NCT07413250

Brief Summary

This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry. The primary objectives seek to characterize the long-term safety profile of danicopan as add-on therapy to ravulizumab/eculizumab in participants with PNH.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at P25-P50 for all trials

Timeline
45mo left

Started Jan 2026

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress8%
Jan 2026Jan 2030

Study Start

First participant enrolled

January 14, 2026

Completed
27 days until next milestone

First Submitted

Initial submission to the registry

February 10, 2026

Completed
7 days until next milestone

First Posted

Study publicly available on registry

February 17, 2026

Completed
3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 15, 2030

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 15, 2030

Last Updated

February 17, 2026

Status Verified

January 1, 2026

Enrollment Period

4 years

First QC Date

February 10, 2026

Last Update Submit

February 10, 2026

Conditions

Paroxysmal Nocturnal HemoglobinuriaPNH

Keywords

Paroxysmal Nocturnal HemoglobinuriaPNHDanicopanRavulizumabEculizumab

Outcome Measures

Primary Outcomes (4)

  • Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

    From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years

  • Event Rate of Meningococcal Infections

    From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years

  • Event Rate of Serious Infections

    Up to approximately 5 years

  • Event Rate of Malignancies and Hematologic Abnormalities

    Up to approximately 5 years

Secondary Outcomes (7)

  • Number of Participants with AEs and SAEs in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding

    Up to approximately 5 years

  • Number of Infant Health Abnormalities in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding

    Up to 12 months

  • Number of Participants with PNH symptoms at Diagnosis

    Baseline (Day 1)

  • Number of Participants with a History Bone Marrow Transplant

    Baseline (Day 1)

  • Number of Participants with a history of Major Adverse Vascular Events, including Thrombosis

    Baseline (Day 1)

  • +2 more secondary outcomes

Study Arms (2)

Cohort 1

Participants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment.

Drug: Danicopan

Cohort 2

Participants initiating treatment with Ultomiris or Soliris on or after IPIG or Alexion International PNH Registry enrollment and without any danicopan treatment experience during follow-up.

Drug: Soliris/Ultomiris

Interventions

DanicopanDRUG

Participants treated with danicopan as an add-on therapy.

Cohort 1
Soliris/UltomirisDRUG

Participants treated with Soliris/Ultomiris monotherapy.

Cohort 2

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

The study population will consist of 2 treatment cohorts: * Participants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment. * Participants initiating treatment with Ultomiris or Soliris on or after IPIG or Alexion International PNH Registry enrollment and without any danicopan treatment experience during follow-up.

You may qualify if:

  • Adult participants aged ≥ 18 years at treatment initiation.
  • Initiated treatment with Ultomiris, Soliris, and/or danicopan on or after IPIG or Alexion International PNH Registry enrollment.

You may not qualify if:

  • Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and Ultomiris and/or Soliris.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Clinical Research Site

Boston, Massachusetts, 02210, United States

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

danicopaneculizumabravulizumab

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic SyndromesBone Marrow Diseases

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
OTHER
Target Duration
6 Years
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 10, 2026

First Posted

February 17, 2026

Study Start

January 14, 2026

Primary Completion (Estimated)

January 15, 2030

Study Completion (Estimated)

January 15, 2030

Last Updated

February 17, 2026

Record last verified: 2026-01

Locations

US(1)