277. リンパ管腫症／ゴーハム病
［臨床試験数：7，薬物数：2（DrugBank：2），標的遺伝子数：1，標的パスウェイ数：50］
Searched query = "Lymphangiomatosis", "Generalized lymphatic anomaly", "Gorham disease", "Gorham-Stout disease", "Diffuse lymphangiomatosis", "Mass osteolysis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-UMIN000038973 | 2020/01/06 | 25/12/2019 | A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomalies | A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomalies - Sirolimus for Intractable Vascular Anomalies(SIVA) | Kaposiform hemangioendothelioma or Tufted angiomaLymphangioma (cystic lymphatic malformation), lymphangiomatosis (generalized lymphatic anomaly) or Gorham-Stout diseaseVenous malformation or blue rubber bleb nevus syndromeComplex-combined vascular malformations or Klippel-Trenanay-Weber syndrome | An initial dose of sirolimus is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. | Gifu University Hospital | NULL | Pending | 1months-old | Not applicable | Male and Female | 10 | Phase 3 | Japan |
| 2 | JPRN-jRCTs031180290 | 16/11/2017 | 15/03/2019 | A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies | A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies - Sirolimus for intractable vascular anomalies | Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lympha Vascular disorders | Body surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day. BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day. | Ozeki Michio | NULL | Recruiting | Not applicable | Not applicable | Both | 100 | Phase 3 | Japan |
| 3 | JPRN-UMIN000030522 | 2017/11/14 | 22/12/2017 | A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies | Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lymphatic anomaly, Kaposiform lymphangiomatosis), Gorham-Stout disease, Kaposiform hemangioendothelioma and Tuffted angioma with Kasabach-Merritt phenomenon, Nenous malformation, Arteriovenous malformation, Klippel-Trenaunay-Weber syndrome,Bluerubber bleb nevus syndrome, Complex-combined vascular malformations | Body surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day. BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day. | Gifu University | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 50 | Not selected | Japan | |
| 4 | JPRN-UMIN000029843 | 2017/11/06 | 06/11/2017 | Sirolimus for the treatment of refractory lymphangiomatosis/ Gorham Stout disease | Refractory lymphangiomatosis, Gorham-Stout disease | Oral sirolimus | Kyushu University Hospital | NULL | Recruiting | 8years-old | 30years-old | Male and Female | 2 | Not selected | Japan | |
| 5 | JPRN-UMIN000016580 | 2015/02/01 | 19/02/2015 | Sirolimus for the treatment of lymphangiomatosis, Gorham-Stout disease and vascular anomalies | Sirolimus for the treatment of lymphangiomatosis, Gorham-Stout disease and vascular anomalies - Sirolimus for vascular anomalies | Lymphangiomatosis, Gorham-Stout disease and vascular anomalies | Oral sirolimus | Gifu university | NULL | Complete: follow-up complete | Not applicable | 30years-old | Male and Female | 30 | Phase 1;Phase 2 | Japan |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT00975819 (ClinicalTrials.gov) | October 2009 | 10/9/2009 | Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies | A Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular Anomalies | Kaposiform Hemangioendotheliomas;Tufted Angioma;Capillary Venous Lymphatic Malformation;Venous Lymphatic Malformation;Microcystic Lymphatic Malformation;Mucocutaneous Lymphangiomatosis and Thrombocytopenia;Capillary Lymphatic Arterial Venous Malformations;PTEN Overgrowth Syndrome With Vascular Anomaly;Lymphangiectasia Syndromes | Drug: sirolimus | Children's Hospital Medical Center, Cincinnati | NULL | Active, not recruiting | N/A | 31 Years | Both | 60 | Phase 2 | United States |