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.TrialIDDate_
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PhaseCountries
1NCT04768803
(ClinicalTrials.gov)
March 15, 202116/2/2021Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or ObesityCirculating Levels of Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and / or Overweight, and / or ObesityAngelman Syndrome;Smith-Magenis Syndrome;X Fragile Syndrome;Epilepsy;Prader-Willi SyndromeBiological: acylated and unacylated ghrelin dosagesUniversity Hospital, ToulouseNULLNot yet recruiting3 Years50 YearsAll300NULL
2EUCTR2017-000387-14-GR
(EUCTR)
10/01/202001/11/2019Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesitySetmelanotide (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.NULLNot RecruitingFemale: yes
Male: yes
222Phase 2France;United States;Canada;Greece;Spain;Israel;Netherlands;Germany;United Kingdom
3NCT03492970
(ClinicalTrials.gov)
March 7, 20181/3/2018Melatonin in Adults With SMSCharacterization of Behavioral Disorders and 24 H-melatonin Level in Adults With Smith Magenis SyndromeSmith Magenis SyndromeBiological: Hourly dosing of the nychtemeral secretion of melatoninHôpital le VinatierHospices Civils de LyonCompleted18 Years65 YearsAll10N/AFrance
4EUCTR2017-000387-14-GB
(EUCTR)
24/05/201717/03/2017Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesitySetmelanotide (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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
150Phase 2United States;France;Greece;Canada;Spain;Israel;Netherlands;Germany;United Kingdom
5NCT03013543
(ClinicalTrials.gov)
January 20173/1/2017Setmelanotide Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of ObesitySetmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of ObesityPro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic);Leptin Receptor Deficiency Obesity;Smith-Magenis Syndrome;Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder)Drug: SetmelanotideRhythm Pharmaceuticals, Inc.NULLActive, not recruiting6 YearsN/AAll150Phase 2/Phase 3United States;Canada;France;Germany;Greece;Israel;Netherlands;Spain;United Kingdom
6NCT02776215
(ClinicalTrials.gov)
September 201616/5/2016Study of the Pharmacokinetics and Safety of Tasimelteon in Children and AdolescentsOpen-label Study to Investigate the Pharmacokinetics and Safety of Tasimelteon in Children and AdolescentsCircadian Rhythm Sleep Disorders;Non-24 Hour Sleep-Wake Disorder;Autism Spectrum Disorder;Smith-Magenis SyndromeDrug: tasimelteonVanda PharmaceuticalsNULLRecruiting3 Years17 YearsAll24Phase 1United States
7NCT02231008
(ClinicalTrials.gov)
September 201529/8/2014Evaluating the Effects of Tasimelteon vs Placebo on Sleep Disturbances in SMSSmith-Magenis Syndrome;CircadianDrug: tasimelteonVanda PharmaceuticalsNULLRecruiting16 Years65 YearsBoth48Phase 2/Phase 3United States
8NCT00506259
(ClinicalTrials.gov)
July 17, 200721/7/2007Treatment Strategies for Children With Smith-Magenis SyndromeA 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 BehaviorDrug: dTR Melatonin (NIH CC PDS);Device: Phototherapy (Bright Light);Drug: Melatonin CRNational Human Genome Research Institute (NHGRI)NULLCompleted3 Years45 YearsAll23Phase 1United States
9NCT00691574
(ClinicalTrials.gov)
September 199830/5/2008Melatonin Levels in Smith Magenis Syndrome (SMS)Melatonin Levels in Sleep-disordered Smith-Magenis Syndrome: a Pilot StudyChromosome DisordersDietary Supplement: Melatonin;Device: Enviro-light artificial light boxOregon Health and Science UniversityNULLTerminated3 Years85 YearsAll5N/AUnited States
10EUCTR2017-000387-14-FR
(EUCTR)
14/03/2019Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesitySetmelanotide (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.NULLNA Female: yes
Male: yes
100 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom;France;Canada;Australia;Germany;Netherlands;Israel;Turkey;Ireland;Spain;Greece;Portugal;United States