Hemophilia APhase 1/2NCT05987449
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
A Phase 1/2 study testing NXT007 in people with Hemophilia A, run by Hoffmann-La Roche at 14 sites in 6 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 59 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- NXT007
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 14 sitesin 6 countries
- Study dates
- 21 Sep 2023to 11 Dec 2033
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 59 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of severe (Factor VIII coagulant activity [FVIII:C] <1 IU/dL) or moderate (FVIII:C ≥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
Show all 10Show fewer
- Adequate hepatic function defined as total bilirubin ≤1.5× age-adapted upper limit of normal (ULN) (excluding Gilbert syndrome) and both aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3× age-adapted ULN at the time of screening, and no clinical signs or known laboratory/radiographic evidence consistent with cirrhosis. For patients with Gilbert syndrome, bilirubin should be <4 mg/dL or 68.4 umol/L at the time of screening.
- For Part 1 only: Adequate renal function, defined as serum creatinine ≤2.5× age-adapted ULN and calculated creatinine clearance ≥30 mL/min by Cockroft-Gault formula
- For Part 2 only: Adequate renal function, defined as serum creatinine ≤1.5× age-adapted ULN. When the serum creatinine is ≥1.5× ULN, creatinine clearance by Bedside Schwartz formula must be >70 mL/min/1.73m^2.
- Willingness and ability to comply with schedules visits, treatment plans, laboratory tests, and other study procedures
Who cannot join
- 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
- For Part 1 only: Personal history of ischemic heart disease, cerebrovascular disease, or diabetes mellitus
- For Part 1 only: 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 and females under the age of 65 years
Show all 19Show fewer
- For Part 1 only: Previous or concomitant malignancies or leukemia
- 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
- Receipt of any of the following:
- 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; v) Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or planned use during the study, with the exception of anti-retroviral therapy to treat HIV.
- 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
Trial locations and contacts
14 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
Reference Study ID Number: WP44714 https://forpatients.roche.com/ No attachments to email below. contact
Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry contact
14 sites in 6 countries
United States4 sites, 3 recruiting
- Recruiting
Indiana Hemophilia & Thrombosis center
Indianapolis, Indiana, 46260
- Recruiting
University of Iowa Hospitals and Clnics Dept of Pediatrics
Iowa City, Iowa, 52242
- Recruiting
UC Davis Cancer Center
Sacramento, California, 95817
- Withdrawn
Georgetown Uni Medical Center
Washington D.C., District of Columbia, 20007
Spain3 sites, 2 recruiting
- Recruiting
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, 08950
- Recruiting
Hospital Universitario la Paz
Madrid, 28046
- Active, not recruiting
Hospital Regional Universitario Carlos Haya
Málaga, 29010
Canada2 sites, 2 recruiting
- Recruiting
Hamilton Health Sciences Corporation
Hamilton, Ontario, L8N 3Z5
- Recruiting
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3N1
Italy2 sites, 2 recruiting
- Recruiting
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy, 20122
- Recruiting
Istituto Clinico Humanitas
Rozzano (MI), Lombardy, 20089
Poland2 sites, 2 recruiting
- Recruiting
Uniwersyteckie Centrum Kliniczne
Gda?sk, 80-214
- Recruiting
Instytut Hematologii i Transfuzjologii
Warsaw, 02-776
New Zealand1 site, 1 recruiting
- Recruiting
Auckland Cancer Trial Centre
Auckland, 1023
About this study
Official 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
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.
- Conditions
- Hemophilia A
- Being tested
- NXT007
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NXT007Drug
Also called Zemocimig, RO7589655, RG6512
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.
| Study group | Role | What they receive |
|---|---|---|
| Part 1: Cohort 1 - NXT007 Dose Level 1 (Low) | Experimental | Not described Receives: Drug: NXT007 |
| Part 1: Cohort 2 - NXT007 Dose Level 2 | Experimental | Not described Receives: Drug: NXT007 |
| Part 1: Cohort 3 - NXT007 Dose Level 3 | Experimental | Not described Receives: Drug: NXT007 |
| Part 1: Cohort 4 - NXT007 Dose Level 4 | Experimental | Not described Receives: Drug: NXT007 |
| Part 1: Cohort 5 - NXT007 Dose Level 5 (High) | Experimental | Not described Receives: Drug: NXT007 |
| Part 2: Cohort A - NXT007 | Experimental | Not described Receives: Drug: NXT007 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Adverse Events, with Severity Determined According to National Cancer Institute Common Terminology Criteria for Adverse Events Grading Scale
Measured: 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
Measured: 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
Measured: 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.
Measured: 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.
Measured: From Baseline until study completion or discontinuation (up to 7.5 years)
Secondary outcomes (18)
Plasma Concentration of NXT007 at Specified Timepoints
Measured: 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
Measured: At prespecified timepoints from Day 1 to Day 15
Time to Maximum Observed Plasma Concentration (tmax) of NXT007 After the First Dose
Measured: At prespecified timepoints from Day 1 to Day 15
Area Under the Plasma Concentration-Time Curve (AUC) of NXT007 After the First Dose
Measured: 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
Measured: 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
Measured: 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
Measured: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Mean Calculated Annualized Bleeding Rate for Treated Bleeds
Measured: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Median Calculated Annualized Bleeding Rate for Treated Bleeds
Measured: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Model-Based Annualized Bleeding Rate for All Bleeds
Measured: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Mean Calculated Annualized Bleeding Rate for All Bleeds
Measured: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Median Calculated Annualized Bleeding Rate for All Bleeds
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: From first dose of study drug until study completion or discontinuation (up to 7.5 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2023
- Study start21 Sep 2023
- Primary completion11 Dec 2033 (estimated)
- Study completion11 Dec 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05987449
- Sponsor's protocol ID
- WP44714
- Other IDs
- 2023-503906-35-00, 2023
- Status last verified
- Nov 2026
- First submitted
- 4 Aug 2023
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05987449
- Acronym / sponsor ID
- WP44714
- Conditions
- Hemophilia A
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 21 Sep 2023 (actual)
- Primary completion
- 11 Dec 2033 (estimated)
- Study completion
- 11 Dec 2033 (estimated)
- First posted
- 14 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Part 1: Cohort 1 - NXT007 Dose Level 1 (Low) | Experimental | Drug: NXT007 |
| Part 1: Cohort 2 - NXT007 Dose Level 2 | Experimental | Drug: NXT007 |
| Part 1: Cohort 3 - NXT007 Dose Level 3 | Experimental | Drug: NXT007 |
| Part 1: Cohort 4 - NXT007 Dose Level 4 | Experimental | Drug: NXT007 |
| Part 1: Cohort 5 - NXT007 Dose Level 5 (High) | Experimental | Drug: NXT007 |
| Part 2: Cohort A - NXT007 | Experimental | Drug: NXT007 |
Who may qualify
- Ages
- 2 to 59 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Diagnosis of severe (Factor VIII coagulant activity [FVIII:C] <1 IU/dL) or moderate (FVIII:C ≥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
Key exclusion criteria (19 listed)
- 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
- For Part 1 only: Personal history of ischemic heart disease, cerebrovascular disease, or diabetes mellitus
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NXT007Also: Zemocimig, RO7589655, RG6512Participants 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. | Drug | Part 1: Cohort 1 - NXT007 Dose Level 1 (Low), Part 1: Cohort 2 - NXT007 Dose Level 2, Part 1: Cohort 3 - NXT007 Dose Level 3, Part 1: Cohort 4 - NXT007 Dose Level 4, Part 1: Cohort 5 - NXT007 Dose Level 5 (High), Part 2: Cohort A - NXT007 |
Primary outcome
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
Time frame: From Baseline until study completion or discontinuation (up to 7.5 years)
The vital signs that will be assessed are body temperature, pulse rate, respiratory rate, and systolic and diastolic blood pressure.
Number of Participants with at Least One Abnormality on Electrocardiogram (ECG) Recordings
Time frame: From Baseline until study completion or discontinuation (up to 7.5 years)
The ECG parameters that will be assessed are heart rate, PR interval, QRS interval, QT interval, and QTcF inteval.
18 secondary outcomes
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)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- Please use this form to submit your questions for a faster response: https://www.gene.com/contact-us/submit-medical-inquiry. Do not include or attach any medical records when emailing or completing the form. A nurse will respond within 24 business hours.Link provided by the sponsor.
- Individual participant dataSharing planned: No. For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Reference Study ID Number: WP44714 https://forpatients.roche.com/ No attachments to email below. contact
Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry contact
Clinical Trials study director
Hoffmann-La Roche
14 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NXT007 in people with Hemophilia A, run by Hoffmann-La Roche at 14 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Spain, Canada, Italy, Poland and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Reference Study ID Number: WP44714 https://forpatients.roche.com/ No attachments to email below. (888-662-6728 (U.S. Only), global-roche-genentech-trials@gene.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026.