BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy

Phase 2TerminatedNCT05116787

BioCryst Pharmaceuticals12 enrolled

Overview

The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of adult participants with PNH not currently receiving complement inhibitor therapy.

This was a randomized, placebo-controlled, double-blind, parallel-group, 2-part study. Parts 1 and 2 was to be conducted in the same participants. Part 1 of the study was designed to evaluate the efficacy, safety, and tolerability of treatment with oral BCX9930 monotherapy for 12 weeks versus placebo in participants with PNH who were not currently receiving treatment with complement inhibitor therapy. Participants were randomized to receive BCX9930 or placebo under double blind conditions for the 12-week randomized treatment period. The primary efficacy and safety analyses were based on Part 1. Part 2 of the study was designed to evaluate the long-term safety, tolerability, and effectiveness of open-label BCX9930 monotherapy when administered through Week 52. All participants in Part 2 received BCX9930. Participants who were randomized to BCX9930 monotherapy in Part 1 continued to receive BCX9930 in Part 2. Participants who were randomized to placebo in Part 1 discontinued that therapy at the Week 12 visit and received BCX9930 in Part 2.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

PlaceboDRUG

Administered orally twice daily

BCX9930 monotherapyDRUG

Administered orally twice daily

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male or female, aged ≥ 18 years old * Body weight ≥ 40 kg * Documented diagnosis of PNH * No complement inhibitor therapy for ≥ 12 months prior to screening * Contraindication to or no access to approved (C3 or C5) complement inhibitor therapies * Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willingness to start vaccination series * At screening: PNH clone of ≥ 10%, hemoglobin ≤ 10.5 g/dL and lactate dehydrogenase ≥ 2 × upper limit of normal Exclusion Criteria: * Known history of or existing diagnosis of hereditary complement deficiency * History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation * Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition * History of malignancy within 5 years prior to the screening visit * Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening * Treatment with anti-thymocyte globulin within 180 days prior to screening * Initiation of treatment with an erythrocyte or platelet growth factor, or danazol within 28 days prior to screening * Receiving iron supplementation with an unstable dose in the 28 days prior to screening

Locations (5)

Investigative Site

Ampang, Malaysia

Investigative Site

Bloemfontein, South Africa

Investigative Site

Cape Town, South Africa

Investigative Site

Pretoria, South Africa

InvestigativeSite

Outcomes

Primary Outcomes

Part 1: Change From Baseline in Hemoglobin at Week 12

Time frame: Baseline, Week 12

Secondary Outcomes

Part 1: Number of Participants Who Were Transfusion-free

The number of participants who did not receive any transfusions (packed red blood cells \[pRBCs\] or whole blood) during the period of interest were reported. Participants who were transfusion free were defined for each treatment group as the number of participants who did not receive any transfusions (pRBCs or whole blood) during the period of interest from the start to the end, inclusive, divided by the total number of participants in that treatment group at the start of the period of interest. Participants who (1) discontinued treatment prior to Week 12, or (2) did not receive a transfusion during the period of interest despite recording a Hemoglobin (Hb) value ≤ 9 g/dL with symptoms assessed by the investigator as warranting transfusion or a Hb value ≤ 7 g/dL regardless of symptoms were not considered transfusion free.

Time frame: From Week 4 to Week 12

Part 1: Number of Units of pRBCs Transfused

Time frame: From Week 4 to Week 12

Part 1: Percent Change From Baseline in Lactate Dehydrogenase

Time frame: Baseline, Week 12

Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score

The FACIT-Fatigue scale questionnaire was used to determine the level of fatigue experienced by participants. This questionnaire was a 13-item measure that assessed self-reported fatigue and its impact upon daily activities and function. Item scores ranged from 0 ("not at all") to 4 ("very much"), and the total score ranged from 0 to 52, with higher scores indicating greater quality of life.

Time frame: Baseline, Week 12

Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused

Data from ClinicalTrials.gov

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