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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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)
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
Secondary ID [1] 0 0
2017-000665-79
Secondary ID [2] 0 0
ACH471-103
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in LDH Level at Week 25
Timepoint [1] 0 0
Baseline, Week 25
Primary outcome [2] 0 0
Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25
Timepoint [2] 0 0
Baseline, Week 25
Primary outcome [3] 0 0
Change From Baseline in Reticulocyte Counts at Week 25
Timepoint [3] 0 0
Baseline, Week 25
Primary outcome [4] 0 0
Number of RBC Units Transfused
Timepoint [4] 0 0
Baseline up to Week 169
Primary outcome [5] 0 0
Number of RBC Transfusion Instances
Timepoint [5] 0 0
Baseline up to Week 169
Primary outcome [6] 0 0
Change From Baseline in PNH Clone Size at Week 25
Timepoint [6] 0 0
Baseline, Week 25
Primary outcome [7] 0 0
Change From Baseline in AP Complement Functional Activity at Week 25
Timepoint [7] 0 0
Baseline, Week 25
Primary outcome [8] 0 0
Change From Baseline in Free Hgb at Week 25
Timepoint [8] 0 0
Baseline, Week 25
Primary outcome [9] 0 0
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
Timepoint [9] 0 0
Baseline up to 4.5 years
Secondary outcome [1] 0 0
Change From Baseline in LDH Level at Weeks 49 and 169
Timepoint [1] 0 0
Baseline, Weeks 49 and 169
Secondary outcome [2] 0 0
Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169
Timepoint [2] 0 0
Baseline, Weeks 49 and 169
Secondary outcome [3] 0 0
Change From Baseline in Reticulocyte Counts at Weeks 49 and 169
Timepoint [3] 0 0
Baseline, Weeks 49 and 169
Secondary outcome [4] 0 0
Change From Baseline in PNH Clone Size at Weeks 49 and 73
Timepoint [4] 0 0
Baseline, Weeks 49 and 73
Secondary outcome [5] 0 0
Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145
Timepoint [5] 0 0
Baseline, Weeks 49 and 145
Secondary outcome [6] 0 0
Change From Baseline in Free Hgb at Weeks 49 and 169
Timepoint [6] 0 0
Baseline, Weeks 49 and 169
Secondary outcome [7] 0 0
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153
Timepoint [7] 0 0
Baseline, Weeks 21, 41, and 153
Secondary outcome [8] 0 0
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
Timepoint [8] 0 0
Baseline, Weeks 21, 41, and 153

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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
22/06/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
4/01/2022
Sample size
Target
Accrual to date
Final
8
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
Italy
State/province [1] 0 0
Avellino
Country [2] 0 0
Italy
State/province [2] 0 0
Naples
Country [3] 0 0
Korea, Republic of
State/province [3] 0 0
Seoul
Country [4] 0 0
New Zealand
State/province [4] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Achillion, a wholly owned subsidiary of Alexion
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT03181633