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	JPRN-UMIN000008527	2009/01/01	25/07/2012	Study of efficacy and safety of NDDPX08 in ALS patients		Amyotrophic Lateral Sclerosis	For patients who begin to receive Rilutek treatment at the start or 4 weeks before the start of the observation period, the 12-week treatment with Rilutek alone (100 mg/day) during the observation period is followed by combined treatment (Rilutek + NDDPX08). The NDDPX08 dose level begins at 1.25 mg/day and is increased in steps to 15 mg/day during the 12-week treatment period according to the dose escalation schedule given on the next page (Fig. 1). If any serious adverse reaction arises following a dose increase to 10 mg/day and it is judged to be difficult to maintain this dose level, the dose level of 7.5 mg/day is regarded as the maintenance dose level. If alleviation of symptoms is noted during dose escalation steps, the dose level producing alleviation of symptoms is used as the maintenance dose level. The total NDDPX08 treatment period is 58-90 weeks (including the 4 weeks during which the dose level is reduced in steps). Follow-up of adverse events is continued until 1 month after the end of NDDPX08 treatment. Placebo (lactose) is administered to 10 of the 50 subjects planned to be enrolled in the study.	FeGALS	NULL	Complete: follow-up complete	20years-old	75years-old	Male and Female	50	Not selected	Japan

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

JPRN-UMIN000008527

2009/01/01

25/07/2012

Study of efficacy and safety of NDDPX08 in ALS patients

Amyotrophic Lateral Sclerosis

For patients who begin to receive Rilutek treatment at the start or 4 weeks before the start of the observation period, the 12-week treatment with Rilutek alone (100 mg/day) during the observation period is followed by combined treatment (Rilutek + NDDPX08). The NDDPX08 dose level begins at 1.25 mg/day and is increased in steps to 15 mg/day during the 12-week treatment period according to the dose escalation schedule given on the next page (Fig. 1). If any serious adverse reaction arises following a dose increase to 10 mg/day and it is judged to be difficult to maintain this dose level, the dose level of 7.5 mg/day is regarded as the maintenance dose level. If alleviation of symptoms is noted during dose escalation steps, the dose level producing alleviation of symptoms is used as the maintenance dose level.
The total NDDPX08 treatment period is 58-90 weeks (including the 4 weeks during which the dose level is reduced in steps). Follow-up of adverse events is continued until 1 month after the end of NDDPX08 treatment.
Placebo (lactose) is administered to 10 of the 50 subjects planned to be enrolled in the study.

FeGALS

NULL

Complete: follow-up complete

20years-old

75years-old

Male and Female

Not selected

Japan