3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details
臨床試験数 : 217 / 薬物数 : 149 - (DrugBank : 33) / 標的遺伝子数 : 54 - 標的パスウェイ数 : 80
| 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 | 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 Inventory (BPI) (Questionnaire);Diagnostic Test: German Pain Inventory (Questionnaire);Diagnostic Test: Fatigue Severity and Disability Scale (FSS) (Questionnaire);Diagnostic Test: Rotterdam Handicap Scale (RHS) (Questionnaire);Diagnostic Test: R-PAct (Questionnaire);Diagnostic Test: Quick Motor Function Test;Diagnostic Test: Handheld Dynamometry (HHD);Diagnostic Test: Six-minute walk test (6MWT);Diagnostic Test: Pressure pain threshold;Diagnostic Test: Muscle ultrasound;Diagnostic Test: Vital signs;Diagnostic Test: Borg Scale;Diagnostic Test: Laboratory assessment: Creatine kinase;Diagnostic Test: Laboratory assessment: Vitamin D Level;Diagnostic Test: Laboratory assessment: calcium;Diagnostic Test: Laboratory assessment: magnesium;Diagnostic Test: Laboratory assessment: phosphate;Genetic: Genetic test: ACE polymorphism;Genetic: Genetic test: ACTN3 polymorphism;Genetic: Blood draw for optional genetic exome sequencing | LMU Klinikum | NULL | Not yet recruiting | 18 Years | N/A | All | 95 | Germany | |
| 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 Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3 | 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 Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3 | 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 phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3 | Long term safety study of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3 - SMA-002 | Spinal Muscular Atrophy (SMA) Type 3 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE Product Name: Amifampridina fosfato Product Code: [Amifampridina fosfato] INN or Proposed INN: AMIFAMPRIDINA | 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 Phosphate in Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3. | A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3 - SMA-001 | Spinal Muscular Atrophy (SMA) Type 3 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE Product Name: Amifampridina fosfato Product Code: [Amifampridina fosfato] INN or Proposed INN: AMIFAMPRIDINA Other descriptive name: 3,4-Diaminopyridine Phosphate | 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) (STEER) | A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are = 2 to < 18 years of age, treatment naive, sitting, and never ambulatory - STEER | Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Zolgensma Product Name: OAV101 Product Code: OAV101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101 Trade Name: Okrido 6mg/mL oral solution Product Name: Okrido INN or Proposed INN: prednisolone sodium phosphate Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE | 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;Vietnam;India;Egypt;Mexico;Brazil;Malaysia;Singapore;Denmark;South Africa;China |