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Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS (Aspire) (GTX-102-CL301)

ASPIRE (GTX-102-CL301)

HCPs Contact Medical Information

Ultragenyx Pharmaceutical Inc

60 Leveroni Court, Novato, CA 94949 USA

medinfo@ultragenyx.com

Worldwide Clinical Trials Japan K.K.

Worldwide Clinical Trials Japan K.K., Clinical Operations Site Management

Shinagawa Intercity, Tower A, Level 28, 2-15-1, Konan, Minato-ku

+81-3-6717-4360

yuriko-mae@cmic.co.jp

Not Recruiting

May. 27, 2025

Other

randomized controlled trial

double blind

no treatment control/standard of care control

parallel assignment

treatment purpose

- 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 assistance at the Screening Visit (note, a child whose primary means of mobility is by wheelchair is excluded from the study)
- Platelet count, prothrombin time / international normalized ratio, and partial thromboplastin time within 1.5x the normal limits at the Screening Visit
- Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, and all study procedures, including LP procedure and tolerating anesthesia without intubation

- Any change in medications or diet/supplements intended to treat symptoms of AS (eg, sleeping aids, antiseizure medications, supplements, dietary change including ketogenic or low-glycemic index diet, other) within the month prior to the Screening Visit (excluding weight-based adjustments)
- Any condition that creates an increased risk of unsuccessful LP
- Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors)
- Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results
-human immunodeficiency virus (HIV) antibodypositive,hepatitis B (HBs) antigen-positive, HBs or HBc antibody-positive, or TB
positive by laboratory testing at the Screening Visit
- Use of any investigational product or investigational medical device within 6 months or 5 half-lives prior to the Screening Visit or any prior use of gene therapy or ASO regardless of duration since last administration
- Concurrent participation in any study, including observational natural history studies

Both

Angelman Syndrome

GTX-102
Dose Formulation 2.0mg/mL vial
Unit Dose Strength(s)/Dosage Level(s) 8mg to 14 mg
Route of Administration IT injection via LP

Lumber puncture

D017204

D013129

the change from baseline to Day 338 in Bayley-4 Cognitive raw score (without caregiver input) for the GTX-102 group compared with the Sham-LP group

Net response in the MDRI at Day 338

Feb. 18, 2025

Feb. 18, 2025

Feb. 18, 2025

No

Canada/Germany/Poland/Spain/United States