臨床研究等提出・公開システム

Multi-center clinical study to describe the relationship between anti-emicizumab antibodies and plasma emicizumab concentration and various coagulation tests

ACANE Study

Keiji Nogami

Nara Medical University Hospital

840 Shijo-Cho, Kashihara, Nara

roc-noga@naramed-u.ac.jp

Keiji Nogami

Nara Medical University Hospital

840 Shijo-Cho, Kashihara, Nara

+81-744-22-3051

roc-noga@naramed-u.ac.jp

Recruiting

Oct. 01, 2024

Observational

single arm study

open(masking not used)

uncontrolled control

single assignment

other

Patients who will be collected blood samples prospectively after study enrollment.
1) Informed consent to participate in the study has been obtained from the patient personally or from a legal representative using the Informed Consent Form. If possible, assent to participate in the study will be obtained from pediatric patients personally using the Informed Assent Form.
2) Diagnosis of congenital hemophilia A or acquired hemophilia A at the time of enrollment.
3) Investigator have confirmed both of the following conditions at the time of blood sample submission to the Measurement Program prior to enrollment.
a. Recieving emicizumab in accordance with the indications and dosage and administration of the package insert.
b. Suspect of having a decreased plasma emicizumab concentration, as evidenced by prolonged APTT
and increased bleeding frequency, etc.

Patients who have been stored blood samples at the central laboratory and will not be collected new blood samples after study enrollment.
1) Patients who meet any of the following criteria
a. Informed consent to participate in the study has been obtained from the patient personally or from a legal representative
using the Informed Consent Form. If possible, assent to participate in the study will be obtained from pediatric patients
personally using the Informed Assent Form.
b. Patients who have not indicated their refusal during the period when the documents regarding the clinical study and use of
existing samples are posted on the website of the medical institution, etc.
2) Diagnosis of congenital hemophilia A or acquired hemophilia A at the time of initial sample submission to the Measurement Program.
3) Patients who Investigator have confirmed both of the following conditions at the first time of blood sample submission to the Measurement Program.
a. Recieving emicizumab in accordance with the indications and dosage and administration of the package insert.
b. Suspect of having a decreased plasma emicizumab concentration, as evidenced by prolonged APTT and increased bleeding
frequency, etc.

1) Inherited or acquired bleeding disprder other than congenital hemophilia A and acquired hemophilia A
2) In the judge of the investigator, patient would be unsuitable for study participation

Both

Congenital hemophilia A and acquired hemophilia A

The following endpoints will be described in congenital hemophilia A and aquired hemophilia A patients who are suspected of having decreased plasma emicizumab concentrations, as evidenced by prolonged APTT and increased bleeding frequency, etc., and who have tested positive for anti-emicizumab antibodies
APTT
Anti-emicizumab antibodies (antibody titer and presence of neutralizing activity)
- Plasma emicizumab concentration
- FVIII activity (emicizumab non-response, one-stage clotting assay of emicizumab-neutralizing activity)
- FVIII activity (emicizumab response, chromogenic assay using human coagulation factor)
- FVIII inhibitor titer
- Clot waveform analysis
- Thrombin generation test

The following endpoints will be described in congenital hemophilia A and aquired hemophilia A patients who are suspected of having decreased plasma emicizumab concentrations, as evidenced by prolonged APTT and increased bleeding frequency, etc., and who have tested negative for anti-emicizumab antibodies
- APTT
- Plasma emicizumab concentration
- FVIII activity (emicizumab non-response, one-stage clotting assay of emicizumab-neutralizing activity)
- FVIII activity (emicizumab response, chromogenic assay using human coagulation factor)
- FVIII inhibitor titer
- Clot waveform analysis
- Thrombin generation test
Changes over time in anti-emicizumab antibody titers, coagulation test values and clinical findings in anti-emicizumab antidody-positive patients.
Characteristic analysis of anti-emicizumab antibodies.
Exploratory study on future hemophilia therapeutic drug.

+81-3-6416-1868

Oct. 05, 2023

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