202. スミス・マギニス症候群 Smith-Magenis syndrome Clinical trials / Disease details
臨床試験数 : 9 / 薬物数 : 10 - (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 | NCT04768803 (ClinicalTrials.gov) | March 15, 2021 | 16/2/2021 | Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or Obesity | Circulating Levels of Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and / or Overweight, and / or Obesity | Angelman Syndrome;Smith-Magenis Syndrome;X Fragile Syndrome;Epilepsy;Prader-Willi Syndrome | Biological: acylated and unacylated ghrelin dosages | University Hospital, Toulouse | NULL | Not yet recruiting | 3 Years | 50 Years | All | 300 | NULL | |
| 2 | 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 | ||
| 3 | NCT03492970 (ClinicalTrials.gov) | March 7, 2018 | 1/3/2018 | Melatonin in Adults With SMS | Characterization of Behavioral Disorders and 24 H-melatonin Level in Adults With Smith Magenis Syndrome | Smith Magenis Syndrome | Biological: Hourly dosing of the nychtemeral secretion of melatonin | Hôpital le Vinatier | Hospices Civils de Lyon | Completed | 18 Years | 65 Years | All | 10 | N/A | France |
| 4 | 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 | ||
| 5 | NCT02776215 (ClinicalTrials.gov) | September 2016 | 16/5/2016 | Study of the Pharmacokinetics and Safety of Tasimelteon in Children and Adolescents | Open-label Study to Investigate the Pharmacokinetics and Safety of Tasimelteon in Children and Adolescents | Circadian Rhythm Sleep Disorders;Non-24 Hour Sleep-Wake Disorder;Autism Spectrum Disorder;Smith-Magenis Syndrome | Drug: tasimelteon | Vanda Pharmaceuticals | NULL | Recruiting | 3 Years | 17 Years | All | 24 | Phase 1 | United States |
| 6 | NCT02231008 (ClinicalTrials.gov) | September 2015 | 29/8/2014 | Evaluating the Effects of Tasimelteon vs Placebo on Sleep Disturbances in SMS | A Double-blind, Randomized, Two-period Crossover Study Evaluating the Effects of Tasimelteon vs. Placebo on Sleep Disturbances of Individuals With Smith-Magenis Syndrome (SMS) | Smith-Magenis Syndrome;Circadian | Drug: tasimelteon;Drug: placebo | Vanda Pharmaceuticals | NULL | Completed | 3 Years | 65 Years | All | 49 | Phase 2/Phase 3 | United States |
| 7 | NCT00506259 (ClinicalTrials.gov) | July 17, 2007 | 21/7/2007 | Treatment Strategies for Children With Smith-Magenis Syndrome | A Phase One Treatment Trial of the Circadian Sleep Disturbance in Smith-Magenis Syndrome (SMS) | Developmental Delay Disorders;Chromosome Deletion;Mental Retardation;Sleep Disorders, Circadian Rhythm;Self Injurious Behavior | Drug: dTR Melatonin (NIH CC PDS);Device: Phototherapy (Bright Light);Drug: Melatonin CR | National Human Genome Research Institute (NHGRI) | NULL | Completed | 3 Years | 45 Years | All | 23 | Phase 1 | United States |
| 8 | NCT00691574 (ClinicalTrials.gov) | September 1998 | 30/5/2008 | Melatonin Levels in Smith Magenis Syndrome (SMS) | Melatonin Levels in Sleep-disordered Smith-Magenis Syndrome: a Pilot Study | Chromosome Disorders | Dietary Supplement: Melatonin;Device: Enviro-light artificial light box | Oregon Health and Science University | NULL | Terminated | 3 Years | 85 Years | All | 5 | N/A | United States |
| 9 | 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 States;Portugal;Greece;Spain;Ireland;Turkey;Israel;United Kingdom;France;Canada;Australia;Germany;Netherlands |