Clinical trial NCT03181633: A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: PNH
Paroxysmal
Hemoglobinuria
ACH-0144471
ALXN2040
Danicopan
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Achillion, a wholly owned subsidiary of Alexion
First posted: 2017-06-09
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Change From Baseline in LDH Level at Week 25
timeFrame: Baseline, Week 25
measure: Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25
timeFrame: Baseline, Week 25
measure: Change From Baseline in Reticulocyte Counts at Week 25
timeFrame: Baseline, Week 25
measure: Number of RBC Units Transfused
timeFrame: Baseline up to Week 169
measure: Number of RBC Transfusion Instances
timeFrame: Baseline up to Week 169
measure: Change From Baseline in PNH Clone Size at Week 25
timeFrame: Baseline, Week 25
measure: Change From Baseline in AP Complement Functional Activity at Week 25
timeFrame: Baseline, Week 25
measure: Change From Baseline in Free Hgb at Week 25
timeFrame: Baseline, Week 25
measure: 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
timeFrame: Baseline up to 4.5 years
Secondary outcomes:
measure: Change From Baseline in LDH Level at Weeks 49 and 169
timeFrame: Baseline, Weeks 49 and 169
measure: Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169
timeFrame: Baseline, Weeks 49 and 169
measure: Change From Baseline in Reticulocyte Counts at Weeks 49 and 169
timeFrame: Baseline, Weeks 49 and 169
measure: Change From Baseline in PNH Clone Size at Weeks 49 and 73
timeFrame: Baseline, Weeks 49 and 73
measure: Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145
timeFrame: Baseline, Weeks 49 and 145
measure: Change From Baseline in Free Hgb at Weeks 49 and 169
timeFrame: Baseline, Weeks 49 and 169
measure: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153
timeFrame: Baseline, Weeks 21, 41, and 153
measure: 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
timeFrame: Baseline, Weeks 21, 41, and 153
Other outcomes:
Locations:
city: Avellino
country: Italy
facility: Clinical Trial Site
city: Naples
country: Italy
facility: Clinical Trial Site
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site
city: Auckland
country: New Zealand
facility: Clinical Trial Site
Study officials:
References: