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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03181633




Registration number
NCT03181633
Ethics application status
Date submitted
5/06/2017
Date registered
9/06/2017
Date last updated
20/08/2019

Titles & IDs
Public title
A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Scientific title
An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment With ACH-0144471 in Patients 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 patients will receive ACH-0144471 during the treatment period.


Treatment: Drugs: ACH-0144471
ACH-0144471 will be administered to all patients enrolled in the study.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), and AEs leading to discontinuation
Timepoint [1] 0 0
Through study completion, an average of 2 years
Primary outcome [2] 0 0
Serum lactate dehydrogenase (LDH) levels
Timepoint [2] 0 0
Through study completion, an average of 2 years
Primary outcome [3] 0 0
Hemoglobin (Hgb) levels
Timepoint [3] 0 0
Through study completion, an average of 2 years
Primary outcome [4] 0 0
Number of red blood cell (RBC) transfusions
Timepoint [4] 0 0
Through study completion, an average of 2 years
Primary outcome [5] 0 0
Reticulocyte counts
Timepoint [5] 0 0
Through study completion, an average of 2 years
Secondary outcome [1] 0 0
FACIT Fatigue Scale scores
Timepoint [1] 0 0
Week 1, Week 13, Week 37, and then every 6 months through study completion, an average of 2 years
Secondary outcome [2] 0 0
EORTC QLQ-C30 scores
Timepoint [2] 0 0
Week 1, Week 13, Week 37, and then every 6 months through study completion, an average of 2 years

Eligibility
Key inclusion criteria
- Study designed to include up to 12 patients 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
Gender
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 patient inappropriate for 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 patient
inappropriate for the study or put the patient 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 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Achillion Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate long term safety and efficacy of ACH-0144471 in
patients with PNH who have demonstrated clinical benefit from ACH-0144471 in Clinical Study
ACH471-100. This study is designed to include up to 12 patients.
Trial website
https://clinicaltrials.gov/show/NCT03181633
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

Summary results
Other publications