Setmelanotide ( DrugBank: Setmelanotide )


2 diseases
告示番号疾患名(ページ内リンク)臨床試験数
202スミス・マギニス症候群4
265脂肪萎縮症1

202. スミス・マギニス症候群


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_
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
3NCT03013543
(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
4EUCTR2017-000387-14-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

265. 脂肪萎縮症


臨床試験数 : 116 薬物数 : 170 - (DrugBank : 61) / 標的遺伝子数 : 26 - 標的パスウェイ数 : 97
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03262610
(ClinicalTrials.gov)
February 17, 201722/8/2017Setemelanotide in a Single Patient With Partial LipodystrophyExpanded-access for the Use of Setemelanotide in a Single Patient With Partial Lipodystrophy (LD) Associated With Leptin Deficiency and Multiple Autoimmune DiseasesHypertriglyceridemiaDrug: SetmelanotideRhythm Pharmaceuticals, Inc.NULLNo longer available15 Years15 YearsFemaleUnited States