Phase 2 N=8 Treatment

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

Paroxysmal Nocturnal Hemoglobinuria

Bottom Line

View on ClinicalTrials.gov: NCT03181633 ↗

Enrolled (actual)

Serious AEs

37.5%

Results posted

Mar 2023

Primary outcome: Primary: Change From Baseline in LDH Level at Week 25 — -683.29 U/L

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: ACH-0144471 (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Alexion Pharmaceuticals, Inc.
Primary completion: Jan 2022

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline in LDH Level at Week 25	-683.29	—
PRIMARY Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25	24.67	—
PRIMARY Change From Baseline in Reticulocyte Counts at Week 25	-0.07	—
PRIMARY Number of RBC Units Transfused	6.5	—
PRIMARY Number of RBC Transfusion Instances	3.4	—
PRIMARY Change From Baseline in PNH Clone Size at Week 25	22.00	—
PRIMARY Change From Baseline in AP Complement Functional Activity at Week 25	-46.36	—
PRIMARY Change From Baseline in Free Hgb at Week 25	59.83	—
PRIMARY 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	8; 3; 2; 0; 0	—
SECONDARY Change From Baseline in LDH Level at Weeks 49 and 169	-862.50; -1388.00	—
SECONDARY Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169	21.40; 54.50	—
SECONDARY Change From Baseline in Reticulocyte Counts at Weeks 49 and 169	-0.05; -0.12	—
SECONDARY Change From Baseline in PNH Clone Size at Weeks 49 and 73	30.83; 32.00	—
SECONDARY Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145	-59.80; -52.43	—
SECONDARY Change From Baseline in Free Hgb at Weeks 49 and 169	105.92; -30.75	—
SECONDARY Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153	8.4; 9.1; 3.0	—
SECONDARY 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	16.67; 11.90; 4.17	—

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03181633). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.