202. スミス・マギニス症候群
［臨床試験数：10，薬物数：10（DrugBank：4），標的遺伝子数：4，標的パスウェイ数：6］
Searched query = "Smith-Magenis syndrome"
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 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 150 | Phase 2 | United States;France;Canada;Greece;Spain;Israel;Netherlands;Germany;United Kingdom | ||
| 2 | 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 |
| 3 | 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 | ||
| 4 | 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 | Recruiting | 6 Years | N/A | All | 150 | Phase 2;Phase 3 | United States;Canada;Germany;Israel;Netherlands;Spain;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 |
| 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 | NCT02231008 (ClinicalTrials.gov) | September 2015 | 29/8/2014 | Evaluating the Effects of Tasimelteon vs Placebo on Sleep Disturbances in SMS | Smith-Magenis Syndrome;Circadian | Drug: tasimelteon | Vanda Pharmaceuticals | NULL | Recruiting | 16 Years | 65 Years | Both | 48 | 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-NL (EUCTR) | 21/08/2019 | 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 (two different mutations in gene) or homozygous deficiency obesity POMC/PCSK1/LEPR composite heterozygous deficiency obesity Smith-Magenis Syndrome (SMS) 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] | Rhythm Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 150 | Phase 2 | Portugal;France;United States;Greece;Canada;Spain;Israel;Germany;Netherlands;Italy;United Kingdom;Sweden | ||||
| 10 | 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] | Rhythm Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 100 | Phase 2 | United States;Portugal;Greece;Spain;Ireland;Turkey;Israel;United Kingdom;France;Canada;Australia;Germany;Netherlands |