Clinical Trial of NXT007 for the Prophylactic Treatment of Hemophilia A in Infants and Children

ConditionHemophilia A

Trial statusNot yet recruiting

Trial phasePhase 3

Trial typeInterventional

Biological sexAll

Age0-17

SponsorHoffmann-La Roche

About this trial

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis in pediatric patients aged 0 to 11 years with severe or moderate congenital hemophilia A without factor VIII inhibitors or congenital hemophilia A of any severity (severe, moderate, and mild) with inhibitors (emicizumab-naïve and treated). Patients with hemophilia A aged ≥12 to \<18 years old with a body weight of \<40 kilograms (kg) are also able to participate.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age <12 years at the time of signing Informed Consent Form; or age ≥12 and <18 years with body weight <40 kg

Diagnosis of severe (Factor VIII coagulation protein activity [FVIII:C] <1 International Unit per decilitre [IU/dL]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A (HA) with or without inhibitors against factor VIII (FVIII)

For potential participants with moderate HA without inhibitors, fulfillment of at least one of the following criteria: Current prophylaxis with long-term prophylaxis intention due to a severe bleed phenotype; For those not treated with prophylaxis: at least one traumatic joint or critical muscle bleed in the last 6 months or ≥2 spontaneous bleeds/year or 5 bleeds/year, including traumatic bleeds; Signs of joint degeneration compatible with hemophilic arthropathy (e.g., subchondral bone changes or the presence of synovitis) as assessed by any imaging assessment (e.g., ultrasound, MRI, radiograph); Bleeding at a critical site (e.g., intracranial).

Diagnosis of mild (FVIII:C between >5 IU/dL and <40 IU/dL) congenital hemophilia A with chronic FVIII inhibitors, defined as documented FVIII inhibitor (≥0.6 BU/mL or ≥1.0 BU/mL only for laboratories with a historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and chronic reduction of endogenous baseline FVIII:C to <5 IU/dL for ≥12 months

Disqualifiers

Sensitivity to any of the study investigations, or components thereof, or drug or other allergy that, in the opinion of the investigator, contraindicates participation in the study

Use of systemic immunomodulators (e.g., interferon or rituximab) at the time of enrollment or planned use during the study, except for antiretroviral therapy to treat HIV

Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario

History or conditions, other than HA, which may indicate hypo- or hypercoagulopathy risk

Trial design

Design model

Single group

Treatments tested in this trial

NXT007
Drug
NXT007 will be administered subcutaneously (SC) according to the schedule in the protocol.

Treatment groups

50 Participants

are divided into 1 treatment group

Group A: NXT007 ProphylaxisExperimental treatment 1 intervention