DDrare: Database of Drug Development for Rare Diseases

2. 筋萎縮性側索硬化症
［臨床試験数：508，薬物数：530（DrugBank：146），標的遺伝子数：170，標的パスウェイ数：221］

Searched query = "Amyotrophic lateral sclerosis", "ALS"
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 = (2R)-2-Propyloctanoic acid; (6R)-4, 5, 6, 7 - tetrahydro-N6-propyl-2,6-benzothiazolidiamine dihydrochloride monohydrate; (R)-(-)-2-Propyloctaroic acid; 1.0 MIU IL-2 per day; 2.0 MIU IL-2 per day; 250 mg CK-2017357; 4 cholesten-3-one, oxime; 4-cholesten-3-one, oxime; 40 mg GA; 40 mg glatiramer acetate; 5% glucose water solution; 500 mg CK-2017357; 6-ethynyl-1-(pentan-3-yl)-1H-imidazo[4,5-b]pyrazin-2(3H)-one; AB1010; ALPHA-LIPOIC ACID; ALXN1210; ALZT-OP1a (cromolyn); AMSC; AMX0035; ANX005; AP; AP-101; APO-P001; ARIMOCLOMOL; AT-1501; AVP-923; AZILECT® 1 mg Tabletten; Acetate; Acetylcarnitine; Acthar; Actos 15 mg; Actos 30 mg; Actos 45 mg; Albumin; Albutein 5%; Albúmina humana; Amivita; Anakinra; Arimoclomol; Arundic Acid; Arundic acid; AstroRx; Atropine; Autologous Adipose-derived Mesenchymal Stromal Cells; Autologous MSC-NTF cells; Autologous Mesenchymal stem cells (MSCs); Autologous T-regulatory lymphocytes; Autologous adult bone marrow mononuclear cells (BM-MNC) unexpanded; Autologous adult bone marrow mononuclear cells unexpanded; Autologous bone marrow mononucleated cells; Autologous bone marrow-derived stem cells; Autologous mesenchymal stem cells; Azilect; BHV-0223; BIIB050 / KNS-760704; BIIB050/KNS-760704; BIIB067; BIIB067 (ISIS666853); BIIB078; BIIB100; BIIB105; BIOTIN; BLZ945; BOTOX 100 UNITES ALLERGAN (PR1); BOTULINUM TOXIN TYPE A; BRX-345; Baclofen; Basiliximab; Behavioral: BCI-FIT active querying; Behavioral: BCI-FIT adaptive signal modeling; Behavioral: BCI-FIT language modeling; Behavioral: BCI-FIT multi-modal access; Betamethasone; Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days; Biologic: IC14 (monoclonal antibody against human CD14); Biotin; Blood sample and environmental survey; Bosutinib; Botox; Botox injection; Botulinum Toxin Type B; Botulinum toxin type A; Botulinum toxin type B (Myobloc); Butylphthalide; CARBOLITHIUM; CARBOLITHIUM*100CPS 150MG; CBD; CC100; CK-2017357; CK-2017357 (Part A); CK-2017357 (Part B); CK-2127107; CK2017357; CNM-Au8; COLCHICINA; COLCHICINA LIRCA - 1 MG COMPRESSE 60 COMPRESSE; Caffeine; Calogen; Cannabis; Cannabis Sativa extract Oromucosal spray; Cannabis, Medical; Capsaicin; Capsaicin Challenge; Carbidopa; Carbidopa-levodopa; Carbonate; Carbonate de lithium; Ceftriaxone; Celecoxib; Cell-based therapeutics; Cell-based therapy; Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients; Cimetidine; Cimetidine plus Dexpramipexole; Ciprofloxacin; Cistanche Total Glycosides; Clenbuterol; Coenzyme Q10; Colchicina; Colchicine; Colchicine 1 MG Oral Tablet; Combination Product: Active treatment with dual therapy; Control group; Copegus; Copper; Cord blood therapy; Corticotropin; Creatine; Creatine Monohydrate; Creatine monohydrate; Creatinine; Cromolyn; Cu(II)ATSM; Curcumin; Células Mesenquimales de tejido adiposo; Células madre mesenquimales de tejido adiposo autólogo; Células troncales hematopoyéticas autólogas adultas extraidas de médula ósea; DEXMEDETOMIDINE; DEXTRAN SULFATE SODIUM; DNL747; DPA; DaTSCAN; DaTscan; Daraprim; Darunavir; Datscan; Deferiprone; Device: Electrical Impedance Myography; Device: Iowa Oral Performance Instrument; Device: Iowa Oral Pressure Instrument; Device: Micro Mouth Pressure Meter; Device: PowerLung trainer; Device: Pulmonary Function Testing; Device: Stereotactic surgical device; Device: Voluntary Peak Cough Flow Testing; Dexdor; Dexmedetomidine; Dexpramipexole; Dexpramipexole (dose 1); Dexpramipexole (dose 2); Dexpramipexole Dichydrochloride; Dexpramipexole Dihydrochloride; Dextran; Dextran Sodium Sulfur Salt 5, Mw 5 kDa. a type of Low Molecular Weight Dextran Sulfate (LMW-DS); Dextromethorphan; Dextromethorphan HBr and quinidine sulfate; Dextromethorphan hydrobromide 20 mg and quinidine sulfate 10 mg; Dextromethorphan hydrobromide 30 mg and quinidine sulfate 10 mg; Dolutegravir; Dronabinol; Drug: F 18 T807; Dutasteride; Dutasteride 0.5 mg; E0302 (mecobalamin); EBIXA; EDARAVONE; EH301 (Nicotinamide Riboside/Pterostilbene); EPERISONE; EPI-589; EPREX IV SC 1FL 1ML 40000 UI; EPREX*1SIR 40000UI/ML 1ML; ERYTHROPOIETIN; ESKETAMINE; Edaravone; Endocannabinoid palmitoylethanolamide (PEA); Engensis; Eperisone; Eril®; Erythropoetin (EPO); Erythropoietin; Escitalopram; Esketamine; Ezogabine; FASUDIL HYDROCHLORIDE; FLX-787-ODT; FLX-787-ODT (orally disintigrating tablet); Fasudil; Febuxostat; Fecal microbiota transplantation; Fig; Filgrastim; Fixed dose combination Ciprofloxacin/Celecoxib; Fycompa; G-CSF; GDC-0134; GM604; GSK1223249; GUANA2015; GUANABENZ; GUANABENZ ACETATO; Gilenya; Glatiramer; Glucose; Gold; Gold Nanocrystals; Granulocyte - colony stimulating factor (G-CSF); Granulocyte Colony Stimulating Factor; Growth Hormone (Somatropin); Growth hormone; Guanabenz; HB-adMSCs; HK-001; HUMAN ALBUMIN SOLUTION; HYNR-CS inj; HYNRCS-Allo inj; High-caloric fatty diet; Human Neural Stem Cells; Human albumin; Human autologous mesenchymal stem cells; Hyperpolarized [1-13C]pyruvate; I-123; IBUDILAST; IC14; IL-2; ILB; ILB: 100mg/ml solution for injection and infusion; INTERLEUKIN-2; IPL344; ISIS 333611; Ibudilast; Immunoglobulin G1, anti (protein Nogo A) (human Mus musculus monoclonal heavy; Immunoglobulin G1, anti (protein Nogo A) (human Mus musculus monoclonal heavy chain), disulfide with human Mus musculus monoclonal light chain, dimer; Incobotulinum Toxin A; Inosine; Insulin like growth factor, type 1; Interleukin-2; Intra venous injection of stem cell; Intrathecal autologous stem cell; Intrathecal injection; Intravenous administration of 1 million of MSC; Intravenous administration of 2 million of MSC; Intravenous administration of 4 million of MSC; Intraventricular injection; Ioflupane 123I; Isocaloric Diet; Jevity 1.0; Jevity 1.5; KNS-760704; Ketanest; Ketanest-S; KetoCal; L-Serine; LEVOSIMENDAN; LIORESAL*50CPR 10MG; LITHIUM CARBONATE; LITHIUM CITRATE; LITIO CARBONATO; Lacosamide; Lactose; Lenograstim; Leuprolide; Levetiracetam; Levodopa; Levosimendan; Lipoic acid; Lipoic acid group; Liposomed polyphenols resveratrol and curcumin; Lithium; Lithium Apogepha; Lithium Carbonate; Lithium Citrate; Lithium carbonate; Lithium citrate; Lithiumcarbonaat; Lithiumcarbonaat 400 PCH tabletten; Lunasin; Lunasin Regimen; MANNITOLO 18%; MANNITOLO 18% LDB; MASITINIB; MASITINIB MESYLATE; MCI-186; MCI-186 in open label phase; MD1003; MEMANTINE HYDROCHLORIDE; MN-166; MNC (Mononuclear cells); MONITOR; MSC-NTF cells transplantation (IM); MSC-NTF cells transplantation (IT); MSC_NTF cells transplantation by multiple intramuscular injections at 24 separate sites, in addition to a single intrathechal injection into the CSF; MT-1186; MYELOSTIM 34*1FL 263MCG+1SIR; MYONAL*30CPR RIV 100MG; Masitinib; Masitinib (3.0); Masitinib (4.5); Masitinib (6.0); Masitinib 100 mg Tablets; Masitinib 100mg; Masitinib 200 mg Tablets; Masitinib 200mg; Masitinib Mesylate; Masitinib mesylate; Mecobalamin; MediCabilis CBD Oil; Medicine; Memantine; Memantine (Ebixa); Memantine Hydrochloride Oral Solution; Mesenchymal Stromal Cells; Mesenchymal stem cell; Mesenchymal stem cells; Mesenchymal stem cells isolated from Wharton's jelly; Metformin; Methylcobalamin; Methylprednisolone; Mexiletine; Midazolam; Minocycline; Modal; Monthly autologous Treg cells infusions + 3 times per week Interleukin-2 injections; Moxifloxacin; Multiple Dose; Mycophenolate; Mycophenolate mofetil; Myonal; NP001; NS; Nicotinamide; Nicotinamide riboside; Nuedexta; NurOwn® (MSC-NTF cells); ODM-109; ODM-109 capsule 1 mg; OLANZAPINE; ONO-2506; ONO-2506PO; Olanzapine; Olesoxime; Oral LEVOSIMENDAN; Oral nutritional supplementation; Other:; Other: Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised; Other: Communicative Effectiveness Survey; Other: Control group; Other: Eating Assessment Tool 10; Other: Eating Assessment Tool-10; Other: Functional Oral Intake Scale; Other: Historical control; Other: Other: Symptomatic treatment of ALS; Other: Saline; Other: Swallowing Quality of Life Questionnaire; Other: Swallowing Related Quality of Life Questionnaire; Other: The Center for Neurologic Studies Bulbar Function Scale; Other: The Center for Neurologic Study Bulbar Function Scale; Oxepa; Ozanezumab; PARACETAMOLO; PBMC autotransplantation; PERFALGAN; PLECONARIL; PLENUR; PU-AD; PYRIMETHAMINE; Palmitoylethanolamide; Pegcetacoplan (APL-2); Perampanel; Phenylbutyrate; Phosphate; Pimozide; Pimozide 2 mg per day; Pimozide 2mg/day (current) or 4 mg/day (study initiation); Pimozide 4 mg per day; Pioglitazone; Pioglitazone and Tretinoin; Plasma from healthy young people treatment + Riluzole; Pleconaril; PolyMVA; Pramipexole; Prednisone; Pridopidine; Probiotic; Procedure: Injection of salivary glands; Procedure: Pulmonary Function Testing; Procedure: Umbilical cord blood therapy; Procedure: Videofluoroscopic Swallowing Study; Procedure: Videofluoroscopic Swallowing Study (VFSS); Procedure: Videofluoroscopic swallowing study; Proleukin; Proleukin 18 MUI; Prototype capsule GDC-0134; Pyrimethamine; Q-Cells; Quinidine; R(+) pramipexole dihydrochloride monohydrate; RAPA-501 Autologous T cells; RAPAMUNE - 1 MG 100 COMPRESSE RIVESTITE IN BLISTER USO ORALE; RASAGILINE MESILATE; RAVULIZUMAB; RETIGABINE; RIBAVIRIN; RILUTEK; RILUTEK 50 mg, film-coated tablets; RILUZOLE; RILUZOLO; RNS60; Rabeprazole; Ranolazine; Ranolazine 1000 MG; Ranolazine 500 MG; Rapamune; Rapamycin; Rasagiline; Rasagiline Mesilate; Ravulizumab; ReQuip CR; Recombinant human erythropoietin(rhEPO); Reference capsule GDC-0134; Reldesemtiv; Repository corticotropin injection; Requip CR tablets; Resveratrol; Retigabine; Ribavirin; Rilutek; Rilutek 50 mg film-coated tablets; Riluzole; Riluzole (100 mg); Riluzole 50 MG; Riluzole 50 MG (Part B); Riluzole Oral Soluble Film; Riluzole Oral Soluble film (ROSF) 50 mg; Ritonavir; SB-509; SNN0029; SNN0029 infusion solution; SODIUM PYRUVATE; SPECT : 123 I-FP-CIT (DATSCAN) and 123I-ADAM; SUB126308; Saline solution; Sativex; Scopolamine; Scopolamine, atropine, edaravone and dexmedetomidine; Selective Serotonin Uptake Inhibitors (SSRI); Serine; Serotonin; Single dose reduced; Single dose standard; Sodium Valproate; Sodium phenylbutyrate; Somatropin; Spirit1; Stem Cell; Stem Cells; Stem cell; Stem cell (HPC) implantation; Sulfate; Suspension of human autologous MSC 3P in 1,5 ml; Suspension of human autologous MSC 3P in 1.5 ml; TAUROURSODEOXYCHOLIC ACID; TCH346; TEVAGRASTIM; TRO19622; TUDCA; TUDCABIL; TV-7110; Tacrolimus; Talampanel; Tallampanel; Tamoxifen; Tamoxifen 40 mg daily for one year; Taurolite; Tauroursodeoxycholic Acid; Tauroursodeoxycholic acid; Tauroursodeoxycholic acid (TUDCA); Tenofovir; Tenofovir alafenamide; Tenofovir alafenamide (TAF); Testosterone; Thalidomide; Thalidomide (drug); Theracurmin HP; Tioctic acid; Tirasemtiv; Tocilizumab; Tocotrienols; Trametinib; Trametinib (0.5 mg); Trametinib (1 mg); Trametinib (2 mg); Trazodone; Trazodone Hydrochloride oral solution; Tretinoin; Tretinoin and Pioglitazone HCL; Tricyclic Antidepressants (TCA); Triheptanoin; Triumeq; Trobalt; Téralithe LP; Ultomiris; Ultra-high-caloric carbohydrate-rich diet; Ultra-high-caloric fatty diet; VM202; Valproate; Verdiperstat; Vitamin E; Water; YAM80; ZIBREN*20BUST 500MG; Zibren; Zilucoplan; Zinc; Zinc and Copper; Zyprexa; [11C]MPC6827; [18F] GE-180; [18F]DPA-714 PET; [18F]PBR06; [I-123] ADAM

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	EUCTR2011-004798-99-DE (EUCTR)	27/02/2013	27/12/2012	Safety and Efficacy of SOD1 Inhibition By Pyrimethamine in Familial (ALS)	Safety and Efficacy of SOD1 Inhibition By Pyrimethamine in Familial (ALS) - Pyrimethamine In Familial ALS	Familial amyotrophic lateral sclerosis (FALS) MedDRA version: 14.1;Level: LLT;Classification code 10036704;Term: Primary lateral sclerosis;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Trade Name: Daraprim INN or Proposed INN: PYRIMETHAMINE	Weill Medical College of Cornell University	NULL	Not Recruiting			Female: yes Male: yes	40		United States;Germany;Italy
2	NCT01083667 (ClinicalTrials.gov)	November 2009	17/12/2009	SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS)	Phase I/II Study of SOD1 Inhibition by Pyrimethamine in Familial ALS	Familial Amyotrophic Lateral Sclerosis	Drug: Pyrimethamine	Weill Medical College of Cornell University	Muscular Dystrophy Association	Completed	18 Years	N/A	All	32	Phase 1;Phase 2	United States;Germany;Italy;Sweden

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

EUCTR2011-004798-99-DE
(EUCTR)

27/02/2013

27/12/2012

Safety and Efficacy of SOD1 Inhibition By Pyrimethamine in Familial (ALS)

Safety and Efficacy of SOD1 Inhibition By Pyrimethamine in Familial (ALS) - Pyrimethamine In Familial ALS

Familial amyotrophic lateral sclerosis (FALS)
MedDRA version: 14.1;Level: LLT;Classification code 10036704;Term: Primary lateral sclerosis;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Trade Name: Daraprim
INN or Proposed INN: PYRIMETHAMINE

Weill Medical College of Cornell University

NULL

Not Recruiting

Female: yes
Male: yes

United States;Germany;Italy

NCT01083667
(ClinicalTrials.gov)

November 2009

17/12/2009

SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS)

Phase I/II Study of SOD1 Inhibition by Pyrimethamine in Familial ALS

Familial Amyotrophic Lateral Sclerosis

Drug: Pyrimethamine

Weill Medical College of Cornell University

Muscular Dystrophy Association

Completed

18 Years

N/A

All

Phase 1;Phase 2

United States;Germany;Italy;Sweden