Efficacy and safety of Sirolimus granules twice daily administration in patients with intractuable vascular malformations
- Conditions
- Intractable vascular malformationsVascular disordersD054079
- Registration Number
- JPRN-jRCTs031200055
- Lead Sponsor
- Mochizuki Shinji
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 11
1) one month and older
2) Patients with intractable vascular anomaly diagnosed by the investigator/subinvestigator
3) Have one or more measurable target lesions on MRI before treatment starts.
4) Severe disability or refractory symptoms depending on target disease
5) Have sufficient liver, kidney and heart functions at the time of registration
6) Written consent to participate in this clinical trial has been given by the subject in person or by a legal guardian (when the subject is younger than 20 years at consent).
1) Patients who have received a targeted drug related to the mTOR pathway within 8 weeks before the start of study drug administration.
2) Patients with infections that require systemic treatment.
3) Patients with a Karnofsky PS score of 30 or less (10 years or older) or Lansky play-PS of 30 or less (under 10 years) due to permanent sequelae due to cerebral disorders.
4) Patients with any of the following complications:
Uncontrolled diabetes, Uncontrolled hypertension, Uncontrolled hyperlipidemia, Severe liver disease, Severe renal disease
5) Patients receiving long-term immunosuppressive drugs (cyclosporine, tacrolimus, etc.) or steroids (4 weeks or more) at the time of registration.
6) Patients who require administration of a drug that affects CYP3A4 activity one week before starting sirolimus administration.
7) Patients with immunodeficiency such as HIV and primary immunodeficiency.
8) Patients who are carriers of hepatitis B virus and / or carriers of hepatitis C virus.
9) Patients who have undergone surgery (resection, sclerotherapy, endovascular treatment) for the target lesion within 2 weeks before obtaining consent.
10) Patients who have received a therapeutic drug (propranolol, Eppikajutsuto, Tokikenchuto, interferon, octreotide, bisphosphonate, denosumab, etc.) for the target disease within 2 weeks before obtaining consent.
11) Patients who received myelosuppressive chemotherapy, biologics, drugs not covered by insurance, etc. within 4 weeks before obtaining consent.
12) Patients who received radiotherapy for the target lesion within 24 weeks before obtaining consent.
13) Patients who meet any of the following.
may be pregnant or pregnant, lactating, Disagree with contraception during this study
14) If administering tablets, persons diagnosed with lymphangioma, lymphangiomatosis, or Gorham's disease.
15) In addition, patients whose investigator / assigning doctor determines that participation in this study is inappropriate.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Target lesion response rate determined by Independent Review Facility after 24 weeks of treatments
- Secondary Outcome Measures
Name Time Method Response rates of target lesions 12, 52 weeks after administration.<br>Improvement of lesions other than target lesions (skin lesions) at 12, 24, and 52 weeks after administration<br>Respiratory function 24 and 52 weeks after administration<br>Pleural effusion 12, 24 and 52 weeks after administration.<br>Ascites 12, 24, and 52 weeks after administration<br>Anemia and blood coagulation parameters at 12, 24, and 52 weeks after <br>administration<br>Bleeding at 12, 24, and 52 weeks after administration<br>Pain at 12, 24 and 52 weeks after administration<br>QOL improvement after starting treatment<br>ADL improvement after starting treatment<br>Adverse events and side effects<br>Clinical test values vital signs<br>Pharmacokinetics