Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
GTX-102 · 4 trials · 1 indication
The following assessments will be included to calculate the MDRI net response: Bayley-4 Cognitive and Receptive Communication, Aberrant Behavior Checklist- Community (ABC-C) Hyperactivity/Noncompliance (H/N), Angelman Severity Assessment (ASA) Sleep, ASA Gross Motor. For each assessment a meaningful score difference (MSD) is defined. A single net response score per participant will be derived accordingly, and a summary measure of net response will then be calculated across all participants.
The following assessments will be included to calculate the MDRI net response: Vineland-3 Expressive and Receptive Communication, ABC-C Irritability, ASA Gross Motor. For each assessment a meaningful score difference (MSD) is defined. A single net response score per participant will be derived accordingly, and a summary measure of net response will then be calculated across all participants.
| Arm | Type | Description |
|---|---|---|
| GTX-102 | EXPERIMENTAL | Participants will receive GTX-102 via lumbar puncture (LP) during both the double-blind and open-label period |
| Sham-LP then GTX-102 | SHAM_COMPARATOR | Participants will receive sham procedure during the double-blind period and then will receive GTX-102 via LP during the open-label period |
| Subprotocol A GTX-102 | EXPERIMENTAL | Participants with deletion-type Angelman syndrome, ≥1 to \<4 years of age will receive increasing doses of GTX-102 via intrathecal (IT) injection until the target dose is achieved. Dosing occurs every 3 months (Q3M) thereafter. |
| Subprotocol B GTX-102 | EXPERIMENTAL | Participants with paternal uniparental disomy (UPD)/imprinting center defect (ICD) Angelman syndrome, ≥4 to \<18 years of age will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter. |
| Subprotocol C GTX-102 | EXPERIMENTAL | Participants with all genotypes of Angelman syndrome, ≥18 to \<65 years of age will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter. |
| Subprotocol D GTX-102 | EXPERIMENTAL | Participants with mutation-type Angelman syndrome, ≥4 to \<18 years of age will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter. |
| Subprotocol D No Intervention then GTX-102 | EXPERIMENTAL | Participants with mutation-type Angelman syndrome, ≥4 to \<18 years of age will receive no treatment during the initial period. At the end of the no treatment period, participants will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter. |
| GTX-102 Cohort 1 | EXPERIMENTAL | 3.3 mg starting dose followed by intra-patient dose escalation up to 36 mg and then a maintenance phase (in U.S participants 4 to \<17 years of age) |
| GTX-102 Cohort 2 | EXPERIMENTAL | 10 mg starting dose followed by intra-patient dose escalation up to 36 mg and then a maintenance phase (in U.S participants 4 to \<17 years of age) |
| GTX-102 Cohort 3 | EXPERIMENTAL | 20 mg starting dose followed by intra-patient dose escalation up to 55 mg and then a maintenance phase (in U.S participants 4 to \<17 years of age) |
| GTX-102 Cohort 4 | EXPERIMENTAL | 3.3 mg starting dose followed by slow intra-patient dose escalation up to 5 mg and then a maintenance phase (in Ex-U.S participants 4 to \<8 years of age) |
| GTX-102 Cohort 5 | EXPERIMENTAL | 5 mg starting dose followed by slow intra-patient dose escalation up to 7.5 mg and then a maintenance phase (in Ex-U.S participants ≥ 8 to 17 years of age) |
| GTX-102 Cohort 6 | EXPERIMENTAL | 7.5 mg starting dose followed by slow intra-patient dose escalation up to 10 mg and then a maintenance phase (in Ex-U.S participants 4 to \<8 years of age) |
| GTX-102 Cohort 7 | EXPERIMENTAL | 10 mg starting dose followed by slow intra-patient dose escalation up to 12 mg and then a maintenance phase (in Ex-U.S participants ≥ 8 to 17 years of age) |
| GTX-102 Cohort US | EXPERIMENTAL | 2 mg for 4 monthly doses followed by a quarterly maintenance regimen |
| GTX-102 Expanded Enrollment Cohort A | EXPERIMENTAL | Sponsor selected dose followed by slow intra-patient dose escalation and then a maintenance phase (in Ex-U.S participants 4 to \<8 years of age) |
| GTX-102 Expanded Enrollment Cohort B | EXPERIMENTAL | Sponsor selected dose followed by slow intra-patient dose escalation and then a maintenance phase (in Ex-U.S participants ≥ 8 to 17 years of age) |
| GTX-102 Expanded Enrollment Cohort C | EXPERIMENTAL | Sponsor selected dose followed by slow intra-patient dose escalation and then a maintenance phase (in U.S participants 4 to \<8 years of age) |
| GTX-102 Expanded Enrollment Cohort D | EXPERIMENTAL | Sponsor selected dose followed by slow intra-patient dose escalation and then a maintenance phase (in U.S participants ≥ 8 to 17 years of age) |
| GTX-102 Cohort E | EXPERIMENTAL | Sponsor selected dose followed by slow intra-patient dose escalation and then a maintenance phase (in participants that transition from GTX-102 US Cohort only) |
| Name | Type | Description |
|---|---|---|
| GTX-102 | DRUG | antisense oligonucleotide |
| Sham-LP | PROCEDURE | Small needle prick on the lower back at the location where the LP injection is normally made |
| No intervention | OTHER | During the no treatment period participants do not receive any study drug |
Key Inclusion Criteria: * Signed informed consent from parent(s) or legal guardian(s) * Confirmed diagnosis of AS with genetic confirmation of full maternal ubiquitin-protein ligase E3A (UBE3A) gene deletion causing AS in the region of 15q11.2 q13 * Able to ambulate independently, or with assistanc...
GTX-102 is an investigational small molecule being developed for the treatment of Angelman Syndrome, a rare genetic disorder. It is currently in Phase 3 clinical development and has not yet been approved by the FDA. The drug is being studied in pediatric patients with this condition.
GTX-102 is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol RARE. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy for Angelman Syndrome.
GTX-102 is currently in Phase 3 clinical development for Angelman Syndrome. It has received Breakthrough Therapy designation from the FDA. The drug is investigational and has not been approved for commercial use. Clinical trials are ongoing to assess its safety and efficacy.
GTX-102 is being studied in four clinical trials. These include a completed Phase 1 study (NCT04259281), a Phase 3 long-term extension study (NCT06415344), a Phase 3 efficacy and safety study (NCT06617429), and a Phase 2 study in deletion- and nondeletion-type Angelman Syndrome (NCT07157254).
No, GTX-102 is not FDA approved. It is an investigational drug currently in Phase 3 clinical trials for Angelman Syndrome. The FDA has granted it Breakthrough Therapy designation, which is intended to expedite development and review, but the drug remains in clinical development.