Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Identifier:
ACH471-100
ClinicalTrials.gov Identifier:
NCT03053102
EudraCT Identifier:
2016-002652-25
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Available Documents

ProtocolStatistical Analysis Plan

Study Details

Medical Condition
  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study Drug
  • Drug: Danicopan
Date
Mar 2017 - Nov 2018
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
  • Currently untreated PNH participants with PNH Type III erythrocyte and/or granulocyte clone size ≥10% and anemia (hemoglobin \<12 grams/deciliter) with adequate reticulocytosis (as determined by the Investigator).
  • LDH ≥1.5 x the upper limit of normal.
  • Platelets ≥50,000/microliter without the need for platelet transfusions.
  • Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and Streptococcus pneumoniae, or willingness to receive vaccinations during the screening period.
  • Negative pregnancy test for females prior to dosing and throughout the study.
Exclusion Criteria
  • History of a major organ transplant (for example, heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant.
  • Participants who had received another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study entry, whichever is greater.
  • Participants who had received eculizumab at any dose or interval within the past 75 days before study entry.
  • Participants with known or suspected complement deficiency.
  • Participants with active bacterial infection or clinically significant active viral infection, a body temperature \>38°Celsius, or other evidence of infection on Day 1, or with a history of febrile illness within 14 days prior to first study drug administration.
  • History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection.
  • Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who was pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration.
Sex
Female & Male
Age
18+ years

Study Details

Medical Condition
  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study Drug
  • Drug: Danicopan
Date
Mar 2017 - Nov 2018
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
  • Currently untreated PNH participants with PNH Type III erythrocyte and/or granulocyte clone size ≥10% and anemia (hemoglobin \<12 grams/deciliter) with adequate reticulocytosis (as determined by the Investigator).
  • LDH ≥1.5 x the upper limit of normal.
  • Platelets ≥50,000/microliter without the need for platelet transfusions.
  • Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and Streptococcus pneumoniae, or willingness to receive vaccinations during the screening period.
  • Negative pregnancy test for females prior to dosing and throughout the study.
Exclusion Criteria
  • History of a major organ transplant (for example, heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant.
  • Participants who had received another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study entry, whichever is greater.
  • Participants who had received eculizumab at any dose or interval within the past 75 days before study entry.
  • Participants with known or suspected complement deficiency.
  • Participants with active bacterial infection or clinically significant active viral infection, a body temperature \>38°Celsius, or other evidence of infection on Day 1, or with a history of febrile illness within 14 days prior to first study drug administration.
  • History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection.
  • Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who was pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration.

Protocol Summary

The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.

Trial Locations

Location
Status
Location
Clinical Trial Site
Florence, Italy
Status
N/A
Location
Clinical Trial Site
Naples, Italy
Status
N/A
Location
Clinical Trial Site
Auckland, New Zealand
Status
N/A
Location
Clinical Trial Site
Seoul, Republic of Korea
Status
N/A
Location
Clinical Trial Site
London, United Kingdom
Status
N/A