Danicopan Early Access Program
Study Identifier:
D7332R00001
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
Recruiting
Study Details
Medical Condition
- Paroxysmal Nocturnal Hemoglobinuria
- PNH
- Extravascular Hemolysis
Study Drug
- Drug: Danicopan
Date
N/A - N/A
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Key Inclusion Criteria
- 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.
- Key Exclusion Criteria
- 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.
Sex
Female & Male
Age
18+ years
Study Details
Medical Condition
- Paroxysmal Nocturnal Hemoglobinuria
- PNH
- Extravascular Hemolysis
Study Drug
- Drug: Danicopan
Date
N/A - N/A
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Key Inclusion Criteria
- 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.
- Key Exclusion Criteria
- 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.
Protocol 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 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.
Trial Locations
Location
Status
Location
Research Site
Bologna, Italy, 40138
Status
Available
Location
Research Site
Cagliari, Italy, 09121
Status
Available
Location
Research Site
Catania, Italy, 95123
Status
Available
Location
Research Site
Civitanova Marche, Italy, 62012
Status
Available
Location
Research Site
Cremona, Italy, 26100
Status
Available
Location
Research Site
Cuneo, Italy, 12100
Status
Available
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