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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03053102
Recruitment Status : Completed
First Posted : February 14, 2017
Last Update Posted : August 28, 2019
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
The purpose of this study is to determine the safety and effectiveness of ACH-0144471 in currently untreated patients with PNH. This study is designed to include 4 to 12 patients.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: ACH-0144471 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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)
Actual Study Start Date : March 31, 2017
Actual Primary Completion Date : November 14, 2018
Actual Study Completion Date : November 14, 2018

Arm Intervention/treatment
Experimental: ACH-0144471
All patients will receive ACH-0144471 during the treatment period.
Drug: ACH-0144471
ACH-0144471 will be administered to all patients enrolled in the study.

Primary Outcome Measures :
  1. Change-from-baseline in serum lactate dehydrogenase (LDH) levels [ Time Frame: Day 28 ]

Secondary Outcome Measures :
  1. Change-from-baseline in hemoglobin (Hgb) [ Time Frame: At Day 28 and Day 84 ]
  2. Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), and AEs leading to discontinuation [ Time Frame: Up to 15 weeks ]
  3. Grade 3 and Grade 4 laboratory abnormalities [ Time Frame: Up to 15 weeks ]
  4. Profile of Pharmacokinetics: Area under the curve (AUC) [ Time Frame: Up to Day 28 ]
  5. Profile of Pharmacokinetics: Maximum plasma concentration (Cmax) [ Time Frame: Up to Day 28 ]
  6. Profile of Pharmacokinetics: Time to maximum concentration (Tmax) [ Time Frame: Up to Day 28 ]
  7. Profile of Pharmacodynamics: Complement pathway biomarkers (AP-Wieslab, factor D, and Bb) [ Time Frame: Up to Day 28 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03053102

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Careggi University Hospital
Florence, Italy
Federico II University of Naples
Naples, Italy
Korea, Republic of
Seoul St. Mary's Hospital
Seoul, Korea, Republic of
New Zealand
Auckland Clinical Studies Limited
Auckland, New Zealand
United Kingdom
King's College Hospital NHS Foundation Trust
London, United Kingdom
Sponsors and Collaborators
Alexion Pharmaceuticals
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Responsible Party: Alexion Pharmaceuticals Identifier: NCT03053102    
Other Study ID Numbers: ACH471-100
2016-002652-25 ( EudraCT Number )
U1111-1190-3490 ( Other Identifier: UTN )
First Posted: February 14, 2017    Key Record Dates
Last Update Posted: August 28, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Alexion Pharmaceuticals:
Additional relevant MeSH terms:
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Hemoglobinuria, Paroxysmal
Urination Disorders
Urologic Diseases
Urological Manifestations
Anemia, Hemolytic
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases