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NCT05987449

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

Recruiting
PHASE1Ages 2–59InterventionalTreatment
Hoffmann-La Roche
~60 participants
Updated 2026-07-06 on ClinicalTrials.gov
What's tested:NXT007

At a glance

Recruiting sites
11 of 14 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence and Severity of Adverse Events, with Severity Determined According to National Cancer Institute Common Terminology Criteria for Adverse Events Grading Scale
Measured over From Baseline until study completion or discontinuation (up to 7.5 years)
+4 more outcomes measured

Conditions

Hemophilia A

Where it's being run

14 sites across 11 states
Lombardy2
Poland2
Spain2
California1
District of Columbia1
Indiana1
Iowa1
British Columbia1

Study leadership

  • Clinical Trials · STUDY_DIRECTOR · Hoffmann-La Roche

Who to contact

Reference Study ID Number: WP44714 https://forpatients.roche.com/ No attachments to email below.
[email protected]
888-662-6728 (U.S. Only)
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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 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

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:
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

What this trial measures

  • Incidence and Severity of Adverse Events, with Severity Determined According to National Cancer Institute Common Terminology Criteria for Adverse Events Grading ScaleFrom Baseline until study completion or discontinuation (up to 7.5 years)
  • Number of Participants with at Least One Clinical Laboratory Test Abnormality for Hematology ParametersFrom 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 ParametersFrom Baseline until study completion or discontinuation (up to 7.5 years)
  • Number of Participants with at Least One Vital Sign AbnormalityFrom 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) RecordingsFrom 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.