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

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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	NCT05626855 (ClinicalTrials.gov)	April 24, 2023	7/11/2022	Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Api ...	An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegr ...	Spinal Muscular Atrophy;Spinal Muscular AtrophyType 3;Spinal Muscular Atrophy Type II;SMA;Neuromuscu ...	Drug: Apitegromab	Scholar Rock, Inc.	NULL	Not yet recruiting	2 Years	N/A	All	260	Phase 3	United States;Belgium;Italy;Netherlands;Poland;Spain
2	NCT05769465 (ClinicalTrials.gov)	April 1, 2023	3/3/2023	MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular A ...	MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular A ...	Spinal Muscular Atrophy	Drug: disease modifying treatments	Fondazione Policlinico Universitario Agostino Gemelli IRCCS	NULL	Not yet recruiting	N/A	N/A	All	247		NULL
3	NCT05386680 (ClinicalTrials.gov)	January 12, 2023	18/5/2022	Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 A ...	Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Effi ...	Spinal Muscular Atrophy	Genetic: OAV101	Novartis Pharmaceuticals	NULL	Recruiting	2 Years	12 Years	All	28	Phase 3	Canada;Japan;Netherlands;Spain
4	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) (S ...	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 ...
5	NCT05335876 (ClinicalTrials.gov)	December 19, 2022	12/4/2022	Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials	Long-term Follow-up of Patients With Spinal Muscular AtrophyTreated With OAV101 IT or OAV101 IV in C ...	Spinal Muscular Atrophy (SMA)	Biological: onasemnogene abeparvovec	Novartis Pharmaceuticals	NULL	Recruiting	N/A	N/A	All	260	Phase 3	Australia;Belgium
6	NCT05614531 (ClinicalTrials.gov)	November 1, 2022	2/11/2022	Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atro ...	A Multicenter, Nonrandomized, Open-label,Dose Escalation Clinical Trial to Assess the Safety and Eff ...	Spinal Muscular Atrophy Type I	Genetic: EXG001-307 injection	Hangzhou Jiayin Biotech Ltd	NULL	Recruiting	1 Day	180 Days	All	12	Phase 1/Phase 2	China
7	NCT05522361 (ClinicalTrials.gov)	September 15, 2022	24/5/2022	Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen	Risdiplam Exchange in Patients With Spinal Muscular Atrophy(SMA) Previously and Exclusively Treated ...	Spinal Muscular Atrophy	Drug: Risdiplam	Clinic for Special Children	Genentech, Inc.	Not yet recruiting	2 Years	35 Years	All	10	Phase 4	United States
8	NCT05337553 (ClinicalTrials.gov)	July 6, 2022	14/4/2022	A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscula ...	A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefg ...	Spinal Muscular Atrophy;Neuromuscular Diseases;SMA	Drug: taldefgrobep alfa;Drug: Placebo	Biohaven Pharmaceuticals, Inc.	NULL	Recruiting	4 Years	21 Years	All	225	Phase 3	United States;Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom
9	NCT05115110 (ClinicalTrials.gov)	June 2, 2022	1/11/2021	A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067 ...	A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigat ...	Spinal Muscular Atrophy (SMA)	Drug: RO7204239;Drug: Placebo;Drug: Risdiplam	Hoffmann-La Roche	NULL	Recruiting	2 Years	25 Years	All	259	Phase 2/Phase 3	United States;Belgium;Italy;Netherlands;Poland;United Kingdom;Germany
10	JPRN-jRCT2031220035	26/05/2022	23/04/2022	Long-Term Extension Study of Nusinersen in Participants With SMA	A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants Wit ...	Spinal muscular atrophy	Research Name: BIIB058 Generic Name: nusinersen Trade Names: Spinraza Participants in Study 203 who rec ...	Irzhevsky Victoria	NULL	Pending	Not applicable	Not applicable	Both	172	Phase 3	United States;Canada;Estonia;Hungary;Ireland;Italy;Latvia;Poland;Spain;Taiwan;France;Germany;Greece; ...

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