A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Register: ClinicalTrials.gov
• Last refreshed on: 10 October 2022
• Main ID: NCT03181633
• Date of registration: 05/06/2017
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals
• 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
• Date of first enrolment: June 22, 2017
• Target sample size: 8
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03181633
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Italy, Korea, Republic of, New Zealand

Key inclusion & exclusion criteria

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.

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Paroxysmal Nocturnal Hemoglobinuria

Intervention(s)

Drug: ACH-0144471

Primary Outcome(s)

Number Of Red Blood Cell (RBC) Transfusions [Time Frame: Through study completion, an average of 2 years]

Reticulocyte Counts [Time Frame: Through study completion, an average of 2 years]

Serious Adverse Events (SAEs), Grade 3 And Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation [Time Frame: Through study completion, an average of 2 years]

Hemoglobin (Hgb) Levels [Time Frame: Through study completion, an average of 2 years]

Serum Lactate Dehydrogenase (LDH) Levels [Time Frame: Through study completion, an average of 2 years]

Secondary Outcome(s)

Functional Assessment Of Chronic Illness Therapy (FACIT) Fatigue Scale Scores [Time Frame: Week 1, Week 13, Week 37, and then every 6 months through study completion, an average of 2 years]

European Organization For Research And Treatment (EORTC) Cancer Core Quality Of Life Questionnaire (QLQ-C30) Scores [Time Frame: Week 1, Week 13, Week 37, and then every 6 months through study completion, an average of 2 years]

Secondary ID(s)

2017-000665-79

ACH471-103

U1111-1196-0653

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Achillion, a wholly owned subsidiary of Alexion