Long-term Safety of Danicopan: IPIG Registry-based Cohort... | NCT07413679

Studies
Long-term Safety of Danicopan: IPIG Registry-based Cohort Study

NCT07413679

Sponsor

Alexion Pharmaceuticals, Inc.

Status

Active, not recruiting

Last Update Posted

Feb 17, 2026Actual

Enrollment

50Estimated

Phase

Not provided

Conditions

Paroxysmal Nocturnal Hemoglobinuria
PNH

Interventions

Danicopan

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
ALX-PNH-503	Other Identifier	Alexion

Status Module

Record Verification Date

Feb 2026

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Aug 18, 2025Actual

Primary Completion Date

Jul 1, 2029Estimated

Completion Date

Jul 1, 2029Estimated

First Submitted Date

Feb 10, 2026

First Submission Date that Met QC Criteria

Feb 10, 2026

First Posted Date

Feb 17, 2026Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 10, 2026

Last Update Posted Date

Feb 17, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

This is a noninterventional cohort study using primary and secondary data from the International PNH Interest Group (IPIG) PNH registry. It is designed to characterize the long-term safety and tolerability of danicopan as add-on therapy to eculizumab or ravulizumab in adult participants with PNH.

Detailed Description

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Yes

Target Follow-Up Duration

5 Years

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

50Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Danicopan		Participants with PNH treated with Danicopan as an add-on therapy to eculizumab or ravulizumab.	Drug: Danicopan

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Danicopan	Drug	Participants will receive Danicopan as an add-on therapy.	Danicopan

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants with Serious Adverse Events (SAEs)		Up to approximately 5 years
Number of Participants with Serious Infections Caused by Encapsulated Bacteria		Up to approximately 5 years

Secondary Outcomes

Measure	Description	Time Frame
Number of Participants Who Discontinue Study Treatment		Up to approximately 5 years
Frequency of Reasons for Discontinuation		Up to approximately 5 years

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Adult participants aged ≥ 18 years at danicopan treatment initiation (baseline) as add-on therapy to ravulizumab/eculizumab.
Initiated treatment with danicopan on or after registry enrollment

Exclusion Criteria:

Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and ravulizumab and/or eculizumab treatment status.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

The study population will consist of adult participants ≥ 18 years at danicopan treatment initiation (baseline) as add-on therapy to ravulizumab/eculizumab. Participants will have initiated treatment with danicopan on or after registry enrollment.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Alexion Pharmaceuticals, Inc. (Sponsor)		Boston	Massachusetts	02210	United States

Condition Browse Module

MeSH Terms

ID	Term
D006457	Hemoglobinuria, Paroxysmal

Ancestor Terms

ID	Term
D000743	Anemia, Hemolytic
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C000718467	danicopan

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided