3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details

臨床試験数 : 237 / 薬物数 : 123 - (DrugBank : 29) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 75

6 trials found

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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	EUCTR2021-003474-31-GR (EUCTR)	03/01/2023	15/09/2022	Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) ( ...	A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal ...	Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ...	Trade Name: Zolgensma INN or Proposed INN: Onasemnogene abeparvovec Other descriptive name: previously ...	Novartis Pharma AG	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	125	Phase 3	United States;United Arab Emirates;Saudi Arabia;Taiwan;Greece;Thailand;Russian Federation;Colombia;I ...
2	NCT03819660 (ClinicalTrials.gov)	March 7, 2019	23/1/2019	Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3	Long Term Safety Study of Amifampridine Phosphatein Ambulatory Patients With Spinal Muscular Atrophy ...	Muscle Atrophy	Drug: Amifampridine Phosphate 10 MG Oral Tablet	Catalyst Pharmaceuticals, Inc.	NULL	Enrolling by invitation	6 Years	50 Years	All	12	Phase 2	Italy
3	NCT03781479 (ClinicalTrials.gov)	January 21, 2019	18/12/2018	Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients	A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridi ...	Muscular Atrophy, Spinal	Drug: Amifampridine Phosphate;Drug: Placebo Oral Tablet	Catalyst Pharmaceuticals, Inc.	NULL	Completed	6 Years	50 Years	All	13	Phase 2	Italy
4	EUCTR2018-000160-28-IT (EUCTR)	06/11/2018	24/05/2021	Clinical study evaluating the long term safety of amifampridine phosphatein ambulatory patients with ...	Long term safety study of amifampridine phosphatein ambulatory patients with Spinal Muscular Atrophy ...	Spinal Muscular Atrophy (SMA) Type 3 MedDRA version: 20.1;Level: PT;Classification code 10041582;Ter ...	Trade Name: FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE Product ...	CATALYST PHARMACEUTICALS INC.	NULL	Not Recruiting			Female: yes Male: yes	12	Phase 2	Italy
5	EUCTR2017-004600-22-IT (EUCTR)	04/04/2018	07/04/2021	Clinical study evaluating the effect of Amifampridine Phosphatein Ambulatory Patients with Spinal Mu ...	A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridi ...	Spinal Muscular Atrophy (SMA) Type 3 MedDRA version: 20.1;Level: PT;Classification code 10041582;Ter ...	Trade Name: FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE Product ...	CATALYST PHARMACEUTICALS INC.	NULL	Not Recruiting			Female: yes Male: yes	12	Phase 2	Italy
6	EUCTR2021-003474-31-DK (EUCTR)		02/11/2021	Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) ( ...	A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal ...	Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ...	Trade Name: Zolgensma Product Name: OAV101 Product Code: OAV101 INN or Proposed INN: ONASEMNOGENE ABEPA ...	Novartis Pharma AG	NULL	NA			Female: yes Male: yes	125	Phase 3	United States;United Arab Emirates;Saudi Arabia;Taiwan;Thailand;Russian Federation;Colombia;Italy;Vi ...

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