34. Neurofibromatosis Clinical trials / Disease details
Clinical trials : 133 / Drugs : 186 - (DrugBank : 67) / Drug target genes : 79 - Drug target pathways : 190
| 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 | NCT03433183 (ClinicalTrials.gov) | October 2, 2019 | 29/1/2018 | SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors | SARC031: A Phase 2 Trial of the MEK Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Combination With the mTOR Inhibitor Sirolimus for Patients With Unresectable or Metastatic Malignant Peripheral Nerve Sheath Tumors | Malignant Peripheral Nerve Sheath Tumors;Neurofibromatosis 1 | Drug: Selumetinib;Drug: Sirolimus | Sarcoma Alliance for Research through Collaboration | United States Department of Defense;AstraZeneca | Active, not recruiting | 12 Years | N/A | All | 21 | Phase 2 | United States |
| 2 | JPRN-UMIN000021030 | 2016/03/16 | 22/02/2016 | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1 | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1 - A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1 | Neurofibromatosis type 1 | 0.2% Sirolimus gel twice daily 24 weeks topical application 0.4% Sirolimus gel twice daily 24 weeks topical application Placebo gel twice daily 24 weeks topical application | Osaka University Hospital | NULL | Complete: follow-up complete | 16years-old | 70years-old | Male and Female | 18 | Phase 2 | Japan |
| 3 | JPRN-UMIN000015081 | 2014/09/08 | 07/09/2014 | Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1 | Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1 - Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1 | neurofibromatosis type1 | 1) Application of 0.2% rapamycin gel on one third of the tumor once a day for 2 weeks. 2) Check the skin findings, blood test results, blood level of rapamycin and adverse events after 2 weeks of treatment. If there is no problem, perform application of 0.2% rapamycin gel on two-thirds of the tumor once a day for another 2 weeks. 3) Check the skin findings, blood test results, blood level of rapamycin and adverse events after a total of 4 weeks of treatment. If there is no problem, perform application of 0.2% rapamycin gel on the whole surface of the tumor once a day for another 8 weeks. | Department of Dermatology Graduate School of Medicine, Osaka University | NULL | Recruiting | 20years-old | Not applicable | Male and Female | 3 | Phase 2,3 | Japan |
| 4 | NCT01031901 (ClinicalTrials.gov) | December 2009 | 10/12/2009 | Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1) | Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1 | Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;Neurofibroma | Drug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycin | The University of Texas Health Science Center, Houston | Society for Pediatric Dermatology | Completed | 13 Years | N/A | Both | 52 | Phase 1 | United States |
| 5 | NCT00634270 (ClinicalTrials.gov) | April 2008 | 20/2/2008 | A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas | A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas | Neurofibromatosis Type 1 | Drug: Sirolimus | University of Alabama at Birmingham | Boston Children’s Hospital;Children's Hospital of Philadelphia;Children's Research Institute;Children's Hospital Medical Center, Cincinnati;National Cancer Institute (NCI);University of Chicago;University of Utah;Washington University School of Medicine | Completed | 3 Years | 75 Years | All | 58 | Phase 2 | United States |
| 6 | NCT00652990 (ClinicalTrials.gov) | March 2008 | 3/4/2008 | Sirolimus to Treat Plexiform Neurofibromas in Patients With Neurofibromatosis Type I | A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas | Neurofibromatosis Type 1;Plexiform Neurofibromas;Paraspinal Plexiform Neurofibromas | Drug: Sirolimus | University of Alabama at Birmingham | National Cancer Institute (NCI) | Active, not recruiting | 3 Years | N/A | Both | 18 | Phase 2 | United States |
| 7 | NCT00901849 (ClinicalTrials.gov) | May 2007 | 13/5/2009 | Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1) | Phase 1 Study of Tarceva and Rapamycin For Recurrent Low-Grad Gliomas in Children With or Without Neurofibromatosis Type 1 (NF1) | Low-grade Gliomas | Drug: Tarceva and Rapamycin | Roger Packer | Children's Research Institute | Completed | N/A | 21 Years | Both | 21 | Phase 1 | United States |