Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05987449
Registration number
NCT05987449
Ethics application status
Date submitted
4/08/2023
Date registered
14/08/2023
Date last updated
25/06/2024
Titles & IDs
Public title
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Query!
Scientific title
A Phase I/II Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Query!
Secondary ID [1]
0
0
2023-503906-35-00
Query!
Secondary ID [2]
0
0
WP44714
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Hemophilia A
0
0
Query!
Condition category
Condition code
Blood
0
0
0
0
Query!
Clotting disorders
Query!
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - NXT007
Experimental: NXT007 Dose Escalation -
Treatment: Drugs: NXT007
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.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Incidence and Severity of Adverse Events, with Severity Determined According to National Cancer Institute Common Terminology Criteria for Adverse Events Grading Scale
Query!
Assessment method [1]
0
0
Query!
Timepoint [1]
0
0
From Baseline until study completion or discontinuation (up to 7.5 years)
Query!
Primary outcome [2]
0
0
Number of Participants with at Least One Clinical Laboratory Test Abnormality for Hematology Parameters
Query!
Assessment method [2]
0
0
Query!
Timepoint [2]
0
0
From Baseline until study completion or discontinuation (up to 7.5 years)
Query!
Primary outcome [3]
0
0
Number of Participants with at Least One Clinical Laboratory Test Abnormality for Blood Chemistry Parameters
Query!
Assessment method [3]
0
0
Query!
Timepoint [3]
0
0
From Baseline until study completion or discontinuation (up to 7.5 years)
Query!
Primary outcome [4]
0
0
Number of Participants with at Least One Vital Sign Abnormality
Query!
Assessment method [4]
0
0
The vital signs that will be assessed are body temperature, pulse rate, respiratory rate, and systolic and diastolic blood pressure.
Query!
Timepoint [4]
0
0
From Baseline until study completion or discontinuation (up to 7.5 years)
Query!
Primary outcome [5]
0
0
Number of Participants with at Least One Abnormality on Electrocardiogram (ECG) Recordings
Query!
Assessment method [5]
0
0
The ECG parameters that will be assessed are heart rate, PR interval, QRS interval, QT interval, and QTcF inteval.
Query!
Timepoint [5]
0
0
From Baseline until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [1]
0
0
Plasma Concentration of NXT007 at Specified Timepoints
Query!
Assessment method [1]
0
0
Query!
Timepoint [1]
0
0
At prespecified timepoints from Day 1 to Day 155, and every 28 days from Day 169 until study completion (up to 7.5 years)
Query!
Secondary outcome [2]
0
0
Maximum Observed Plasma Concentration (Cmax) of NXT007 After the First Dose
Query!
Assessment method [2]
0
0
Query!
Timepoint [2]
0
0
At prespecified timepoints from Day 1 to Day 15
Query!
Secondary outcome [3]
0
0
Time to Maximum Observed Plasma Concentration (tmax) of NXT007 After the First Dose
Query!
Assessment method [3]
0
0
Query!
Timepoint [3]
0
0
At prespecified timepoints from Day 1 to Day 15
Query!
Secondary outcome [4]
0
0
Area Under the Plasma Concentration-Time Curve (AUC) of NXT007 After the First Dose
Query!
Assessment method [4]
0
0
Query!
Timepoint [4]
0
0
At prespecified timepoints from Day 1 to Day 15
Query!
Secondary outcome [5]
0
0
Number of Participants Testing Positive for Anti-Drug Antibodies Against NXT007 at Baseline and During Treatment with Study Drug
Query!
Assessment method [5]
0
0
Query!
Timepoint [5]
0
0
Baseline (predose on Day 1) and from first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [6]
0
0
Number of Participants Testing Positive for Anti-Factor VIII Inhibitors at Baseline and During Treatment with Study Drug
Query!
Assessment method [6]
0
0
Query!
Timepoint [6]
0
0
Baseline (predose on Day 1) and from first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [7]
0
0
Model-Based Annualized Bleeding Rate for Treated Bleeds
Query!
Assessment method [7]
0
0
Query!
Timepoint [7]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [8]
0
0
Mean Calculated Annualized Bleeding Rate for Treated Bleeds
Query!
Assessment method [8]
0
0
Query!
Timepoint [8]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [9]
0
0
Median Calculated Annualized Bleeding Rate for Treated Bleeds
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [10]
0
0
Model-Based Annualized Bleeding Rate for All Bleeds
Query!
Assessment method [10]
0
0
Query!
Timepoint [10]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [11]
0
0
Mean Calculated Annualized Bleeding Rate for All Bleeds
Query!
Assessment method [11]
0
0
Query!
Timepoint [11]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [12]
0
0
Median Calculated Annualized Bleeding Rate for All Bleeds
Query!
Assessment method [12]
0
0
Query!
Timepoint [12]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [13]
0
0
Model-Based Annualized Bleeding Rate for Treated Spontaneous Bleeds
Query!
Assessment method [13]
0
0
Query!
Timepoint [13]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [14]
0
0
Mean Calculated Annualized Bleeding Rate for Treated Spontaneous Bleeds
Query!
Assessment method [14]
0
0
Query!
Timepoint [14]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [15]
0
0
Median Calculated Annualized Bleeding Rate for Treated Spontaneous Bleeds
Query!
Assessment method [15]
0
0
Query!
Timepoint [15]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [16]
0
0
Model-Based Annualized Bleeding Rate for Treated Joint Bleeds
Query!
Assessment method [16]
0
0
Query!
Timepoint [16]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [17]
0
0
Mean Calculated Annualized Bleeding Rate for Treated Joint Bleeds
Query!
Assessment method [17]
0
0
Query!
Timepoint [17]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Secondary outcome [18]
0
0
Median Calculated Annualized Bleeding Rate for Treated Joint Bleeds
Query!
Assessment method [18]
0
0
Query!
Timepoint [18]
0
0
From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Query!
Eligibility
Key inclusion criteria
* Body weight =40 kilograms (kg) at screening
* Diagnosis of severe (Factor VIII [FVIII] coagulant activity <1 IU/dL) or moderate (FVIII coagulant activity =1 IU/dL and =5 IU/dL) congenital hemophilia A with or without inhibitors against FVIII
* Participants with FVIII inhibitors: participants using recombinant activated factor VII (rFVIIa) or willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds, trauma, or procedures
* Historic local FVIII inhibitor test results being available during screening to confirm any previous inhibitor history and current status
* Participants who previously successfully completed immune tolerance induction (ITI) must have done so at least 5 years before screening and must have no evidence of inhibitor recurrence (permanent or temporary) since. FVIII tolerance defined as <0.6 Bethesda unit (BU)/mL (<1.0 BU/mL only for laboratories with an historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and in vivo recovery >66%
* Documentation of number and type of bleeding episodes in the last 24 weeks prior to enrollment
* Adequate hematologic function, defined as platelet count =100,000 cells/µL and hemoglobin =11 g/dL at the time of screening
* Adequate hepatic function defined as total bilirubin =1.5× age-adapted upper limit of normal (ULN) (excluding Gilbert syndrome) and both AST and ALT =3× age-adapted ULN at the time of screening, and no clinical signs or known laboratory/radiographic evidence consistent with cirrhosis
* Adequate renal function, defined as serum creatinine =2.5× age-adapted ULN and calculated creatinine clearance =30 mL/min by Cockroft-Gault formula
* Willingness and ability to comply with schedules visits, treatment plans, laboratory tests, and other study procedures
Query!
Minimum age
12
Years
Query!
Query!
Maximum age
59
Years
Query!
Query!
Sex
Males
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
* Inherited or acquired bleeding disorders other than congenital hemophilia A
* Ongoing or planned ITI therapy
* Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
* At high risk for thrombotic microangiopathy (TMA), including past personal or family history of TMA, in the investigator's judgment
* Personal history of ischemic heart disease, cerebrovascular disease, or diabetes mellitus
* Strong family history of ischemic heart disease or cerebrovascular disease (i.e., first degree relatives such as parents, full siblings, or children): male relatives diagnosed under the age of 55 years, females under the age of 65 years
* Other conditions (e.g., autoimmune conditions such as Systemic Lupus erythematosus and other systemic inflammatory disorders) that may currently increase the risk of bleeding or thrombosis
* History of clinically significant allergies
* Previous or concomitant malignancies or leukemia
* Receipt of any of the following:
i) An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration or normalization of targeted parameters (e.g., anti-thrombin), whichever is longer; ii) A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter; iii) Any other investigational drug currently being administered or planned to be administered; iv) Prior gene therapy or gene therapy planned to be administered.
* Protein C activity, protein S free antigen, or anti-thrombin III activity levels below the lower limit of the reference range at screening
* Known HIV infection with CD4 counts <200 cells/µL
* History of severe allergic or anaphylactic reactions to monoclonal antibody therapy and to chimeric or humanized antibodies or fusion proteins
* Known hypersensitivity to Chinese hamster ovary cell products or to excipient content
* History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third -degree atrioventricular heart block), including atrial fibrillation or evidence of prior myocardial infarction
* QT interval corrected through use of Fridericia's formula (QTcF) >450 ms demonstrated by at least two ECGs >30 minutes apart
* History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart disease (symptomatic or with ischemia demonstrated by diagnostic testing), clinically significant electrolyte abnormalities (e.g., hypokalemia, hypomagnesemia, hypocalcemia), or family history of sudden unexplained death or long QT syndrome
* Current treatment with medications that are well known to prolong the QT interval
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
NA
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Other
Query!
Other design features
Query!
Phase
Phase 1
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
21/09/2023
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
16/06/2032
Query!
Actual
Query!
Sample size
Target
40
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Indiana
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Iowa
Query!
Country [3]
0
0
Canada
Query!
State/province [3]
0
0
Ontario
Query!
Country [4]
0
0
Italy
Query!
State/province [4]
0
0
Lombardia
Query!
Country [5]
0
0
New Zealand
Query!
State/province [5]
0
0
Auckland
Query!
Country [6]
0
0
Poland
Query!
State/province [6]
0
0
Gda?sk
Query!
Country [7]
0
0
Poland
Query!
State/province [7]
0
0
Warsaw
Query!
Country [8]
0
0
Spain
Query!
State/province [8]
0
0
Madrid
Query!
Country [9]
0
0
Spain
Query!
State/province [9]
0
0
Malaga
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Hoffmann-La Roche
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
Study WP44714 is a Phase I/II, open-label, non-randomized, global, multicenter, multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia A with or without factor VIII (FVIII) inhibitors. The aim is to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of multiple ascending doses of NXT007.
Query!
Trial website
https://clinicaltrials.gov/study/NCT05987449
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Clinical Trials
Query!
Address
0
0
Hoffmann-La Roche
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Reference Study ID Number: WP44714 https://forpatients.roche.com/
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
888-662-6728 (U.S. Only)
Query!
Fax
0
0
Query!
Email
0
0
[email protected]
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/study/NCT05987449
Download to PDF