DDrare: Database of Drug Development for Rare Diseases

3. 脊髄性筋萎縮症
［臨床試験数：179，薬物数：102（DrugBank：26），標的遺伝子数：52，標的パスウェイ数：78］

Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 2011-2012 seasonal flu vaccine Intramuscular; 2011-2012 seasonal flu vaccine Subcutaneous; 4-Aminopyridine; 4-aminopyridine; 4-cholesten-3-one, oxime; 4-cholesten-3-one,oxime; AVXS-101; AVXS-101 (previously known as scAAV9.CB.SMN); Adipose derived mesenchymal stem cell; Air; Amifampridine; Amifampridine Phosphate; Amifampridine Phosphate 10 MG Oral Tablet; Biocarn; Branaplam; Celecoxib; GH; HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY; Hydroxyurea; ISIS 396443; ISIS 396443 (BIIB058); ISIS 396443, BIIB058; Itraconazole; LEVOCARNITINE; LMI070; Leuprolide; Levocarnitine; Mestinon; Midazolam; N.a.; NUSINERSEN; NUSINERSEN SODIUM; Nerve Growth Factor; Norditropin SimpleXx; Norditropin SimpleXx 15 mg/1.5 ml; Nursinersen; Nusinersen; Nusinersen Injectable Product; Nusinersen Treatments; OLESOXIME; ONASEMNOGENE ABEPARVOVEC; Olesoxime; Onasemnogene Abeparvovec-xioi; Onasemnogene abeparvovec; Onasemnogene abeparvovec-xioi; Orfiril Saft; Other: Monthly assessments of functional motor abilities by a trained therapist; Other: Prospective observational registry; PARACETAMOL; Paracetamol; Paracetamol 120Mg/5mL Oral Suspension; Phenylbutyrate; Phosphate; Placebo; Procedure: Air stacking manuever; Procedure: Sham Procedure; Procedure: Sham procedure; Pyridostigmine; Pyridostigmine Bromide; Pyridostigmine bromide; RO6885247; RO6885247/F02; RO6885247/F03; RO7034067; RO7034067 / F12; RO7034067 / F13; RO7034067/F06 + solvente (RO; RO7034067/F06 with solvent (RO7034067/F08); RO7034067/F07 + solvente (RO; RO7034067/F07 with solvent (RO7034067/F09); RO7034067/F12; RO7034067/F13; RO7090919; RVG 03820; Reldesemtiv; Reldesemtiv 150 mg; Reldesemtiv 450 mg; Riluzole; Risdiplam; SESAME OIL, REFINED; SMART; SRK-015; Salbutamol; Sesame oil; Sodium phenylbutyrate; Sodium phenylbutyrate (NaPB); Somatotropin; Somatropin; Spinraza; Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide; TRO19622; Testosterone; VALPROATE SODIUM; VOLMAX*30CPR 4MG R.P.; VPA; Valproate; Valproate, Levocarnitine; Valproic Acid; Valproic Acid (VPA); Valproic Acid and Levocarnitine; Valproic acid; Zolgensma

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	NCT00481013 (ClinicalTrials.gov)	July 2007	30/5/2007	Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy	Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study	Spinal Muscular Atrophy	Drug: Valproic Acid (VPA);Drug: Placebo	University of Utah	Families of Spinal Muscular Atrophy;Abbott	Completed	18 Years	60 Years	Both	33	Phase 2	United States
2	NCT00374075 (ClinicalTrials.gov)	September 2003	6/9/2006	Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy	In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy	Spinal Muscular Atrophy	Drug: Valproic Acid	University of Utah	Families of Spinal Muscular Atrophy;Sigma Tau Pharmaceuticals, Inc.;Abbott	Completed	2 Years	N/A	Both	42	Phase 1	United States

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

NCT00481013
(ClinicalTrials.gov)

July 2007

30/5/2007

Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy

Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study

Spinal Muscular Atrophy

Drug: Valproic Acid (VPA);Drug: Placebo

University of Utah

Families of Spinal Muscular Atrophy;Abbott

Completed

18 Years

60 Years

Both

Phase 2

United States

NCT00374075
(ClinicalTrials.gov)

September 2003

6/9/2006

Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy

In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy

Spinal Muscular Atrophy

Drug: Valproic Acid

University of Utah

Families of Spinal Muscular Atrophy;Sigma Tau Pharmaceuticals, Inc.;Abbott

Completed

2 Years

N/A

Both

Phase 1

United States