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"
5 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
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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
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.NULLRecruiting6 YearsN/AAll150Phase 2;Phase 3United States;Canada;Germany;Israel;Netherlands;Spain;United Kingdom
4EUCTR2017-000387-14-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
5EUCTR2017-000387-14-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