A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03053102
• Date of registration: 01/02/2017
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals
• Public title: A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Scientific title: A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Date of first enrolment: March 31, 2017
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03053102
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Italy, Korea, Republic of, New Zealand, United Kingdom

Key inclusion & exclusion criteria

Inclusion Criteria:

- Study designed to include 4 to 12 male and female patients age 18 years or older

- Currently untreated PNH patients with PNH Type III erythrocyte and/or granulocyte
clone size = 10% and anemia (Hgb < 12g/dL) with adequate reticulocytosis (as
determined by the investigator)

- Lactate dehydrogenase (LDH) = 1.5X the upper limit of normal (ULN)

- Platelets = 50,000/uL without the need for platelet transfusions

- Documentation of vaccination for N. meningitidis (quadrivalent ACWY and serogroup B),
H. influenza, and S. 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 (e.g., heart, lung, kidney, liver) or
hematopoietic stem cell/marrow transplant

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

- Patients who have received eculizumab at any dose or interval within the past 75 days
before study entry

- Patients with known or suspected complement deficiency

- Patients with active bacterial infection or clinically significant active viral
infection, a body temperature > 38°C, 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 are pregnant, nursing, or planning to become pregnant during the study or
within 90 days of study drug administration or patients with a female partner who is
pregnant, nursing, or planning to become pregnant during the study or within 90 days
of study drug administration



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Intervention(s)

Drug: ACH-0144471

Primary Outcome(s)

Change-from-baseline in serum lactate dehydrogenase (LDH) levels [Time Frame: Day 28]

Secondary Outcome(s)

Profile of Pharmacodynamics: Complement pathway biomarkers (AP-Wieslab, factor D, and Bb) [Time Frame: Up to Day 28]

Grade 3 and Grade 4 laboratory abnormalities [Time Frame: Up to 15 weeks]

Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), and AEs leading to discontinuation [Time Frame: Up to 15 weeks]

Profile of Pharmacokinetics: Area under the curve (AUC) [Time Frame: Up to Day 28]

Profile of Pharmacokinetics: Maximum plasma concentration (Cmax) [Time Frame: Up to Day 28]

Change-from-baseline in hemoglobin (Hgb) [Time Frame: At Day 28 and Day 84]

Profile of Pharmacokinetics: Time to maximum concentration (Tmax) [Time Frame: Up to Day 28]

Secondary ID(s)

ACH471-100

2016-002652-25

U1111-1190-3490

Source(s) of Monetary Support

Please refer to primary and secondary sponsors