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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/NCT03181633
Registration number
NCT03181633
Ethics application status
Date submitted
5/06/2017
Date registered
9/06/2017
Date last updated
14/03/2023
Titles & IDs
Public title
A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Scientific title
An Open-Label Study to Evaluate Efficacy and Safety of Long-Term Treatment With ACH-0144471 in Participants Who Completed Clinical Study ACH471-100
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Secondary ID [1]
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2017-000665-79
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Secondary ID [2]
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ACH471-103
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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
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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 - ACH-0144471
Experimental: ACH-0144471 - All participants will receive ACH-0144471 during the treatment period.
Treatment: Drugs: ACH-0144471
ACH-0144471 will be administered to all participants enrolled in the study.
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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 LDH Level at Week 25
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Assessment method [1]
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Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [1]
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Baseline, Week 25
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Primary outcome [2]
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Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25
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Assessment method [2]
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Change from Baseline = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [2]
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Baseline, Week 25
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Primary outcome [3]
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Change From Baseline in Reticulocyte Counts at Week 25
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Assessment method [3]
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Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [3]
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Baseline, Week 25
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Primary outcome [4]
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Number of RBC Units Transfused
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Assessment method [4]
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Timepoint [4]
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Baseline up to Week 169
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Primary outcome [5]
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Number of RBC Transfusion Instances
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Assessment method [5]
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Timepoint [5]
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Baseline up to Week 169
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Primary outcome [6]
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Change From Baseline in PNH Clone Size at Week 25
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Assessment method [6]
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The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 25 - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [6]
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Baseline, Week 25
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Primary outcome [7]
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Change From Baseline in AP Complement Functional Activity at Week 25
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Assessment method [7]
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Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at Week 25 - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [7]
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Baseline, Week 25
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Primary outcome [8]
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Change From Baseline in Free Hgb at Week 25
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Assessment method [8]
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Change from Baseline = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [8]
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Baseline, Week 25
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Primary outcome [9]
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation
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Assessment method [9]
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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 all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
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Timepoint [9]
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Baseline up to 4.5 years
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Secondary outcome [1]
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Change From Baseline in LDH Level at Weeks 49 and 169
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Assessment method [1]
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Change from Baseline = Serum LDH levels at specified postbaseline visit - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [1]
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Baseline, Weeks 49 and 169
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Secondary outcome [2]
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Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169
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Assessment method [2]
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Change from Baseline = Hgb levels at specified postbaseline visit - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [2]
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Baseline, Weeks 49 and 169
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Secondary outcome [3]
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Change From Baseline in Reticulocyte Counts at Weeks 49 and 169
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Assessment method [3]
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Change from Baseline = reticulocyte count at specified postbaseline visit - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [3]
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Baseline, Weeks 49 and 169
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Secondary outcome [4]
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Change From Baseline in PNH Clone Size at Weeks 49 and 73
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Assessment method [4]
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The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at specified postbaseline visit - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [4]
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Baseline, Weeks 49 and 73
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Secondary outcome [5]
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Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145
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Assessment method [5]
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Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at specified postbaseline visit - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [5]
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Baseline, Weeks 49 and 145
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Secondary outcome [6]
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Change From Baseline in Free Hgb at Weeks 49 and 169
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Assessment method [6]
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Change from Baseline = free Hgb at specified postbaseline visit - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.
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Timepoint [6]
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Baseline, Weeks 49 and 169
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Secondary outcome [7]
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Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153
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Assessment method [7]
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The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.
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Timepoint [7]
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Baseline, Weeks 21, 41, and 153
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Secondary outcome [8]
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Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153
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Assessment method [8]
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EORTC-QLQ-C30 is comprised of 30 questions. First 28 questions used 4-point scale (1=not at all,2=a little,3=quite a bit,4=very much) for evaluating 5 functional scales (physical, role, emotional, cognitive, social), 3 symptom scales (fatigue, nausea/vomiting, pain) & other single items. For each item, high score = high level of symptomatology/problem. Last 2 questions represented participant's assessment of overall health (global health status) and quality of life, coded on 7-point scale (1=very poor to 7=excellent). Answers were converted into grading scale, with total score between 0 and 100. A high score represented a favourable outcome with a best quality of life for participant.
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Timepoint [8]
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Baseline, Weeks 21, 41, and 153
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Eligibility
Key inclusion criteria
- Study designed to include up to 12 participants who completed treatment in Study
ACH471-100 and demonstrated clinical benefit from ACH-0144471 with no significant
safety or tolerability concerns.
- 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
- Have developed any clinically relevant co-morbidities while participating in Study
ACH471-100 that would make the participant inappropriate for the continuation of
treatment with ACH-0144471, in the opinion of the Investigator.
- Have developed any clinically significant laboratory abnormalities while participating
in Study ACH471-100 that, in the opinion of the Investigator, would make the
participant inappropriate for the study or put the participant at undue risk.
- Females who are 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
is 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
22/06/2017
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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
4/01/2022
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Sample size
Target
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Accrual to date
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Final
8
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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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Avellino
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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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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 is to evaluate the long-term safety and efficacy of ACH-0144471 in
participants with paroxysmal nocturnal hemoglobinuria (PNH) who have demonstrated clinical
benefit from ACH-0144471 in Study ACH471-100. This study is designed to include up to 12
participants.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03181633
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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/NCT03181633
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