Paroxysmal Nocturnal HemoglobinuriaNCT05982938
Danicopan Early Access Program
A study testing Danicopan in people with Paroxysmal Nocturnal Hemoglobinuria and PNH, run by Alexion Pharmaceuticals, Inc. at 15 sites in Italy.
Trial summary
This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
- What is being tested?
- Danicopan
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 15 sitesItaly
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
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
Who may be able to join
- Male or female, aged 18 years and older
- Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5 g/dL) with absolute reticulocyte count ≥ 120 × 109/L.
- Currently receiving treatment with SOLIRIS® or ULTOMIRIS® for at least 6 months prior to initiation of danicopan treatment.
- Platelet count ≥ 30,000/µL without the need for platelet transfusions.
- Absolute neutrophil counts ≥ 500/µL.
- Vaccinated against N meningitidis.
Who cannot join
- Participants who are nursing or pregnant (or women who are planning to become pregnant during treatment with danicopan).
- Laboratory abnormalities at screening, as follows:
- Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN)
- Direct bilirubin > 2 × ULN, with the exception of:
- participants with increased bilirubin due to EVH in the opinion of the Treating Physician or
- participants with documented Gilbert's syndrome
- Active bacterial or viral infection, a body temperature > 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to initiating danicopan treatment.
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
Italy15 sites
- Available
Research Site
Bologna, 40138
- Available
Research Site
Cagliari, 09121
- Available
Research Site
Catania, 95123
- Available
Research Site
Civitanova Marche, 62012
- Available
Research Site
Cremona, 26100
- Available
Research Site
Cuneo, 12100
- Available
Research Site
Frosinone, 03100
- Available
Research Site
Novara, 28100
- Available
Research Site
Palermo, 90146
- Available
Research Site
Pescara, 65100
- Available
Research Site
Ravenna, 48121
- Available
Research Site
Reggio Calabria, 89100
- Available
Research Site
Roma, 00161
- Available
Research Site
Roma, 00168
- Available
Research Site
Torino, 10126
About this study
Official title: Early Access Program (EAP) for Danicopan as Add-on Treatment to SOLIRIS® or ULTOMIRIS® in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Clinically Significant Extravascular Hemolysis (csEVH)
This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.
- Being tested
- Danicopan
- Keywords
- paroxysmal nocturnal hemoglobinuria, PNH, component 5 inhibitors, C5i, intravascular hemolysis, IVH, anemia, EVH, extravascular hemolysis
- Medical subject headings
- Hemoglobinuria, Paroxysmal, Hemolysis, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DanicopanDrug
Also called ALXN2040
Participants will receive 150 mg danicopan three times per day (TID) orally as an add-on to a background C5i therapy (SOLIRIS® or ULTOMIRIS®). Participants may be dose-escalated to 200 mg TID based on safety and clinical effect.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2023
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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05982938
- Sponsor's protocol ID
- D7332R00001
- Status last verified
- Jun 2026
- First submitted
- 1 Aug 2023
- Last updated
- 4 Jun 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Access Program (EAP) for Danicopan as Add-on Treatment to SOLIRIS® or ULTOMIRIS® in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Clinically Significant Extravascular Hemolysis (csEVH)
- ClinicalTrials.gov ID
- NCT05982938
- Acronym / sponsor ID
- D7332R00001
- Keywords
- paroxysmal nocturnal hemoglobinuria, PNH, component 5 inhibitors, C5i, intravascular hemolysis, IVH, anemia, EVH
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- First posted
- 9 Aug 2023 (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 an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female, aged 18 years and older
- Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5 g/dL) with absolute reticulocyte count ≥ 120 × 109/L.
- Currently receiving treatment with SOLIRIS® or ULTOMIRIS® for at least 6 months prior to initiation of danicopan treatment.
- Platelet count ≥ 30,000/µL without the need for platelet transfusions.
- Absolute neutrophil counts ≥ 500/µL.
Key exclusion criteria (7 listed)
- Participants who are nursing or pregnant (or women who are planning to become pregnant during treatment with danicopan).
- Laboratory abnormalities at screening, as follows:
- Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN)
- Direct bilirubin > 2 × ULN, with the exception of:
- Active bacterial or viral infection, a body temperature > 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to initiating danicopan treatment.
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 |
|---|---|---|
| DanicopanAlso: ALXN2040Participants will receive 150 mg danicopan three times per day (TID) orally as an add-on to a background C5i therapy (SOLIRIS® or ULTOMIRIS®). Participants may be dose-escalated to 200 mg TID based on safety and clinical effect. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 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.
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Danicopan in people with Paroxysmal Nocturnal Hemoglobinuria and PNH, run by Alexion Pharmaceuticals, Inc. at 15 sites in Italy.
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 6 inclusion and 7 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: status not confirmed, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Italy. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Alexion Pharmaceuticals, Inc. (Sponsor) (1-855-752-2356, clinicaltrials@alexion.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
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 4 Jun 2026; AzTrials last refreshed its copy on 26 Oct 2026.