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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03053102
Registration number
NCT03053102
Ethics application status
Date submitted
1/02/2017
Date registered
14/02/2017
Date last updated
23/06/2022
Titles & IDs
Public title
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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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
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Secondary ID [1]
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2016-002652-25
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Secondary ID [2]
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ACH471-100
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Condition category
Condition code
Blood
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Haematological diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Danicopan
Experimental: Danicopan - Starting doses of danicopan ranged from 100 to 150 milligrams (mg) three times daily (TID), with subsequent dose escalation up to 200 mg TID based on response (clinical and biochemical) for 28 days (Part 1). Participants with reductions in lactate dehydrogenase (LDH) meeting specified criteria were offered continued dosing beyond Day 28, for up to 8 additional weeks (Part 2).
Treatment: Drugs: Danicopan
Danicopan was administered as multiple oral doses over a period of at least 28 days.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline In Serum LDH Levels At Day 28
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Assessment method [1]
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Change from Baseline = Serum LDH levels on Day 28 - Baseline Serum LDH levels.
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Timepoint [1]
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Baseline, Day 28
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Secondary outcome [1]
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Change From Baseline In Hemoglobin (Hgb) At Days 28 And 84
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Assessment method [1]
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Change from Baseline = Hgb levels on Days 28 or 84 - Baseline Hgb levels.
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Timepoint [1]
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Baseline, Days 28 and 84
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Secondary outcome [2]
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Change From Baseline In Serum LDH Levels At Day 84
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Assessment method [2]
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Change from Baseline = Serum LDH levels on Day 84 - Baseline Serum LDH levels.
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Timepoint [2]
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Baseline, Day 84
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Secondary outcome [3]
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Paroxysmal Nocturnal Hemoglobinuria (PNH) Type III Red Blood Cell (RBC) Clone Size
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Assessment method [3]
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PNH RBC, summed type III, clone size levels were assessed from Baseline to Day 28 and Day 84. The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population.
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Timepoint [3]
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Baseline, Day 28, and Day 84
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Secondary outcome [4]
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Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation
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Assessment method [4]
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An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of SAEs and other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
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Timepoint [4]
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After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104)
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Secondary outcome [5]
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Grade 3 And Grade 4 Laboratory Abnormalities
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Assessment method [5]
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Laboratory abnormalities were determined from laboratory measurements analyzed at the central or local laboratories, and were graded using CTCAE.
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Timepoint [5]
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After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104).
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Secondary outcome [6]
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Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Post-dose (AUC0-8)
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Assessment method [6]
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Serial blood samples were collected predose and up to 8 hours postdose.
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Timepoint [6]
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Days 6 and 20
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Secondary outcome [7]
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PK: Maximum Plasma Concentration (Cmax)
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Assessment method [7]
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Serial blood samples were collected predose and up to 12 hours postdose.
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Timepoint [7]
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Days 6 and 20
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Secondary outcome [8]
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PK: Time To Maximum Concentration (Tmax)
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Assessment method [8]
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Serial blood samples were collected predose and up to 12 hours postdose.
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Timepoint [8]
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Days 6 and 20
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Secondary outcome [9]
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Complement Alternative Pathway (AP) Functional Activity
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Assessment method [9]
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Serum AP functional activity was measured by the Wieslab functional immunoassay method.
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Timepoint [9]
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Baseline and Day 28
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Secondary outcome [10]
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Complement Bb
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Assessment method [10]
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Plasma Bb was measured by enzyme-linked immunosorbent assay (ELISA).
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Timepoint [10]
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Baseline and Day 28
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Eligibility
Key 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.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
31/03/2017
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
14/11/2018
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Sample size
Target
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Accrual to date
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Final
10
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
Country [1]
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Italy
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State/province [1]
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Florence
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Country [2]
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Italy
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State/province [2]
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Naples
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Country [3]
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Korea, Republic of
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State/province [3]
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Seoul
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Country [4]
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New Zealand
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State/province [4]
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Auckland
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Country [5]
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United Kingdom
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State/province [5]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alexion Pharmaceuticals, Inc.
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Address
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Country
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Other collaborator category [1]
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Commercial sector/Industry
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Name [1]
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Achillion, a wholly owned subsidiary of Alexion
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Address [1]
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Country [1]
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Ethics approval
Ethics application status
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Summary
Brief 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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03053102
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03053102
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