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A Long Term Safety Study of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria, PNH

Status

Active

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

BCX9930

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring BCX9930, Factor D inhibitor, PNH, paroxysmal Nocturnal Hemoglobinuria, oral treatment

Eligibility Criteria

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

Inclusion Criteria:

Male or non-pregnant, non-lactating female subjects
Successfully participated in a previous BCX9930 study of PNH and experienced improvement in their PNH

Exclusion Criteria:

Apart from a diagnosis of PNH, any clinically significant medical or psychiatric condition or medical history, other than those associated with PNH disease, that, in the opinion of the Investigator or Sponsor, would interfere with the subject's ability to participate in the study or participation would increase the risk for that subject
Pregnant, planning to become pregnant, or having been pregnant within 90 days of Day 1, or lactating

Sites / Locations

Study Center
Study Center
Investigative Site
Study Center
Study Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BCX9930

Arm Description

Intervention: Drug: BCX9930

Outcomes

Primary Outcome Measures

Incidence of graded treatment-emergent adverse events

Incidence of graded laboratory chemistry abnormalities

Incidence of graded hematology abnormalities

Incidence of graded coagulation abnormalities

Change in blood pressure

Change in temperature

Change in heart rate

Change in respiratory rate

Change in Electrocardiogram (PR interval)

Change in Electrocardiogram (QT interval)

Change in Electrocardiogram (QRS interval)

Change in Electrocardiogram (RR interval)

Secondary Outcome Measures

Number of blood transfusions

Lactate dehydrogenase

Hemoglobin

Absolute reticulocyte count

Haptoglobin

Clinical PNH symptom assessments

Serum AP complement activity

Plasma Factor Bb

Full Information

NCT ID

NCT04702568

First Posted

January 7, 2021

Last Updated

April 24, 2023

Sponsor

BioCryst Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT04702568

Brief Title

A Long Term Safety Study of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Official Title

A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

December 18, 2020 (Actual)

Primary Completion Date

October 2024 (Anticipated)

Study Completion Date

January 2025 (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

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This study is designed to evaluate the long-term safety of daily oral treatment with BCX9930 in subjects who have participated in a previous BCX9930 trial for PNH and showed a benefit of treatment as determined by the Investigator. The study allows continued access to BCX9930 for enrolled subjects. The study will also evaluate the long-term effectiveness and impact on quality of life and general well-being of BCX9930 treatment, and the subject's satisfaction with the medication.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Paroxysmal Nocturnal Hemoglobinuria, PNH

Keywords

BCX9930, Factor D inhibitor, PNH, paroxysmal Nocturnal Hemoglobinuria, oral treatment

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

200 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

BCX9930

Arm Type

Experimental

Arm Description

Intervention: Drug: BCX9930

Intervention Type

Drug

Intervention Name(s)

BCX9930

Intervention Description

BCX9930 for oral administration

Primary Outcome Measure Information:

Title

Incidence of graded treatment-emergent adverse events

Time Frame

Week 50

Title

Incidence of graded laboratory chemistry abnormalities

Time Frame

Week 50

Title

Incidence of graded hematology abnormalities

Time Frame

Week 50

Title

Incidence of graded coagulation abnormalities

Time Frame

Week 50

Title

Change in blood pressure

Time Frame

Week 50

Title

Change in temperature

Time Frame

Week 50

Title

Change in heart rate

Time Frame

Week 50

Title

Change in respiratory rate

Time Frame

Week 50

Title

Change in Electrocardiogram (PR interval)

Time Frame

Week 50

Title

Change in Electrocardiogram (QT interval)

Time Frame

Week 50

Title

Change in Electrocardiogram (QRS interval)

Time Frame

Week 50

Title

Change in Electrocardiogram (RR interval)

Time Frame

Week 50

Secondary Outcome Measure Information:

Title

Number of blood transfusions

Time Frame

Day 1 through Week 50

Title

Lactate dehydrogenase

Time Frame