## Protocol Section
### Identification Module
**NCT ID:** NCT06420297

**Brief Title:** OLE Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

**Official Title:** A Long-term, Open-label Extension Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

#### Organization Study ID Info
**ID:** ACP-101-303
#### Organization
**Class:** INDUSTRY
**Full Name:** ACADIA Pharmaceuticals Inc.
#### Secondary ID Infos
**ID:** 2023-506201-19-00
**Type:** CTIS
### Status Module
#### Completion Date
**Date:** 2029-06

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-20

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-14

**Overall Status:** ENROLLING_BY_INVITATION

#### Primary Completion Date
**Date:** 2029-05

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-03-11

**Type:** ACTUAL

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-20
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-07
**Study First Submit QC Date:** 2024-05-14
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** ACADIA Pharmaceuticals 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:** To evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS

**Detailed Description:** This is a long-term, OLE study to evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS. Subjects who complete the antecedent double-blind study (ACP-101-302) will be invited to participate in the present study.

### Conditions Module

**Conditions:**

- Hyperphagia in Prader-Willi Syndrome

**Keywords:**

- Opel-label extension

- safety

- tolerability

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 160

**Type:** ESTIMATED

**Phases:**

- PHASE3

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Carbetocin nasal spray 3.2 mg three times daily (TID)

**Intervention Names:**

- Drug: Carbetocin

**Label:** Drug: Carbetocin

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Drug: Carbetocin

**Description:** Carbetocin nasal spray 3.2 mg three times daily (TID)

**Name:** Carbetocin

**Other Names:**

- ACP-101

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** Treatment-emergent adverse events (TEAEs) and device incidents or device malfunctions, TEAEs leading to discontinuation, TEAEs related to study drug, TEAEs by maximum severity, fatal TEAEs, treatment-emergent SAEs, and treatment-emergent SAEs related to study drug will all be summarized for all subjects as well as by previous treatment group.

**Measure:** Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to adverse events (AEs), potentially clinically important changes in other safety assessments, device incidents or device malfunctions

**Time Frame:** Baseline to 36 months

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* Has completed the Week12/EOT visit of the antecedent, Study ACP-101-302
* Met all entry criteria for the antecedent study
* May benefit from long-term treatment with open-label carbetocin in the judgment of the Investigator.
* Lives with a caregiver who understands and is willing and able to adhere to study-related procedures and is willing to participate in all study visits.

Exclusion Criteria:

* History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary.
* Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder
* History of suicide attempt or inpatient psychiatric hospitalization
* Has a clinically significant abnormality in vital signs at Baseline
* Has an average QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of carbetocin is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study)
* Has developed a clinically significant ECG finding during the antecedent study
* Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study

Additional inclusion/exclusion criteria apply. Subjects will be evaluated at screening to ensure that all criteria for study participation are met.

**Maximum Age:** 30 Years

**Minimum Age:** 5 Years

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Nashville

**Country:** United States

**Facility:** Vanderbilt Clinical Research Center

**State:** Tennessee

**Zip:** 37232

### IPD Sharing Statement Module

**IPD Sharing:** NO

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000004194

- Term: Disease

- ID: D000010335

- Term: Pathologic Processes

- ID: D000008607

- Term: Intellectual Disability

- ID: D000019954

- Term: Neurobehavioral Manifestations

- ID: D000009461

- Term: Neurologic Manifestations

- 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

- ID: D000009765

- Term: Obesity

- ID: D000050177

- Term: Overweight

- ID: D000044343

- Term: Overnutrition

- ID: D000009748

- Term: Nutrition Disorders

- ID: D000012817

- Term: Signs and Symptoms, Digestive

### 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: BC18

- Name: Nutritional and Metabolic Diseases

- Abbrev: BXM

- Name: Behaviors and Mental Disorders

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M16355

- Name: Syndrome

- Relevance: HIGH

- As Found: Syndrome

- ID: M14099

- Name: Prader-Willi Syndrome

- Relevance: HIGH

- As Found: Prader-Willi Syndrome

- ID: M10014

- Name: Hyperphagia

- Relevance: HIGH

- As Found: Hyperphagia

- ID: M11589

- Name: Intellectual Disability

- Relevance: LOW

- As Found: Unknown

- ID: M21826

- Name: Neurobehavioral Manifestations

- Relevance: LOW

- As Found: Unknown

- ID: M12404

- Name: Neurologic Manifestations

- 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: M12701

- Name: Obesity

- Relevance: LOW

- As Found: Unknown

- ID: M26186

- Name: Overweight

- Relevance: LOW

- As Found: Unknown

- ID: M25307

- Name: Overnutrition

- Relevance: LOW

- As Found: Unknown

- ID: M12684

- Name: Nutrition Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M15622

- Name: Signs and Symptoms, Digestive

- Relevance: LOW

- As Found: Unknown

- ID: T4665

- Name: Prader-Willi Syndrome

- Relevance: HIGH

- As Found: Prader-Willi Syndrome

### Condition Browse Module - Meshes

- ID: D000011218

- Term: Prader-Willi Syndrome

- ID: D000013577

- Term: Syndrome

- ID: D000006963

- Term: Hyperphagia

### Intervention Browse Module - Ancestors

- ID: D000010120

- Term: Oxytocics

- ID: D000012102

- Term: Reproductive Control Agents

- ID: D000045505

- Term: Physiological Effects of Drugs

### Intervention Browse Module - Browse Branches

- Abbrev: Repr

- Name: Reproductive Control Agents

- Abbrev: All

- Name: All Drugs and Chemicals

### Intervention Browse Module - Browse Leaves

- ID: M205821

- Name: Carbetocin

- Relevance: HIGH

- As Found: Acute Respiratory Distress

### Intervention Browse Module - Meshes

- ID: C000020731

- Term: Carbetocin

### Misc Info Module

- Version Holder: 2024-05-31
