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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-000387-14-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
150Phase 2United States;France;Canada;Greece;Spain;Israel;Netherlands;Germany;United Kingdom
2NCT03492970
(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
3EUCTR2017-000387-14-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
4NCT03013543
(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.NULLRecruiting6 YearsN/AAll150Phase 2;Phase 3United States;Canada;Germany;Israel;Netherlands;Spain;United Kingdom
5NCT02776215
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02231008
(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
7NCT00506259
(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
8NCT00691574
(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
9EUCTR2017-000387-14-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]Rhythm Pharmaceuticals, Inc.NULLNA Female: yes
Male: yes
100Phase 2United States;Portugal;Greece;Spain;Ireland;Turkey;Israel;United Kingdom;France;Canada;Australia;Germany;Netherlands
10EUCTR2017-000387-14-NL
(EUCTR)
21/08/2019Setmelanotide (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 (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.NULLNA Female: yes
Male: yes
150Phase 2Portugal;France;United States;Greece;Canada;Spain;Israel;Germany;Netherlands;Italy;United Kingdom;Sweden