CClinicalTrials.gg
Sign In
RecruitingNCT05987449Updated Sep 11, 2026

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 interventional study of NXT007 in Hemophilia A, sponsored by Hoffmann-La Roche. Recruiting at 14 sites in 6 countries. Open to male participants aged 2 Years to 59 Years. Per ClinicalTrials.gov, last updated 2026-09-11.

Sponsored by Hoffmann-La Roche · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Started Sep 2023; still recruiting 3 years later.
Phase
Phase 1/2
Study type
Interventional
Enrollment
60
Allocation
Non-randomized
Ages
2 Years to 59 Years
Sex
Male
01

Study 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.
  • 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.

02

Conditions studied

  • Hemophilia A

Browse trials for

03

In context

Hemophilia A

866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.

This study's planned enrollment of 60 is above the median of 28 across 512 interventional studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Hoffmann-La Roche is the lead sponsor of 2,061 studies on the registry; 85 are open to participants now.

Of its 319 completed or terminated interventional studies of FDA-regulated products, 239 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 59 Years
Sexes eligible
Male
Accepts healthy volunteers
No

Inclusion criteria

  • 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
  • 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

Exclusion criteria

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
  • 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
  • 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:

    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; 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
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
60 participants (estimated)

Study arms

  • Experimental
    Part 1: Cohort 1 - NXT007 Dose Level 1 (Low)

    Drug: NXT007

  • Experimental
    Part 1: Cohort 2 - NXT007 Dose Level 2

    Drug: NXT007

  • Experimental
    Part 1: Cohort 3 - NXT007 Dose Level 3

    Drug: NXT007

  • Experimental
    Part 1: Cohort 4 - NXT007 Dose Level 4

    Drug: NXT007

  • Experimental
    Part 1: Cohort 5 - NXT007 Dose Level 5 (High)

    Drug: NXT007

  • Experimental
    Part 2: Cohort A - NXT007

    Drug: NXT007

Interventions

  • DrugNXT007

    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.

    Also known as: Zemocimig, RO7589655, RG6512

06

What researchers measure

Primary outcomes

  1. 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)

  2. 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)

  3. 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)

  4. 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)

  5. 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)

Secondary outcomes

  1. 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)

  2. Maximum Observed Plasma Concentration (Cmax) of NXT007 After the First Dose

    Time frame: At prespecified timepoints from Day 1 to Day 15

  3. Time to Maximum Observed Plasma Concentration (tmax) of NXT007 After the First Dose

    Time frame: At prespecified timepoints from Day 1 to Day 15

  4. 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

  5. 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)

  6. 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)

  7. 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)

  8. 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)

  9. 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)

  10. 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)

  11. 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)

  12. 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)

  13. 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)

  14. 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)

  15. 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)

  16. 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)

  17. 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)

  18. 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)

07

Study locations

12 of 14 sites recruiting
  • UC Davis Cancer Center
    Sacramento, California 95817, United States
    Recruiting
  • Georgetown Uni Medical Center
    Washington D.C., District of Columbia 20007, United States
    Withdrawn
  • Indiana Hemophilia & Thrombosis center
    Indianapolis, Indiana 46260, United States
    Recruiting
  • University of Iowa Hospitals and Clnics Dept of Pediatrics
    Iowa City, Iowa 52242, United States
    Recruiting
  • British Columbia Children's Hospital
    Vancouver, British Columbia V6H 3N1, Canada
    Recruiting
  • Hamilton Health Sciences Corporation
    Hamilton, Ontario L8N 3Z5, Canada
    Recruiting
  • IRCCS Ca' Granda Ospedale Maggiore Policlinico
    Milan, Lombardy 20122, Italy
    Recruiting
  • Istituto Clinico Humanitas
    Rozzano (MI), Lombardy 20089, Italy
    Recruiting
  • Auckland Cancer Trial Centre
    Auckland, 1023, New Zealand
    Recruiting
  • Uniwersyteckie Centrum Kliniczne
    Gda?sk, 80-214, Poland
    Recruiting
  • Instytut Hematologii i Transfuzjologii
    Warsaw, 02-776, Poland
    Recruiting
  • Hospital Sant Joan de Deu
    Esplugues de Llobregat, Barcelona 08950, Spain
    Recruiting
  • Hospital Universitario la Paz
    Madrid, 28046, Spain
    Recruiting
  • Hospital Regional Universitario Carlos Haya
    Málaga, 29010, Spain
    Active, not recruiting
08

References and documents

Individual participant data

Plan to share: 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

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 11, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05987449
Lead sponsor
Hoffmann-La Roche
Responsible party
Sponsor
First posted
Aug 14, 2023
Start date
Sep 21, 2023
Primary completion
Dec 11, 2033 (estimated)
Completion
Dec 11, 2033 (estimated)
Last update
Sep 11, 2026

Study contacts

Reference Study ID Number: WP44714 https://forpatients.roche.com/ No attachments to email below.
Contact
global-roche-genentech-trials@gene.com
888-662-6728 (U.S. Only)
Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry
Contact
Clinical Trials
study director · Hoffmann-La Roche

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Interested in this study?

Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.

Contact study team

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Hemophilia A→

Other studies from this sponsor

Hoffmann-La Roche

Start the discussion