## Protocol Section
### Identification Module
**NCT ID:** NCT04678479

**Acronym:** CDD

**Brief Title:** Ganaxolone Expanded Access Program Compassionate Use

**Official Title:** Expanded Access With Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin Dependent Kinase-like 5 Deficiency Disorder

#### Organization Study ID Info
**ID:** 1042-CDD-EAP-3005
#### Organization
**Class:** INDUSTRY
**Full Name:** Marinus Pharmaceuticals
### Status Module
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-01-22

**Type:** ACTUAL

**Last Update Submit Date:** 2024-01-18

**Overall Status:** NO_LONGER_AVAILABLE

**Status Verified Date:** 2024-01

#### Study First Post Date
**Date:** 2020-12-22
**Type:** ACTUAL
**Study First Submit Date:** 2020-12-16
**Study First Submit QC Date:** 2020-12-16
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Marinus Pharmaceuticals

#### Responsible Party

**Type:** SPONSOR

### Description Module

**Brief Summary:** The primary objective is to provide GNX to patients ≥ 2 years with CDD-related seizures who are refractory to, or intolerant of, standard therapy.

**Detailed Description:** This is a multi-center, long-term, open-label, expanded access protocol of adjunctive GNX treatment in children, adolescents, and adults with CDD. Patients with inadequate seizure control on their current anti seizure medications at therapeutic doses will be eligible for enrollment.

### Conditions Module

**Conditions:**

- CDKL5 Disorder

### Design Module

#### Expanded Access Types

**Individual:** True

**Treatment:** True

**Study Type:** EXPANDED_ACCESS

### Arms Interventions Module

### Interventions

#### Intervention 1

**Description:** ganaxolone

**Name:** Ganaxolone

**Type:** DRUG

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* Molecular confirmation of a pathogenic CDKL5 variant, early onset, difficult to control seizures, and neurodevelopmental impairment are required. Male or female patients aged ≥ 2 years. In the opinion of the investigator, the patient has inadequate seizure control on current anti-seizure medications at therapeutic doses.

Exclusion Criteria:

-

**Minimum Age:** 2 Years

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Overall Officials

**Official 1:**

**Affiliation:** Marinus Pharmaceuticals, Inc.

**Name:** Paula Bokesch, MD, FAAP

**Role:** STUDY_DIRECTOR

**Official 2:**

**Affiliation:** Marinus Pharmaceuticals, Inc.

**Name:** Igor Grachev, MD, PhD

**Role:** STUDY_DIRECTOR

## Derived Section

### Condition Browse Module - Browse Branches

- Abbrev: Rare

- Name: Rare Diseases

- Abbrev: All

- Name: All Conditions

### Condition Browse Module - Browse Leaves

- ID: T1938

- Name: Dravet Syndrome

- Relevance: HIGH

- As Found: CDKL5 Disorder

- ID: T1024

- Name: CDKL5 Deficiency Disorder

- Relevance: HIGH

- As Found: CDKL5 Disorder

### Intervention Browse Module - Ancestors

- ID: D000081227

- Term: Neurosteroids

- ID: D000018377

- Term: Neurotransmitter Agents

- ID: D000045504

- Term: Molecular Mechanisms of Pharmacological Action

- ID: D000045505

- Term: Physiological Effects of Drugs

- ID: D000018757

- Term: GABA Modulators

- ID: D000018682

- Term: GABA Agents

### Intervention Browse Module - Browse Branches

- Abbrev: All

- Name: All Drugs and Chemicals

### Intervention Browse Module - Browse Leaves

- ID: M347103

- Name: Ganaxolone

- Relevance: HIGH

- As Found: Meningococcal Vaccine

- ID: M2291

- Name: Neurosteroids

- Relevance: LOW

- As Found: Unknown

- ID: M20504

- Name: Neurotransmitter Agents

- Relevance: LOW

- As Found: Unknown

- ID: M20827

- Name: GABA Modulators

- Relevance: LOW

- As Found: Unknown

### Intervention Browse Module - Meshes

- ID: C000105051

- Term: Ganaxolone

### Misc Info Module

- Version Holder: 2024-05-24
