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Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria

Status
Recruiting
Phase
Phase 1
Locations
South Africa
Study Type
Interventional
Intervention
BCX10013
Sponsored by
BioCryst Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring Paroxysmal Nocturnal Hemoglobinuria, Factor D, complement inhibitor, alternative pathway inhibitor, BioCryst

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria: Male or non-pregnant, non-lactating female adults ≥ 18 years old. Documented diagnosis of PNH confirmed by flow cytometry. Body mass index (BMI) ≤ 40 kg/m^2. Are either: (a) naïve to treatment with a complement inhibitor; or (b) have received no treatment with ravulizumab for at least 12 months prior to the screening visit and have received no treatment with eculizumab or pegcetacoplan for 6 months prior to the screening visit. Documentation of current vaccinations against N. meningitidis, S. pneumoniae, and H. influenzae type B [Hib] or willingness to start vaccination series at least 14 days prior to Day 1. Key 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 during the study. Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition, including unstable angina, severe congestive heart failure, unexplained syncope, arrhythmia, and critical aortic stenosis. History of malignancy within 5 years prior to the screening visit. Treatment with anti-thymocyte globulin within 180 days prior to the screening visit. Initiation of treatment with an erythropoiesis-stimulating agent (eg, erythropoietin), a thrombopoietin receptor agonist (eg, eltrombopag), or danazol within 28 days prior to the screening visit. Receiving iron with an unstable dose (ie, increasing or decreasing) in the 28 days prior to the screening visit.

Sites / Locations

  • BioCryst Investigative Site
  • BioCryst Investigative SiteRecruiting
  • BioCryst Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BCX10013

Arm Description

Participants with PNH will receive BCX10013 daily for 4 weeks before dose escalation may occur.

Outcomes

Primary Outcome Measures

Number of participants with treatment-emergent adverse events (TEAEs) and graded laboratory abnormalities, and changes from baseline (CFB) in laboratory analytes, vital signs, electrocardiograms (ECGs), and physical examination findings.

Secondary Outcome Measures

Change from baseline (CFB) in lactate dehydrogenase
CFB in the ratio of total PNH red blood cell clone size to PNH white blood cell clone size
CFB in hemoglobin
Percentage of participants who are transfusion-free
Percentage of participants achieving a within-subject clinically meaningful CFB in the FACIT-Fatigue Scale
CFB in other clinical biomarkers of PNH disease activity
Number of participants with clinical PNH symptoms
Concentration of BCX10013 and its metabolite(s) in plasma
Concentration of BCX10013 and its metabolite(s) in urine (if applicable)

Full Information

First Posted
October 20, 2023
Last Updated
October 20, 2023
Sponsor
BioCryst Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT06100900
Brief Title
Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria
Official Title
An Open-Label, Multicenter, Intra-Subject Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Therapeutic Potential of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 31, 2023 (Anticipated)
Primary Completion Date
August 31, 2024 (Anticipated)
Study Completion Date
August 31, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
BioCryst Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a multicenter, open-label, intra-subject, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and therapeutic potential of BCX10013 in participants with paroxysmal nocturnal hemoglobinuria (PNH). Approximately 15 participants will be enrolled in this study. Participants may receive treatment for up to 24 weeks.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria
Keywords
Paroxysmal Nocturnal Hemoglobinuria, Factor D, complement inhibitor, alternative pathway inhibitor, BioCryst

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Model Description
Four dose levels of BCX10013 are planned for escalation.
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
BCX10013
Arm Type
Experimental
Arm Description
Participants with PNH will receive BCX10013 daily for 4 weeks before dose escalation may occur.
Intervention Type
Drug
Intervention Name(s)
BCX10013
Intervention Description
Four dose levels may be tested in this study.
Primary Outcome Measure Information:
Title
Number of participants with treatment-emergent adverse events (TEAEs) and graded laboratory abnormalities, and changes from baseline (CFB) in laboratory analytes, vital signs, electrocardiograms (ECGs), and physical examination findings.
Time Frame
up to 24 weeks
Secondary Outcome Measure Information:
Title
Change from baseline (CFB) in lactate dehydrogenase
Time Frame
Baseline, Week 24
Title
CFB in the ratio of total PNH red blood cell clone size to PNH white blood cell clone size
Time Frame
Baseline, Week 24
Title
CFB in hemoglobin
Time Frame
Baseline, Week 24
Title
Percentage of participants who are transfusion-free
Time Frame
24 weeks
Title
Percentage of participants achieving a within-subject clinically meaningful CFB in the FACIT-Fatigue Scale
Time Frame
24 weeks
Title
CFB in other clinical biomarkers of PNH disease activity
Time Frame
Baseline, Week 24
Title
Number of participants with clinical PNH symptoms
Time Frame
up to 24 weeks
Title
Concentration of BCX10013 and its metabolite(s) in plasma
Time Frame
Pre-dose, 0.5, 1, 2, 4, and 6 hours post dose on Day 1, Week 2, and Week 4
Title
Concentration of BCX10013 and its metabolite(s) in urine (if applicable)
Time Frame
Pre-dose and all urine from 0 to 6 hours post dose on Day 1, Week 2, and Week 4

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Male or non-pregnant, non-lactating female adults ≥ 18 years old. Documented diagnosis of PNH confirmed by flow cytometry. Body mass index (BMI) ≤ 40 kg/m^2. Are either: (a) naïve to treatment with a complement inhibitor; or (b) have received no treatment with ravulizumab for at least 12 months prior to the screening visit and have received no treatment with eculizumab or pegcetacoplan for 6 months prior to the screening visit. Documentation of current vaccinations against N. meningitidis, S. pneumoniae, and H. influenzae type B [Hib] or willingness to start vaccination series at least 14 days prior to Day 1. Key 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 during the study. Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition, including unstable angina, severe congestive heart failure, unexplained syncope, arrhythmia, and critical aortic stenosis. History of malignancy within 5 years prior to the screening visit. Treatment with anti-thymocyte globulin within 180 days prior to the screening visit. Initiation of treatment with an erythropoiesis-stimulating agent (eg, erythropoietin), a thrombopoietin receptor agonist (eg, eltrombopag), or danazol within 28 days prior to the screening visit. Receiving iron with an unstable dose (ie, increasing or decreasing) in the 28 days prior to the screening visit.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
BioCryst Pharmaceuticals, Inc.
Phone
919.859.1302
Email
clinicaltrials@biocryst.com
Facility Information:
Facility Name
BioCryst Investigative Site
City
Bloemfontein
Country
South Africa
Individual Site Status
Not yet recruiting
Facility Name
BioCryst Investigative Site
City
Cape Town
Country
South Africa
Individual Site Status
Recruiting
Facility Name
BioCryst Investigative Site
City
Pretoria
Country
South Africa
Individual Site Status
Not yet recruiting

12. IPD Sharing Statement

Learn more about this trial

Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

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