Paroxysmal Nocturnal HemoglobinuriaNCT07413250
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
An observational study of Paroxysmal Nocturnal Hemoglobinuria and PNH, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- What is being tested?
- Danicopan and Soliris/Ultomiris
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Jan 2026to 15 Jan 2030
- How long will I take part?
- 6 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and Ultomiris and/or Soliris.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of International PNH Interest Group (IPIG)-Registry Data
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.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- Being tested
- Danicopan, Soliris/Ultomiris
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Danicopan, Ravulizumab, Eculizumab
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DanicopanDrug
Participants treated with danicopan as an add-on therapy.
Soliris/UltomirisDrug
Participants treated with Soliris/Ultomiris monotherapy.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Participants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment. Receives: 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. Receives: Drug: Soliris/Ultomiris |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years
Event Rate of Meningococcal Infections
Measured: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years
Event Rate of Serious Infections
Measured: Up to approximately 5 years
Event Rate of Malignancies and Hematologic Abnormalities
Measured: 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
Measured: Up to approximately 5 years
Number of Infant Health Abnormalities in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding
Measured: Up to 12 months
Number of Participants with PNH symptoms at Diagnosis
Measured: Baseline (Day 1)
Number of Participants with a History Bone Marrow Transplant
Measured: Baseline (Day 1)
Number of Participants with a history of Major Adverse Vascular Events, including Thrombosis
Measured: Baseline (Day 1)
Number of Participants with Ongoing Severe Hepatic Impairment as Defined by Child-Pugh Class C at Enrollment
Measured: Baseline (Day 1)
Number of Participants Who Discontinue Danicopan Treatment
Measured: Up to approximately 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2026
- Study start14 Jan 2026
- Primary completion15 Jan 2030 (estimated)
- Study completion15 Jan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 50 participants (estimated)
- Target duration
- 6 Years
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07413250
- Sponsor's protocol ID
- D7332R00003
- Other IDs
- ALX-PNH-502
- Status last verified
- Jan 2026
- First submitted
- 10 Feb 2026
- Last updated
- 17 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of International PNH Interest Group (IPIG)-Registry Data
- ClinicalTrials.gov ID
- NCT07413250
- Acronym / sponsor ID
- D7332R00003
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Danicopan, Ravulizumab, Eculizumab
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Jan 2026 (actual)
- Primary completion
- 15 Jan 2030 (estimated)
- Study completion
- 15 Jan 2030 (estimated)
- First posted
- 17 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 6 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | Drug: DanicopanParticipants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment. |
| Cohort 2 | – | Drug: Soliris/UltomirisParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and Ultomiris and/or Soliris.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DanicopanParticipants treated with danicopan as an add-on therapy. | Drug | Cohort 1 |
| Soliris/UltomirisParticipants treated with Soliris/Ultomiris monotherapy. | Drug | Cohort 2 |
Primary outcome
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years
Event Rate of Meningococcal Infections
Time frame: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years
Event Rate of Serious Infections
Time frame: Up to approximately 5 years
Event Rate of Malignancies and Hematologic Abnormalities
Time frame: Up to approximately 5 years
7 secondary outcomes
Number of Participants with AEs and SAEs in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding
Time frame: Up to approximately 5 years
Number of Infant Health Abnormalities in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding
Time frame: Up to 12 months
Number of Participants with PNH symptoms at Diagnosis
Time frame: Baseline (Day 1)
Number of Participants with a History Bone Marrow Transplant
Time frame: Baseline (Day 1)
Number of Participants with a history of Major Adverse Vascular Events, including Thrombosis
Time frame: Baseline (Day 1)
Number of Participants with Ongoing Severe Hepatic Impairment as Defined by Child-Pugh Class C at Enrollment
Time frame: Baseline (Day 1)
Number of Participants Who Discontinue Danicopan Treatment
Time frame: Up to approximately 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Hemoglobinuria and PNH, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 17 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2026.