A Phase III Study of FPF300

JRCT ID: jRCT2071230066

Registered date:16/09/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Recurrent epistaxis in Hereditary Hemorrhagic Telangiectasia
• Date of first enrollment: 27/11/2023
• Target sample size: 44
• Study type: Interventional
• Intervention(s): FPF300 is orally administered once daily. The initial daily doses will be started at 50 mg and adjusted the dose depending on the patient's condition.

Outcome(s)

Primary Outcome

The changing of Epistaxis Severity Score compared to baseline
Adverse event

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1) Patients who have diagnosed as definite HHT by the Curacao criteria, or certified as designated intractable diseases 227(HHT)
2) Patients with an Epistaxis Severity Score (ESS) >= 4.01 who have epistaxis at least three times a week on average since 4weeks before initial patient's consent
3) Patients 18 or older at the time of their initial consent
4) Patients who adhere to FPF300 risk management system

Exclude criteria

1) Women in pregnancy, breastfeeding or child bearing
Men who wish their partners to become pregnant during the study period
2) Patients who have previously been prescribed thalidomide
3) Patients who have been prescribed the following agents since 12months before initial patient's consent
a) thalidomide derivatives (lenalidomide and pomalidomide)
b) angiogenesis inhibitors (e.g. bevacizumab, pazopanib)
4) Patients who have the medical history(except for a)) or complications below
a) anemia by reason of other than HHT
b) hemorrhagic disease (e.g. hemophilia) by reason of other than HHT
c) pulmonary embolism / deep vein thrombosis
d) bradycardic arrhythmia
e) heart failure
f) pulmonary hypertension
g) active cancer within 5 years of disease free interval
5) BNP >= 100 pg/mL or NT-pro BNP >= 300 pg/mL
6) Patients who have a high risk of deep vein thrombosis
7) Patients who are positive for hepatitis B antigen, hepatitis C antibody, or HIV antibody

Related Information

• Primary Sponsor: Kobayashi Hiromi