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	NCT00485511 (ClinicalTrials.gov)	June 2007	11/6/2007	A Trial of Hydroxyurea in Spinal Muscular Atrophy	A Randomized, Double-Blind, Placebo-Controlled Trial of Hydroxyurea in Spinal Muscular Atrophy	Spinal Muscular Atrophy	Drug: Hydroxyurea	Kaohsiung Medical University Chung-Ho Memorial Hospital	NULL	Completed	4 Years	N/A	Both		Phase 2;Phase 3	Taiwan
2	NCT00568802 (ClinicalTrials.gov)	January 2004	4/12/2007	A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients	A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients	Muscular Atrophy, Spinal	Drug: Hydroxyurea;Drug: Placebo to match hydroxyurea	Stanford University	NULL	Completed	1 Year	10 Years	All	27	Phase 1;Phase 2	United States
3	NCT00568698 (ClinicalTrials.gov)	January 2004	4/12/2007	A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients	A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients	Muscular Atrophy, Spinal	Drug: Hydroxyurea;Drug: Placebo to match hydroxyurea	Stanford University	NULL	Completed	N/A	2 Years	All	29	Phase 1;Phase 2	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

NCT00485511
(ClinicalTrials.gov)

June 2007

11/6/2007

A Trial of Hydroxyurea in Spinal Muscular Atrophy

A Randomized, Double-Blind, Placebo-Controlled Trial of Hydroxyurea in Spinal Muscular Atrophy

Spinal Muscular Atrophy

Drug: Hydroxyurea

Kaohsiung Medical University Chung-Ho Memorial Hospital

NULL

Completed

4 Years

N/A

Both

Phase 2;Phase 3

Taiwan

NCT00568802
(ClinicalTrials.gov)

January 2004

4/12/2007

A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients

Muscular Atrophy, Spinal

Drug: Hydroxyurea;Drug: Placebo to match hydroxyurea

Stanford University

NULL

Completed

1 Year

10 Years

All

Phase 1;Phase 2

United States

NCT00568698
(ClinicalTrials.gov)

January 2004

4/12/2007

A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients

Muscular Atrophy, Spinal

Drug: Hydroxyurea;Drug: Placebo to match hydroxyurea

Stanford University

NULL

Completed

N/A

2 Years

All

Phase 1;Phase 2

United States