## Protocol Section
### Identification Module
**NCT ID:** NCT06415448

**Brief Title:** Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

**Official Title:** Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema (HAE)

#### Organization Study ID Info
**ID:** ISIS 721744-E01
#### Organization
**Class:** INDUSTRY
**Full Name:** Ionis Pharmaceuticals, Inc.
### Status Module
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-16

**Type:** ACTUAL

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

**Overall Status:** AVAILABLE

**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:** Ionis Pharmaceuticals, Inc.

#### Responsible Party

**Type:** SPONSOR

### Description Module

**Brief Summary:** The purpose of the Expanded Access Program is to provide pre-approval access of donidalorsen to eligible patients with Hereditary Angioedema (HAE) who complete the ISIS 721744-CS3 clinical trial.

**Detailed Description:** The Expanded Access Program (EAP) is intended to provide pre-approval access to donidalorsen for eligible patients with HAE who complete the ISIS 721744-CS3 clinical trial.

### Conditions Module

**Conditions:**

- Hereditary Angioedema

**Keywords:**

- HAE

- IONIS PKK-LRx

- Donidalorsen

- ISIS 721744

### Design Module

#### Expanded Access Types

**Intermediate:** True

**Study Type:** EXPANDED_ACCESS

### Arms Interventions Module

### Interventions

#### Intervention 1

**Description:** Donidalorsen administered once monthly or every other month by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.

**Name:** Donidalorsen

**Other Names:**

- IONIS PKK-LRx

- ISIS 721744

**Type:** DRUG

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* Patients in the ISIS 721744-CS3 (Open-Label Extension \[OLE\] Study) who have completed 205 weeks of treatment.
* Female patients of childbearing potential, and male patients with partners of childbearing potential must be willing to use acceptable contraception, or refrain from sexual activity.

Exclusion Criteria:

* Any patient who is pregnant or plans to become pregnant.
* Any patient who was withdrawn from the ISIS 721744-CS3 OLE study due to a serious adverse event (SAE) related to donidalorsen therapy or who voluntarily withdrew prior to 205 weeks of treatment.

**Minimum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** MedInfo@ionisph.com

**Name:** Ionis Pharmaceuticals Medical Information

**Phone:** 1-833-644-6647 (833-MI-IONIS)

**Role:** CONTACT

### References Module

#### See Also Links

**Label:** Related Info

**URL:** https://www.ionispharma.com/patients/expanded-access-policy/

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000014652

- Term: Vascular Diseases

- ID: D000002318

- Term: Cardiovascular Diseases

- ID: D000014581

- Term: Urticaria

- ID: D000017445

- Term: Skin Diseases, Vascular

- ID: D000012871

- Term: Skin Diseases

- ID: D000006969

- Term: Hypersensitivity, Immediate

- ID: D000006967

- Term: Hypersensitivity

- ID: D000007154

- Term: Immune System Diseases

- ID: D000081208

- Term: Hereditary Complement Deficiency Diseases

- ID: D000081207

- Term: Primary Immunodeficiency Diseases

- ID: D000030342

- Term: Genetic Diseases, Inborn

- ID: D000007153

- Term: Immunologic Deficiency Syndromes

### Condition Browse Module - Browse Branches

- Abbrev: BC14

- Name: Heart and Blood Diseases

- Abbrev: BC17

- Name: Skin and Connective Tissue Diseases

- Abbrev: BC20

- Name: Immune System Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

- Abbrev: BC18

- Name: Nutritional and Metabolic Diseases

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M4127

- Name: Angioedema

- Relevance: HIGH

- As Found: Angioedema

- ID: M27584

- Name: Angioedemas, Hereditary

- Relevance: HIGH

- As Found: Hereditary Angioedema

- ID: M17400

- Name: Vascular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M17330

- Name: Urticaria

- Relevance: LOW

- As Found: Unknown

- ID: M15674

- Name: Skin Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M19714

- Name: Skin Diseases, Vascular

- Relevance: LOW

- As Found: Unknown

- ID: M10018

- Name: Hypersensitivity

- Relevance: LOW

- As Found: Unknown

- ID: M10020

- Name: Hypersensitivity, Immediate

- Relevance: LOW

- As Found: Unknown

- ID: M10200

- Name: Immune System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M6879

- Name: Deficiency Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M2286

- Name: Hereditary Complement Deficiency Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M10199

- Name: Immunologic Deficiency Syndromes

- Relevance: LOW

- As Found: Unknown

- ID: M2285

- Name: Primary Immunodeficiency Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M23686

- Name: Genetic Diseases, Inborn

- Relevance: LOW

- As Found: Unknown

- ID: M16355

- Name: Syndrome

- Relevance: LOW

- As Found: Unknown

- ID: T2734

- Name: Hereditary Angioedema

- Relevance: HIGH

- As Found: Hereditary Angioedema

### Condition Browse Module - Meshes

- ID: D000000799

- Term: Angioedema

- ID: D000054179

- Term: Angioedemas, Hereditary

### Misc Info Module

- Version Holder: 2024-05-31
