256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details

臨床試験数 : 180 / 薬物数 : 153 - (DrugBank : 30) / 標的遺伝子数 : 31 - 標的パスウェイ数 : 134

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No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04094948 (ClinicalTrials.gov)	January 1, 2023	17/9/2019	Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease	Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe DiseaseStably Treated With Enzym ...	Pompe Disease (Late-onset)	Drug: Clenbuterol;Drug: Placebos	Duke University	NULL	Not yet recruiting	18 Years	N/A	All	40	Phase 2	United States
2	NCT04808505 (ClinicalTrials.gov)	August 2022	10/3/2021	Rossella: A Study to Evaluate the Safety, PK, Efficacy, PD and Immunogenicity of Cipaglucosidase Alf ...	An Open-label Study to Evaluate the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immuno ...	Glycogen Storage Disease Type II Infantile Onset	Biological: Cipaglucosidase alfa (ATB200);Drug: Miglustat (AT2221)	Amicus Therapeutics	NULL	Recruiting	N/A	17 Years	All	22	Phase 3	United States
3	NCT05272969 (ClinicalTrials.gov)	March 31, 2022	28/1/2022	Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease	Pompe & Pain - Observational Study to Assess Musculoskeletal Pain in Late-onset Pompe Disease (LOPD)	Pompe Disease (Late-onset);Inclusion Body Myositis;Spinal Muscular Atrophy Type 3;FSHD	Diagnostic Test: Beck depression inventory fast screen (Questionnaire);Diagnostic Test: Brief Pain I ...	LMU Klinikum	NULL	Not yet recruiting	18 Years	N/A	All	95		Germany
4	NCT05164055 (ClinicalTrials.gov)	February 15, 2022	16/11/2021	A French Multicenter Phase 4 Open Label Extension Study of Long Term Safety and Efficacy in Patients ...	A French Multicenter Phase 4 Open Label Extension Study of Long Term Safety and Efficacy in Patients ...	Glycogen Storage Disease Type II	Drug: Avalglucosidase alfa (GZ402666)	Genzyme, a Sanofi Company	NULL	Not yet recruiting	6 Months	N/A	All	18	Phase 4	NULL
5	NCT04476550 (ClinicalTrials.gov)	February 1, 2022	26/2/2020	Clinical Specimen Collection From Pompe Disease Patients	Developing a Potential Cure for Pompe Disease: Clinical Specimen Collection From Individuals With Po ...	Pompe Disease	Drug: Filgrastim	Serhat Gumrukcu, MD PhD	Frida Therapeutics LLC	Recruiting	3 Years	30 Years	All	12		United States
6	EUCTR2019-001283-30-DK (EUCTR)	03/11/2021	05/10/2021	A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults ...	Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intra ...	Pompe Disease(also known as glycogen storage disease type II) MedDRA version: 20.1;Level: PT;Classif ...	Product Code: SPK-3006 INN or Proposed INN: SPK-3006	Spark Therapeutics	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Phase 1;Phase 2	France;United States;Spain;Denmark;Netherlands;Germany;United Kingdom;Italy
7	EUCTR2020-004686-39-NL (EUCTR)	04/10/2021	04/05/2021	Clinical Study for IOPD Participants Less Than or Equal to 6 Months of age to Evaluate Efficacy and ...	An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharm ...	Glycogen storage disease type II MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: G ...	Product Name: Avalglucosidase Alfa Product Code: GZ402666 INN or Proposed INN: ALGLUCOSIDASE ALFA Other ...	Sanofi-Aventis Recherche & Développement	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Phase 3	France;United States;Taiwan;Belgium;Germany;Netherlands;United Kingdom;Italy;China
8	NCT04910776 (ClinicalTrials.gov)	September 1, 2021	25/5/2021	Clinical Study for IOPD Participants Less Than or Equal to 6 Months of Age to Evaluate Efficacy and ...	An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharm ...	Glycogen Storage Disease Type II	Drug: avalglucosidase alfa	Sanofi	NULL	Recruiting	N/A	6 Months	All	16	Phase 3	Belgium;Germany;Italy;Netherlands;Taiwan;United Kingdom
9	NCT05017402 (ClinicalTrials.gov)	September 1, 2021	5/8/2021	Higher Dose of Alglucosidase Alpha for Pompe Disease	Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study	Glycogen Storage Disease Type II	Drug: Alglucosidase Alfa	Taipei Veterans General Hospital, Taiwan	NULL	Not yet recruiting	N/A	60 Years	All	36		NULL
10	NCT04848779 (ClinicalTrials.gov)	June 10, 2021	29/3/2021	A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patie ...	A Prospective Observational Study to Describe Clinical Outcomes of Alglucosidase Alfa Treatment in P ...	Glycogen Storage Disease Type II	Drug: Alglucosidase alfa GZ419829 (Myozyme)	Sanofi	NULL	Recruiting	N/A	6 Months	All	16		Belgium;Germany;Italy;Taiwan

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