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

  
3 trials found
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.NULLNot RecruitingFemale: yes
Male: yes
222Phase 2France;United States;Canada;Greece;Spain;Israel;Netherlands;Germany;United Kingdom
2EUCTR2017-000387-14-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
3EUCTR2017-000387-14-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 States;Portugal;Greece;Spain;Ireland;Turkey;Israel;United Kingdom;France;Canada;Australia;Germany;Netherlands