↑ 疾患リストへ ← 戻る

 30. 遠位型ミオパチー [臨床試験数:8,薬物数:13(DrugBank:2),標的遺伝子数:1,標的パスウェイ数:1] 

Searched query = "Distal myopathy", "Distal muscular dystrophy", "Miyoshi myopathy", "Oculopharyngodistal myopathy"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2016-000360-42-BG26/04/20175 February 2018A study to see if aceneuramic acid is safe and effective in treating people with Hereditary Inclusion Body Myopathy (HIBM), a rare muscle disease.A Phase 3b Open-label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka's disease, or quadriceps sparing myopathy (QSM)
MedDRA version: 19.1 Level: LLT Classification code 10075048 Term: Hereditary inclusion body myopathy System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 19.1 Level: PT Classification code 10077945 Term: GNE myopathy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: INN: aceneuramic acid; Common Name: Sialic Acid
Product Code: UX001
Pharmaceutical Form: Tablet
INN or Proposed INN: ACENEURAMIC ACID
CAS Number: 131-48-6
Current Sponsor code: UX001
Other descriptive name: sialic acid, N-acetylneuraminic acid
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-
Ultragenyx Pharmaceutical Inc.Not RecruitingFemale: yes
Male: yes
165Phase 3France;United States;Canada;Israel;Bulgaria;Italy;United Kingdom
2EUCTR2015-004553-41-BG05/01/20175 February 2018A study to see if Aceneuramic Acid is safe in treating people with GNE Myopathy (GNEM) patients (a rare muscle disease also known as Hereditary Inclusion Body Myopathy (HIBM)) with severe ambulatory impairment.A Phase 2 Open-label study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (also known as Hereditary Inclusion Body Myopathy (HIBM)) patients with Severe Ambulatory ImpairmentGNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka’s disease, or quadriceps sparing myopathy (QSM)
MedDRA version: 20.0 Level: LLT Classification code 10075048 Term: Hereditary inclusion body myopathy System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0 Level: PT Classification code 10077945 Term: GNE myopathy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Sialic acid (INN: aceneuramic acid)
Product Code: UX001
Pharmaceutical Form: Tablet
INN or Proposed INN: ACENEURAMIC ACID
CAS Number: 131-48-6
Current Sponsor code: UX001
Other descriptive name: sialic acid; N-acetylneuraminic acid
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-
Ultragenyx Pharmaceutical Inc.Not RecruitingFemale: yes
Male: yes
45Phase 2United States;Canada;Bulgaria
3EUCTR2014-005432-33-BG07/06/20162 October 2017A study to see if sialic acid is safe and effective in treating people with Hereditary Inclusion Body Myopathy (HIBM), a rare muscle disease.A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka’s disease, or quadriceps sparing myopathy (QSM)
MedDRA version: 20.0 Level: LLT Classification code 10075048 Term: Hereditary inclusion body myopathy System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Sialic acid (INN: aceneuramic acid)
Product Code: UX001
Pharmaceutical Form: Tablet
INN or Proposed INN: ACENEURAMIC ACID
CAS Number: 131-48-6
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-
Pharmaceutical form of the placebo: Prolonged-release tablet
Route of administration of the placebo: Oral use
Ultragenyx Pharmaceutical Inc.Not RecruitingFemale: yes
Male: yes
89Phase 3France;United States;Canada;Israel;Bulgaria;Italy;United Kingdom
4NCT02736188May 2, 201626 November 2018Study to Evaluate the Safety and Efficacy of Ace-ER Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body MyopathyPhase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)Hereditary Inclusion Body Myopathy;Distal Myopathy With Rimmed Vacuoles;Distal Myopathy, Nonaka Type;GNE Myopathy;Quadriceps Sparing MyopathyDrug: Aceneuramic Acid Extended-Release TabletsUltragenyx Pharmaceutical IncNot recruiting18 YearsN/AAll143Phase 3United States;Bulgaria;Canada;France;Israel;Italy;United Kingdom
5NCT02731690April 29, 201626 November 2018A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory ImpairmentA Phase 2 Open-label Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory ImpairmentHereditary Inclusion Body Myopathy;Distal Myopathy With Rimmed Vacuoles;Distal Myopathy, Nonaka Type;GNE Myopathy;Quadriceps Sparing Myopathy;Inclusion Body Myopathy 2Drug: Aceneuramic Acid Extended-ReleaseUltragenyx Pharmaceutical IncNot recruiting18 YearsN/AAll42Phase 2United States;Bulgaria;Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02377921May 27, 201516 December 2017Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)Hereditary Inclusion Body Myopathy;Distal Myopathy With Rimmed Vacuoles;Distal Myopathy, Nonaka TypeDrug: Sialic Acid Tablets;Drug: Placebo TabletsUltragenyx Pharmaceutical IncNot recruiting18 Years55 YearsAll89Phase 3United States;Bulgaria;Canada;France;Israel;Italy;United Kingdom
7NCT01236898November 201019 February 2015Pharmacokinetic Study on N-acetylneuraminic AcidPharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM)Nonaka Myopathy;Hereditary Inclusion Body MyopathyDrug: NPC-09Tohoku UniversityNot recruiting20 Years40 YearsBoth6Phase 1Japan
8NCT00527228September 200314 September 2015Deflazacort in DysferlinopathiesDeflazacort in Dysferlinopathies (LGMD2B/MM) - a Double Blind, Placebo-controlled Clinical StudyLGMD2B;Miyoshi Myopathy;DysferlinopathyDrug: deflazacort;Drug: placeboLudwig-Maximilians - University of MunichNot recruiting18 YearsN/ABoth25Phase 2/Phase 3Germany

先頭へ