## Protocol Section
### Identification Module
**NCT ID:** NCT06415344

**Brief Title:** Long-term Extension of GTX-102 in Angelman Syndrome

**Official Title:** A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome

#### Organization Study ID Info
**ID:** GTX-102-CL302
#### Organization
**Class:** INDUSTRY
**Full Name:** Ultragenyx Pharmaceutical Inc
#### Secondary ID Infos
**ID:** 2024-510917-14-00
**Type:** CTIS
### Status Module
#### Completion Date
**Date:** 2029-02

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-16

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-10

**Overall Status:** NOT_YET_RECRUITING

#### Primary Completion Date
**Date:** 2029-02

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-08

**Type:** ESTIMATED

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-16
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-10
**Study First Submit QC Date:** 2024-05-10
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Ultragenyx Pharmaceutical Inc

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Oversight Has DMC:** True

### Description Module

**Brief Summary:** The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

**Detailed Description:** This is a Phase 3 long term extension (LTE) study to evaluate the safety and efficacy of GTX-102 in participants with AS.

Participants may remain in the study until GTX-102 is approved and/or becomes available in their geographical region or the sponsor stops the study.

### Conditions Module

**Conditions:**

- Angelman Syndrome

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 75

**Type:** ESTIMATED

**Phases:**

- PHASE3

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Participants will receive GTX-102 via intrathecal lumbar puncture (IT LP) on a intrapatient flexible dosing schedule.

**Intervention Names:**

- Drug: GTX-102

**Label:** GTX-102

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- GTX-102

**Description:** Antisense Oligonucleotide

**Name:** GTX-102

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Measure:** Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

**Time Frame:** 5 Years

#### Secondary Outcomes

**Measure:** Change from LTE Month 0 and Pretreatment in the Bayley-4 Raw Score

**Time Frame:** Month 0, 5 Years

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* Signed informed consent from parent(s) or legal guardian(s).
* Prior participation in a clinical trial with GTX-102; the timing for the roll-over into this study from a prior GTX-102 study is based on the Investigator and Ultragenyx Medical Monitor's approval.
* From the time of informed consent through the end of the study and for at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102.

Exclusion Criteria:

* Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects.
* Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** trialrecruitment@ultragenyx.com

**Name:** Patients Contact: Trial Recruitment

**Phone:** 1-888-756-8657

**Role:** CONTACT

**Contact 2:**

**Email:** medinfo@ultragenyx.com

**Name:** HCPs Contact: Medical Information

**Phone:** 1-888-756-8657

**Role:** CONTACT

#### Locations

**Location 1:**

**City:** Los Angeles

**Country:** United States

**Facility:** University of California, Los Angeles (UCLA)

**State:** California

**Zip:** 90095

**Location 2:**

**City:** San Diego

**Country:** United States

**Facility:** University of California, San Diego - Rady Children's Hospital

**State:** California

**Zip:** 92123

**Location 3:**

**City:** Atlanta

**Country:** United States

**Facility:** Rare Disease Research, LLC

**State:** Georgia

**Zip:** 30329

**Location 4:**

**City:** Chicago

**Country:** United States

**Facility:** Rush University Medical Center

**State:** Illinois

**Zip:** 60612

**Location 5:**

**City:** Boston

**Country:** United States

**Facility:** Boston Children's Hospital

**State:** Massachusetts

**Zip:** 02115

**Location 6:**

**City:** New York

**Country:** United States

**Facility:** Weill Cornell Medicine

**State:** New York

**Zip:** 10021

**Location 7:**

**City:** South Brisbane

**Country:** Australia

**Facility:** Queensland Children's Hospital

**State:** Queensland

**Zip:** 4101

**Location 8:**

**City:** Heidelberg

**Country:** Australia

**Facility:** Austin Health

**State:** Victoria

**Zip:** 3084

**Location 9:**

**City:** Melbourne

**Country:** Australia

**Facility:** The Royal Children's Hospital

**State:** Victoria

**Zip:** 3052

**Location 10:**

**City:** Calgary

**Country:** Canada

**Facility:** MAGIC Clinic Ltd

**State:** Alberta

**Zip:** T2E 7Z4

**Location 11:**

**City:** Vancouver

**Country:** Canada

**Facility:** British Columbia Children's Hospital

**State:** British Columbia

**Zip:** V6H 3V4

**Location 12:**

**City:** London

**Country:** Canada

**Facility:** Childrens Hospital London Health Sciences Centre

**State:** Ontario

**Zip:** N6A 5W9

**Location 13:**

**City:** Ottawa

**Country:** Canada

**Facility:** Childrens Hospital of Eastern Ontario

**State:** Ontario

**Zip:** K1H 8L1

**Location 14:**

**City:** Montreal

**Country:** Canada

**Facility:** The Research Institute of the McGill University Health Centre

**State:** Quebec

**Zip:** H3G 1A4

**Location 15:**

**City:** Marseille

**Country:** France

**Facility:** AP-HM - Hopital de la Timone

**Zip:** 13385

**Location 16:**

**City:** Paris

**Country:** France

**Facility:** AP-HP Hopital Necker-Enfants Malades

**Zip:** 75015

**Location 17:**

**City:** Hamburg

**Country:** Germany

**Facility:** Universitaetsklinikum Hamburg-Eppendorf

**Zip:** 20246

**Location 18:**

**City:** Leipzig

**Country:** Germany

**Facility:** Universitaetsklinikum Leipzig

**Zip:** 04103

**Location 19:**

**City:** Tel HaShomer

**Country:** Israel

**Facility:** Sheba Medical Center

**Zip:** 5265601

**Location 20:**

**City:** Majadahonda

**Country:** Spain

**Facility:** Hospital Universitario Puerta de Hierro

**Zip:** 28222

**Location 21:**

**City:** Sabadell

**Country:** Spain

**Facility:** Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

**Zip:** 08208

**Location 22:**

**City:** Cambridge

**Country:** United Kingdom

**Facility:** University of Cambridge

**Zip:** CB2 0QQ

**Location 23:**

**City:** London

**Country:** United Kingdom

**Facility:** Great Ormond Street Hospital for Children

**Zip:** WC1N 3JH

**Location 24:**

**City:** Oxford

**Country:** United Kingdom

**Facility:** Oxford University Hospitals NHS Foundation Trust

**Zip:** OX3 9DU

#### Overall Officials

**Official 1:**

**Affiliation:** Ultragenyx Pharmaceuticals Inc.

**Name:** Medical Director

**Role:** STUDY_DIRECTOR

### IPD Sharing Statement Module

**IPD Sharing:** NO

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000004194

- Term: Disease

- ID: D000010335

- Term: Pathologic Processes

- ID: D000009069

- Term: Movement Disorders

- ID: D000002493

- Term: Central Nervous System Diseases

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000000015

- Term: Abnormalities, Multiple

- ID: D000000013

- Term: Congenital Abnormalities

- ID: D000025063

- Term: Chromosome Disorders

- ID: D000030342

- Term: Genetic Diseases, Inborn

- ID: D000096803

- Term: Imprinting Disorders

### Condition Browse Module - Browse Branches

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: All

- Name: All Conditions

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M16355

- Name: Syndrome

- Relevance: HIGH

- As Found: Syndrome

- ID: M19508

- Name: Angelman Syndrome

- Relevance: HIGH

- As Found: Angelman Syndrome

- ID: M12029

- Name: Movement Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M5742

- Name: Central Nervous System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12

- Name: Congenital Abnormalities

- Relevance: LOW

- As Found: Unknown

- ID: M14

- Name: Abnormalities, Multiple

- Relevance: LOW

- As Found: Unknown

- ID: M23023

- Name: Chromosome Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M23686

- Name: Genetic Diseases, Inborn

- Relevance: LOW

- As Found: Unknown

- ID: T389

- Name: Angelman Syndrome

- Relevance: HIGH

- As Found: Angelman Syndrome

### Condition Browse Module - Meshes

- ID: D000017204

- Term: Angelman Syndrome

- ID: D000013577

- Term: Syndrome

### Misc Info Module

- Version Holder: 2024-05-31
