202. スミス・マギニス症候群 Smith-Magenis syndrome Clinical trials / Disease details
臨床試験数 : 10 / 薬物数 : 12 - (DrugBank : 5) / 標的遺伝子数 : 4 - 標的パスウェイ数 : 7
| 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 | EUCTR2017-000387-14-GR (EUCTR) | 10/01/2020 | 01/11/2019 | Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesity | Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesity | - POMC/PCSK1/LEPR heterozygous- POMC/PCSK1/LEPR compound heterozygous or homozygous deficiency obesity- POMC/PCSK1/LEPR composite heterozygous deficiency obesity- Smith-Magenis Syndrome - SH2B1 deficiency obesity- Chromosomal rearrangement of the 16p11.2 locus causing obesity- CPE compound heterozygous or homozygous deficiency obesity- Leptin deficiency obesity with loss of response to metreleptin- SRC1 deficiency obesity- MC4R deficiency obesity;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: setmelanotide Product Code: RM-493 INN or Proposed INN: setmelanotide Other descriptive name: RM-493 | Rhythm Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 222 | Phase 2 | France;United States;Canada;Greece;Spain;Israel;Netherlands;Germany;United Kingdom | ||
| 2 | EUCTR2017-000387-14-GB (EUCTR) | 24/05/2017 | 17/03/2017 | Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesity | Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesity | - POMC/PCSK1/LEPR heterozygous- POMC/PCSK1/LEPR compound heterozygous or homozygous deficiency obesity- POMC/PCSK1/LEPR composite heterozygous deficiency obesity- Smith-Magenis Syndrome - SH2B1 deficiency obesity- Chromosomal rearrangement of the 16p11.2 locus causing obesity- CPE compound heterozygous or homozygous deficiency obesity- Leptin deficiency obesity with loss of response to metreleptin- SRC1 deficiency obesity- MC4R deficiency obesity;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: setmelanotide Product Code: RM-493 INN or Proposed INN: setmelanotide Other descriptive name: RM-493 | Rhythm Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 150 | Phase 2 | United States;France;Greece;Canada;Spain;Israel;Netherlands;Germany;United Kingdom | ||
| 3 | NCT03013543 (ClinicalTrials.gov) | January 2017 | 3/1/2017 | Setmelanotide Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity | Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity | Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic);Leptin Receptor Deficiency Obesity;Smith-Magenis Syndrome;Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder) | Drug: Setmelanotide | Rhythm Pharmaceuticals, Inc. | NULL | Active, not recruiting | 6 Years | N/A | All | 150 | Phase 2/Phase 3 | United States;Canada;France;Germany;Greece;Israel;Netherlands;Spain;United Kingdom |
| 4 | EUCTR2017-000387-14-FR (EUCTR) | 14/03/2019 | Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesity | Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesity - Basket Study | -LepR Deficiency Obesity -POMC Heterozygous Deficiency Obesity -POMC Epigenetic Deficiency Obesity -Bardet-Biedl syndrome -Alström syndrome -LEPR Heterozygous Deficiency Obesity -Bi-allelic, homozygous or compound heterozygous genetic status for either the POMC, PCSK1, or LEPR genes, with the loss-of-function variant for each allele conferring a severe obesity phenotype -Smith-Magenis Syndrome -SH2B1 Haploinsufficiency -Carboxypeptidase E deficiency -Leptin deficient obesity;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: setmelanotide Product Code: RM-493 INN or Proposed INN: setmelanotide Other descriptive name: RM-493 | Rhythm Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 100 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom;France;Canada;Australia;Germany;Netherlands;Israel;Turkey;Ireland;Spain;Greece;Portugal;United States |