Clinical Trials List
Protocol NumberNXT001JG
NCT Number(ClinicalTrials.gov Identfier)NCT
Active
2020-04-01 - 2026-09-01
Phase I/II
Recruiting5
ICD-10D66
Hereditary factor VIII deficiency
ICD-9286.0
Congenital factor VIII disorder
A Phase I/II Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Healthy Volunteers and Patients with Hemophilia A
-
Trial Applicant
Chugai Pharma Taiwan Ltd.
-
Sponsor
Chugai Pharma Taiwan Ltd.
-
Trial scale
Multi-Regional Multi-Center
-
Update
2026/02/01
Investigators and Locations
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Yen-Lin Liu Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Hemophilia A
Objectives
Primary Objectives
• To evaluate the safety and tolerability of single or multiple dose(s) of NXT007 in HVs
or patients
• To investigate the PK of single or multiple dose(s) of NXT007 in HVs or patients
• To investigate the PD of single or multiple dose(s) of NXT007 in HVs or patients
• To assess the efficacy of multiple doses of NXT007 in patients
Secondary Objectives
• To investigate the immunogenicity of single or multiple dose(s) of NXT007 in HVs or
patients
• To assess the QoL in patients
• To investigate the PK, PD, efficacy, and immunogenicity of multiple doses of
emicizumab in patients
• To assess the efficacy of NXT007 on joints in patients
Exploratory Objective
• To assess physical activity in patients
• To assess the QoL in caregivers
Test Drug
NXT007
Active Ingredient
NXT007
Dosage Form
Solution for subcutaneous injection
Dosage
80 mg
Endpoints
Primary Endpoints
• Safety and tolerability outcomes
− Incidence and severity of AEs and relationship to NXT007, including SAEs
− Incidence of abnormal laboratory findings
− Incidence of abnormal vital signs and ECG parameters
− Cytokines
− FVIII inhibitors (Parts B and C only)
• PK outcomes
− Plasma NXT007 concentration
• PD outcomes
− APTT
− Thrombin generation
− Plasma FIX concentration
− Plasma FX concentration
• Efficacy outcomes
− Bleeding events (e.g., bleed rate)
• Safety and tolerability outcomes
− Incidence and severity of AEs and relationship to NXT007, including SAEs
− Incidence of abnormal laboratory findings
− Incidence of abnormal vital signs and ECG parameters
− Cytokines
− FVIII inhibitors (Parts B and C only)
• PK outcomes
− Plasma NXT007 concentration
• PD outcomes
− APTT
− Thrombin generation
− Plasma FIX concentration
− Plasma FX concentration
• Efficacy outcomes
− Bleeding events (e.g., bleed rate)
Inclution Criteria
Inclusion Criteria
HVs and patients must meet the following criteria for study entry:
All HVs and Patients: Common Inclusion Criteria
• Signed ICF
• Able to comply with the study protocol, in the investigator’s judgment
• Male sex
• For men who are not surgically sterile: agreement to remain abstinent (refrain from
heterosexual intercourse) or use condoms, and agreement to refrain from donating
sperm, as defined below:
− With female partners of childbearing potential or pregnant female partners, men
must remain abstinent or use a condom during the treatment period and for at
least 5 half-lives or 12 months after the last dose of NXT007, whichever is longer to avoid exposing the embryo. Men must refrain from donating sperm
during this same period
− The reliability of sexual abstinence should be evaluated in relation to the
duration of the clinical trial and the preferred and usual lifestyle of the
HV/patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or
post-ovulation methods) and withdrawal are not acceptable methods of
contraception
All HVs: Additional Inclusion Criteria (Part A)
• Japanese race/ethnicity
• Age ≥20 and <45 years
• BMI at screening: (Body weight [kg]/height [m]2
) ≥18.5 to <25.0
• Absence of evidence of any active or chronic disease following a detailed medical and
surgical history and complete physical examination, vital signs, 12-lead ECG, and
laboratory tests
All Patients: Additional Inclusion Criteria (Parts B and C)
• Age ≥12 and <60 years at time of informed consent.
• Weight ≥40 kg at screening
• An ICF is signed by the patient. If the patient does not understand the ICF, for
example adolescents (age 12 to 17), an IAF is completed instead and the patient’s
parent (or caregiver) will complete an ICF.
All Emicizumab-Naive Patients: Additional Inclusion Criteria (Part B)
• Patients with hemophilia A who have not been treated with emicizumab
• Diagnosis of severe congenital hemophilia A without inhibitors as defined below (all
must be met)
− Endogenous FVIII level <1 IU/dL
− Negative test for inhibitors against FVIII (i.e., <0.6 BU/mL) within 8 weeks
prior to enrollment
− No documented inhibitors against FVIII (i.e., <0.6 BU/mL), FVIII t1/2 <6 hours,
or FVIII recovery <66% within 5 years prior to enrollment
− Use (or plan to use) FVIII products for episodic treatment
• Following items are documented
− Bleeding episodes (including time and site of bleeding) within 24 weeks prior to
enrollment
− Treatment with coagulation factors within 24 weeks prior to enrollment
Patients can be enrolled regardless of the number of bleeds.
All Emicizumab-Treated Patients: Additional Inclusion Criteria (Part C)
• Patients with hemophilia A who have been treated continuously with emicizumab at
an approved dosing regimen for at least 12 weeks prior to enrollment
• Diagnosis of severe congenital hemophilia A with or without inhibitors
− For a diagnosis with inhibitors as defined below (all must be met)
• Endogenous FVIII level <1 IU/dL
• Documented history of high-titer inhibitors against FVIII (i.e., ≥5 BU/mL)
• Use (or plan to use) bypassing agents for episodic treatment
− For a diagnosis without inhibitors as defined below (all must be met)
• Endogenous FVIII level <1 IU/dL
• Negative test for inhibitors against FVIII (i.e., <0.6 BU/mL) within 8 weeks
prior to enrollment
• No documented inhibitors against FVIII (i.e., <0.6 BU/mL), FVIII t1/2 <6
hours or FVIII recovery <66% within 5 years prior to enrollment
• Use (or plan to use) FVIII products for episodic treatment
• Following items are documented
− Bleeding episodes (including time and site of bleeding) within 24 weeks prior to
enrollment
− Treatment with coagulation factors and emicizumab within 24 weeks prior to
enrollment
Patients can be enrolled regardless of the number of bleeds.
HVs and patients must meet the following criteria for study entry:
All HVs and Patients: Common Inclusion Criteria
• Signed ICF
• Able to comply with the study protocol, in the investigator’s judgment
• Male sex
• For men who are not surgically sterile: agreement to remain abstinent (refrain from
heterosexual intercourse) or use condoms, and agreement to refrain from donating
sperm, as defined below:
− With female partners of childbearing potential or pregnant female partners, men
must remain abstinent or use a condom during the treatment period and for at
least 5 half-lives or 12 months after the last dose of NXT007, whichever is longer to avoid exposing the embryo. Men must refrain from donating sperm
during this same period
− The reliability of sexual abstinence should be evaluated in relation to the
duration of the clinical trial and the preferred and usual lifestyle of the
HV/patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or
post-ovulation methods) and withdrawal are not acceptable methods of
contraception
All HVs: Additional Inclusion Criteria (Part A)
• Japanese race/ethnicity
• Age ≥20 and <45 years
• BMI at screening: (Body weight [kg]/height [m]2
) ≥18.5 to <25.0
• Absence of evidence of any active or chronic disease following a detailed medical and
surgical history and complete physical examination, vital signs, 12-lead ECG, and
laboratory tests
All Patients: Additional Inclusion Criteria (Parts B and C)
• Age ≥12 and <60 years at time of informed consent.
• Weight ≥40 kg at screening
• An ICF is signed by the patient. If the patient does not understand the ICF, for
example adolescents (age 12 to 17), an IAF is completed instead and the patient’s
parent (or caregiver) will complete an ICF.
All Emicizumab-Naive Patients: Additional Inclusion Criteria (Part B)
• Patients with hemophilia A who have not been treated with emicizumab
• Diagnosis of severe congenital hemophilia A without inhibitors as defined below (all
must be met)
− Endogenous FVIII level <1 IU/dL
− Negative test for inhibitors against FVIII (i.e., <0.6 BU/mL) within 8 weeks
prior to enrollment
− No documented inhibitors against FVIII (i.e., <0.6 BU/mL), FVIII t1/2 <6 hours,
or FVIII recovery <66% within 5 years prior to enrollment
− Use (or plan to use) FVIII products for episodic treatment
• Following items are documented
− Bleeding episodes (including time and site of bleeding) within 24 weeks prior to
enrollment
− Treatment with coagulation factors within 24 weeks prior to enrollment
Patients can be enrolled regardless of the number of bleeds.
All Emicizumab-Treated Patients: Additional Inclusion Criteria (Part C)
• Patients with hemophilia A who have been treated continuously with emicizumab at
an approved dosing regimen for at least 12 weeks prior to enrollment
• Diagnosis of severe congenital hemophilia A with or without inhibitors
− For a diagnosis with inhibitors as defined below (all must be met)
• Endogenous FVIII level <1 IU/dL
• Documented history of high-titer inhibitors against FVIII (i.e., ≥5 BU/mL)
• Use (or plan to use) bypassing agents for episodic treatment
− For a diagnosis without inhibitors as defined below (all must be met)
• Endogenous FVIII level <1 IU/dL
• Negative test for inhibitors against FVIII (i.e., <0.6 BU/mL) within 8 weeks
prior to enrollment
• No documented inhibitors against FVIII (i.e., <0.6 BU/mL), FVIII t1/2 <6
hours or FVIII recovery <66% within 5 years prior to enrollment
• Use (or plan to use) FVIII products for episodic treatment
• Following items are documented
− Bleeding episodes (including time and site of bleeding) within 24 weeks prior to
enrollment
− Treatment with coagulation factors and emicizumab within 24 weeks prior to
enrollment
Patients can be enrolled regardless of the number of bleeds.
Exclusion Criteria
Exclusion Criteria
HVs and patients who meet any of the following criteria will be excluded from study
entry:
All HVs and Patients: Common Exclusion Criteria
• Previous or current history of drug- or alcohol-dependence
• Previous or concomitant TE disease such as DVT, or signs of TE disease or TMA
• Protein C activity (chromogenic assay), protein S free antigen, or antithrombin III
activity levels below the lower limit of the reference range at screening
• History of clinically significant allergy (anaphylactic shock or anaphylactoid
symptoms)
• Previous or concomitant autoimmune or connective tissue disease
• Previous or concomitant malignancy or leukemia
• History of hypersensitivity associated with globulin preparations
• Received or planned to receive a live vaccine within 4 weeks before the start of the
study drug administration
• Received or planned to receive an inactivated vaccine within 1 week before the start of
the study drug administration
• Planned surgery (including tooth extraction) during the study period
• Participated in another clinical study within 4 weeks before screening, or within 5
half-lives of the investigational product, whichever is longer
• Clinically significant ECG abnormalities at screening such as the following:
− The average QT interval with Fridericia’s correction of triplicate-measurements
in supine position at 10-minute rest >450 msec
− Bradycardia at rest (average heart rate <40 bpm)
− Tachycardia at rest (average heart rate >100 bpm)
− Other clinically significant abnormalities in ECG
• Any family history of congenital long QT syndrome or known congenital arrhythmia
• Any other reason that, in the judgment of the investigator, would render the subject
unsuitable for study participation
All HVs: Additional Exclusion Criteria (Part A)
• Clinically significant respiratory, circulatory, endocrine, hematological,
gastrointestinal, immune, psychological/nervous system, renal, hepatic, or allergic
disorder
• Family history of TE disorder such as serious DVT
• Use or planned use of other drugs (i.e., prescription or over-the-counter drugs) within
2 weeks before the start of study drug administration (provided, however, that nonsystemic topical antiseptics and ophthalmic solutions not affecting the PK or safety of
the study drug will be permitted)
• Blood draw of 200 mL within 4 weeks or 400 mL within 12 weeks prior to screening
(e.g., blood donations), annual total blood draw volume (including blood drawn for
this study) exceeding 1200 mL, or blood component donation within 2 weeks prior to
screening
• FVIII activity ≥120 IU/dL at screening
• Positive screening result for HIV antigen/antibodies, HBs antigen, or HCV antibodies.
HVs who showed HBs antigen positive due to vaccination against hepatitis B are
eligible
• Evidence of infection at screening
• Abnormal clinical findings at screening
• Prior treatment with antibody preparations (commercially available or investigational)
All Patients: Additional Exclusion Criteria (Parts B and C)
• Bleeding disorder other than congenital hemophilia A
• Use or planned use of drugs that affect hemostasis (i.e., prescription or over-thecounter drugs not including coagulation factors) within 2 weeks before the start of
study drug administration (provided, however, that non-systemic topical antiseptics and ophthalmic solutions not affecting the PK or safety of the study drug will be
permitted)
• CD4 count <200 cells/μL at screening
• Platelet count <100,000/μL at screening
• Serum creatinine ≥120% the upper limit of the study site’s reference range, or AST or
ALT ≥5-fold the upper limit of the site reference range at screening
• Clinically significant infection (except HBV, HCV, and HIV) at screening
• Use of systemic immunomodulators other than anti-retroviral therapy (e.g., interferon
and corticosteroids) at enrollment, or planned use during the study period
• Planned ITI therapy during the study period
• Use or planned use of coagulation factors within 72 hours or within 5 half-lives of the
coagulation factors, whichever is longer, before the start of study drug administration
• Treated with gene therapy or planned to start gene therapy
• Inability to tolerate MRI procedures or contraindication to MRI, including, but not
limited to, presence of pacemakers, aneurysm clips, artificial heart valves, ear
implants, or foreign metal objects in the eyes, skin, or body that would contraindicate
an MRI scan; or any other clinical history or examination finding that, in the
judgment of the investigator, would pose a potential hazard in combination with MRI
All Emicizumab-Naive Patients: Additional Exclusion Criteria (Part B)
• Prior treatment with antibody preparations (commercially available or investigational)
All Emicizumab-Treated Patients: Additional Exclusion Criteria (Part C)
• Prior treatment with antibody preparations (commercially available or investigational)
other than emicizumab
HVs and patients who meet any of the following criteria will be excluded from study
entry:
All HVs and Patients: Common Exclusion Criteria
• Previous or current history of drug- or alcohol-dependence
• Previous or concomitant TE disease such as DVT, or signs of TE disease or TMA
• Protein C activity (chromogenic assay), protein S free antigen, or antithrombin III
activity levels below the lower limit of the reference range at screening
• History of clinically significant allergy (anaphylactic shock or anaphylactoid
symptoms)
• Previous or concomitant autoimmune or connective tissue disease
• Previous or concomitant malignancy or leukemia
• History of hypersensitivity associated with globulin preparations
• Received or planned to receive a live vaccine within 4 weeks before the start of the
study drug administration
• Received or planned to receive an inactivated vaccine within 1 week before the start of
the study drug administration
• Planned surgery (including tooth extraction) during the study period
• Participated in another clinical study within 4 weeks before screening, or within 5
half-lives of the investigational product, whichever is longer
• Clinically significant ECG abnormalities at screening such as the following:
− The average QT interval with Fridericia’s correction of triplicate-measurements
in supine position at 10-minute rest >450 msec
− Bradycardia at rest (average heart rate <40 bpm)
− Tachycardia at rest (average heart rate >100 bpm)
− Other clinically significant abnormalities in ECG
• Any family history of congenital long QT syndrome or known congenital arrhythmia
• Any other reason that, in the judgment of the investigator, would render the subject
unsuitable for study participation
All HVs: Additional Exclusion Criteria (Part A)
• Clinically significant respiratory, circulatory, endocrine, hematological,
gastrointestinal, immune, psychological/nervous system, renal, hepatic, or allergic
disorder
• Family history of TE disorder such as serious DVT
• Use or planned use of other drugs (i.e., prescription or over-the-counter drugs) within
2 weeks before the start of study drug administration (provided, however, that nonsystemic topical antiseptics and ophthalmic solutions not affecting the PK or safety of
the study drug will be permitted)
• Blood draw of 200 mL within 4 weeks or 400 mL within 12 weeks prior to screening
(e.g., blood donations), annual total blood draw volume (including blood drawn for
this study) exceeding 1200 mL, or blood component donation within 2 weeks prior to
screening
• FVIII activity ≥120 IU/dL at screening
• Positive screening result for HIV antigen/antibodies, HBs antigen, or HCV antibodies.
HVs who showed HBs antigen positive due to vaccination against hepatitis B are
eligible
• Evidence of infection at screening
• Abnormal clinical findings at screening
• Prior treatment with antibody preparations (commercially available or investigational)
All Patients: Additional Exclusion Criteria (Parts B and C)
• Bleeding disorder other than congenital hemophilia A
• Use or planned use of drugs that affect hemostasis (i.e., prescription or over-thecounter drugs not including coagulation factors) within 2 weeks before the start of
study drug administration (provided, however, that non-systemic topical antiseptics and ophthalmic solutions not affecting the PK or safety of the study drug will be
permitted)
• CD4 count <200 cells/μL at screening
• Platelet count <100,000/μL at screening
• Serum creatinine ≥120% the upper limit of the study site’s reference range, or AST or
ALT ≥5-fold the upper limit of the site reference range at screening
• Clinically significant infection (except HBV, HCV, and HIV) at screening
• Use of systemic immunomodulators other than anti-retroviral therapy (e.g., interferon
and corticosteroids) at enrollment, or planned use during the study period
• Planned ITI therapy during the study period
• Use or planned use of coagulation factors within 72 hours or within 5 half-lives of the
coagulation factors, whichever is longer, before the start of study drug administration
• Treated with gene therapy or planned to start gene therapy
• Inability to tolerate MRI procedures or contraindication to MRI, including, but not
limited to, presence of pacemakers, aneurysm clips, artificial heart valves, ear
implants, or foreign metal objects in the eyes, skin, or body that would contraindicate
an MRI scan; or any other clinical history or examination finding that, in the
judgment of the investigator, would pose a potential hazard in combination with MRI
All Emicizumab-Naive Patients: Additional Exclusion Criteria (Part B)
• Prior treatment with antibody preparations (commercially available or investigational)
All Emicizumab-Treated Patients: Additional Exclusion Criteria (Part C)
• Prior treatment with antibody preparations (commercially available or investigational)
other than emicizumab
The Estimated Number of Participants
-
Taiwan
16 participants
-
Global
76 participants
