | Minnesota | 55905 | United States |
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| Duke Children's Hospital & Health Center | Not yet recruiting | Durham | North Carolina | 27705 | United States |
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| Nationwide Children's Hospital | Not yet recruiting | Columbus | Ohio | 43205 | United States |
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| Oregon Health and Science University (OSHU) | Recruiting | Portland | Oregon | 97239 | United States |
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| Cook Children's Medical Center | Recruiting | Fort Worth | Texas | 76104 | United States |
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| The Royal Children's Hospital | Recruiting | Melbourne | Australia |
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| Queen Elizabeth Hospital | Recruiting | Glasgow | G51 4TF | United Kingdom |
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| Great Ormond Street Hospital | Not yet recruiting | London | WC1N3JH | United Kingdom |
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| ID | Term |
|---|---|
| D004831 | Epilepsies, Myoclonic |
| C565810 | Generalized Epilepsy With Febrile Seizures Plus, Type 2 |
| 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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