WP44714 is a Phase I/II, open-label, non-randomized, global, multicenter trial consisting of two parts: * Part 1 is a multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia A with or without factor VIII (FVIII) inhibitors. * Part 2 is a multiple-dose study in pediatric male participants with severe or moderate hemophilia A with or without FVIII inhibitors. The overall aim of the study is to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of NXT007.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
60
Participants will receive NXT007 administered subcutaneously (SC), 2 loading doses once every two weeks (Q2W) followed by once every 4 weeks (Q4W) maintenance doses based on the schedule.
UC Davis Cancer Center
Sacramento, California, United States
RECRUITINGGeorgetown Uni Medical Center
Washington D.C., District of Columbia, United States
WITHDRAWNIndiana Hemophilia & Thrombosis center
Indianapolis, Indiana, United States
RECRUITINGUniversity of Iowa Hospitals and Clnics Dept of Pediatrics
Iowa City, Iowa, United States
RECRUITINGBritish Columbia Children's Hospital
Vancouver, British Columbia, Canada
RECRUITINGHamilton Health Sciences Corporation
Hamilton, Ontario, Canada
RECRUITINGIRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy, Italy
RECRUITINGIstituto Clinico Humanitas
Rozzano (MI), Lombardy, Italy
RECRUITINGAuckland Cancer Trial Centre
Auckland, New Zealand
RECRUITINGUniwersyteckie Centrum Kliniczne
Gda?sk, Poland
RECRUITING...and 4 more locations
Incidence and Severity of Adverse Events, with Severity Determined According to National Cancer Institute Common Terminology Criteria for Adverse Events Grading Scale
Time frame: From Baseline until study completion or discontinuation (up to 7.5 years)
Number of Participants with at Least One Clinical Laboratory Test Abnormality for Hematology Parameters
Time frame: From Baseline until study completion or discontinuation (up to 7.5 years)
Number of Participants with at Least One Clinical Laboratory Test Abnormality for Blood Chemistry Parameters
Time frame: From Baseline until study completion or discontinuation (up to 7.5 years)
Number of Participants with at Least One Vital Sign Abnormality
The vital signs that will be assessed are body temperature, pulse rate, respiratory rate, and systolic and diastolic blood pressure.
Time frame: From Baseline until study completion or discontinuation (up to 7.5 years)
Number of Participants with at Least One Abnormality on Electrocardiogram (ECG) Recordings
The ECG parameters that will be assessed are heart rate, PR interval, QRS interval, QT interval, and QTcF inteval.
Time frame: From Baseline until study completion or discontinuation (up to 7.5 years)
Plasma Concentration of NXT007 at Specified Timepoints
Time frame: At prespecified timepoints from Week 1 to Week 23, every 28 days from Week 25 until Week 49, and every 12 weeks thereafter until study completion or discontinuation (up to 7.5 years)
Maximum Observed Plasma Concentration (Cmax) of NXT007 After the First Dose
Time frame: At prespecified timepoints from Day 1 to Day 15
Time to Maximum Observed Plasma Concentration (tmax) of NXT007 After the First Dose
Time frame: At prespecified timepoints from Day 1 to Day 15
Area Under the Plasma Concentration-Time Curve (AUC) of NXT007 After the First Dose
Time frame: At prespecified timepoints from Day 1 to Day 15
Number of Participants Testing Positive for Anti-Drug Antibodies Against NXT007 at Baseline and During Treatment with Study Drug
Time frame: Baseline (predose on Day 1) and from first dose of study drug until study completion or discontinuation (up to 7.5 years)
Number of Participants Testing Positive for Anti-Factor VIII Inhibitors at Baseline and During Treatment with Study Drug
Time frame: Baseline (predose on Day 1) and from first dose of study drug until study completion or discontinuation (up to 7.5 years)
Model-Based Annualized Bleeding Rate for Treated Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Mean Calculated Annualized Bleeding Rate for Treated Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Median Calculated Annualized Bleeding Rate for Treated Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Model-Based Annualized Bleeding Rate for All Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Mean Calculated Annualized Bleeding Rate for All Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Median Calculated Annualized Bleeding Rate for All Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Model-Based Annualized Bleeding Rate for Treated Spontaneous Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Mean Calculated Annualized Bleeding Rate for Treated Spontaneous Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Median Calculated Annualized Bleeding Rate for Treated Spontaneous Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Model-Based Annualized Bleeding Rate for Treated Joint Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Mean Calculated Annualized Bleeding Rate for Treated Joint Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Median Calculated Annualized Bleeding Rate for Treated Joint Bleeds
Time frame: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Reference Study ID Number: WP44714 https://forpatients.roche.com/ No attachments to email below.
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