Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab

• Register: ClinicalTrials.gov
• Last refreshed on: 13 February 2023
• Main ID: NCT03472885
• Date of registration: 15/03/2018
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals
• Public title: Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab PNH
• Scientific title: A Phase 2 Open-label Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy
• Date of first enrolment: May 8, 2018
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03472885
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Italy, United Kingdom, United States

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- Diagnosed with PNH

- Have received at least one red blood cell transfusion within last 12 weeks

- Anemia with adequate reticulocytosis

- Must be on a stable regimen of eculizumab

- Platelet count = 40,000/microliter without the need for platelet transfusions

- Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and
Streptococcus pneumoniae or willingness to receive vaccinations based on local
guidelines

- Willingness to receive antibiotic prophylaxis

- Female participants must use highly effective birth control to prevent pregnancy
during the clinical trial and for 30 days after their last dose of study drug

- Male participants must use a highly effective birth control with a female partner to
prevent pregnancy during the clinical trial and for 90 days after the last dose of
study drug

Key Exclusion Criteria:

- Current evidence of bone marrow failure or aplastic anemia requiring treatment

- History of a major organ transplant or hematopoietic stem cell/marrow transplant

- Received another investigational agent within 30 days or 5 half-lives of the
investigational agent prior to study entry, whichever is greater

- Documented C5 complement protein mutations

- Known or suspected complement deficiency

- Contraindication to any of the required vaccinations

- Active bacterial infection or clinically significant active viral infection, a body
temperature >38°C, or other evidence of infection

- History of meningococcal infection, or a first-degree relative or household contact
with a history of meningococcal infection

- History of hypersensitivity reactions to commonly used antibacterial agents

Note: Additional inclusion/exclusion criteria may apply, per protocol.



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Intervention(s)

Drug: Eculizumab

Drug: Danicopan

Primary Outcome(s)

Change From Baseline In Hemoglobin At Week 24 [Time Frame: Baseline, Week 24]

Secondary Outcome(s)

Number Of Units Of Red Blood Cells (RBCs) Transfused During 24 Weeks Of Treatment [Time Frame: From 24 weeks prior to first dose through 24 weeks of treatment]

Number Of Participants Without RBC Transfusions [Time Frame: From 24 weeks prior to first dose through 24 weeks of treatment]

Change From Baseline In Lactate Dehydrogenase At Week 24 [Time Frame: Baseline, Week 24]

Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Adverse Events (AEs), And Events Leading To Discontinuation Of Study Drug [Time Frame: Day 1 (after dosing) through 24 weeks]

Secondary ID(s)

U1111-1209-4655

ACH471-101

2016-003526-16

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Achillion, a wholly owned subsidiary of Alexion

Results

• Results available: Yes
• Date Posted: 25/06/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03472885