277. Lymphangiomatosis Clinical trials / Disease details
Clinical trials : 6 / Drugs : 2 - (DrugBank : 2) / Drug target gene : 1 - Drug target pathways : 51
| 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 | Complete: follow-up continuing | 1months-old | Not applicable | Male and Female | 10 | Phase 3 | Japan |
| 2 | 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 | |
| 3 | 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 | |
| 4 | 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 1,2 | Japan |
| 5 | JPRN-UMIN000014888 | 2014/06/01 | 19/08/2014 | Everolimus for the treatment of lymphangiomatosis, Gorham-Stout disease and vascular anomalies | Lymphangiomatosis, Gorham-Stout disease and vascular anomalies | Oral everolimus | Gifu university | NULL | Complete: follow-up complete | Not applicable | 30years-old | Male and Female | 30 | Phase 1 | Japan | |
| 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 |