DDrare: Database of Drug Development for Rare Diseases

28. 全身性アミロイドーシス
［臨床試験数：212，薬物数：234（DrugBank：72），標的遺伝子数：54，標的パスウェイ数：170］

Searched query = "Systemic amyloidosis", "AL amyloidosis", "Immunoglobulin light chain amyloidosis", "Amyloid light-chain amyloidosis", "Familial amyloidosis", "Familial amyloid polyneuropathy", "Senile transthyretin amyloidosis", "Senile TTR amyloidosis"
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 = 124I-labeled monoclonal antibody Mu 11-1F4; 124I-p5+14 Injection; 1H-Pyrazol; 2',4'-difluoro-4-hydroxy-3-biphenylcarboxylic acid; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylat; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate; 26866138; 3-(3-(3,5-DIMETHYL-1H-PYRAZOL-4-YL)PROPOXY)-4-FLUOROBENZOIC ACID; 4'-iodo-4'-deoxydoxorubicin; A01AC02; AG10; AG10 HCl; AL; ALKERAN; ALN-51547; ALN-TTRSC; Acyclovir; Alkeran; Amifostine; Amifostine trihydrate; Amiodarone; Anakinra; Aprotinin; BD; BMDex; BORTEZOMIB; Behavioral: Questionnaires; Behavioral: The M.D. Anderson Symptom Inventory (MDASI); Belantamab mafodotin; Bendamustine; Betamethasone; Bortezomib; Bortezomib/Dexamethasone (BD), Followed By Autologous STC & Maintenance Bortezomib/Dexamethasone; Busulfan; CAEL-101; CC; CC-4047; CC-5013; CHIMERIC FIBRIL-REACTIVE IGG1K MONOCLONAL ANTIBODY 11-1F4; CHX A-DTPA anti-CD66 antibody; CHX-A-DTPA-Anti-CD66; CPHC (carboxy pyrrolidine hexanoyl pyrrolidone carboxylate); CYCLOPHOSPHAMIDE; Carboplatin; Carfilzomib; Carmustine; Chemotherapy; Chimeric Fibril-Reactive Monoclonal Anti-body 11-1F4; Cisplatin; Cyclophosphamide; Cyclophosphamide, Bortezomib, dexamethasone; Cyclophosphamide, Bortezomib, dexamethasone plus Daratumumab; Cyclophosphamide, bortezomib, and Dexamethasone (CyBorD); Cyclophosphamide, bortezomib, and dexamethasone (CyBorD); Cyclosporine; D-glucitol; DARATUMUMAB; DEXAMETHASONE; DEXAMETHASONE SODIUM SULFATE; DOXYCYCLINE; DTPA; Daratumumab; Daratumumab co-formulated with recombinant human hyaluronidase (rHuPH20); Darzalex; Deaxamethasone; Decadron; Device: High Cut-off Hemodialysis; Dexametasone; Dexamethason; Dexamethasone; Dexamethasone 4 mg JENAPHARM; Dexamethasone, 40 mg; Dexaton; Dezamizumab; Diflunisal; Diflunisal (dolobid); Diuretics (plus antiarrhythmic drugs, i.e. amiodarone, in case of complex ventricular arrhithmias); Diuretics (plus antiarrhythmic drugs, i.e. amiodarone, in case of complex ventricular arrhythmias) plus EGCG; Dolobid; Donor lymphocytes; Doxorubicin; DoxyHEXAL® tabs 100 mg Tabletten; Doxycycline; EGCG; Elotuzumab; Enbrel; Endoxan; Epigallocatechin; Epigallocatechin-3-gallate (EGCG); Etoposide; FORTECORTIN; Filgrastim; Fludarabine; Fludarabine phosphate; Fortecortin; Foscarnet; Foscarnet sodium; Fx-1006A; GRANULOCYTE COLONY STIMULATING FACTOR; GSK2315698; GSK2398852; GSK3039294; Ganciclovir; Gelatin; Genetic: DNA analysis; Genetic: RNA analysis; Genetic: microarray analysis; Genetic: polymerase chain reaction; Genetic: protein expression analysis; Granulocyte Colony Stimulating Factor; Green Tea; Green Tea Extract; H02AB02; H02AB02 Desametasone; HUMAX-CD38; HuMax-CD38; Human Transthyretin Antisense Oligonucleotide; Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody; Hyaluronidase; INOTERSEN; ION-682884; IONIS-TTR Rx; ISIS 420915; Ibrutinib; Imatinib; Imatinib mesylate; Indium; Indium 111-radiolabelled anti-CD66; Indium 111 labelled Anti-CD66; Inoserten; Inotersen; Interferon alfa; Iodo; Isatuximab; Isis 420915/GSK 299872; Ixazomib; JNJ-54767414; JnJ 54767414; KINERET*SC 7SIR 100MG 0,67ML; Kineret; L01AA03; Lenalidomide; Lenalidomide 25mg; Lenalidomide, Dexamethasone and Cyclophosphamide; Lenogastrim; MELFLUFEN; MELPHALAN; Meglumine; Melflufen; Melphalan; Melphalan fulfenamide hydrochloride; Melphalan, thalidomide and dexamethasone; Melphalan-Flufenamide (Melflufen); Miridesap; Mycophenolate; Mycophenolate mofetil; N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; NEOD001; Neupogen; Null; Oral drops solution; Other: Laboratory Biomarker Analysis; Other: Pharmacological Study; Other: flow cytometry; Other: laboratory biomarker analysis; Other: pharmacological study; PEGYLATED RECOMBINANT HUMAN HYALURONIDASE PH20; PF-06291826-83/Fx-1006A; Phosphate; Pomalidomide; Pomalidomide and Dexamethasone; Procedure: Autologous stem cell transplantation; Procedure: Stem Cell Collection; Procedure: Stem Cell Infusion; Procedure: Stem cell infusion; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: antiviral therapy; Procedure: autologous bone marrow transplantation; Procedure: autologous hematopoietic stem cell transplantation; Procedure: autologous hematopoietic stem cell transplantation (ASCT); Procedure: autologous peripheral blood stem cell transplantation; Procedure: autologous stem cell transplant; Procedure: autologous stem cell transplantation; Procedure: autologous-autologous tandem hematopoietic stem cell transplantation; Procedure: bone marrow ablation with stem cell support; Procedure: infection prophylaxis and management; Procedure: management of therapy complications; Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: quality-of-life assessment; Procedure: stem cell infusion; Propylene Glycol-Free Melphalan Hydrochloride; Propylene glycol; REVLIMID; Radiation: total-body irradiation; Recombinant human hyaluronidase; Recombinant human hyaluronidase PH20; Recombinant interferon alfa; Revlimid; Revusiran; SOM0226; STI-6129; Sargramostim; Siltuximab; Single infusion of 124I-labeled anti-amyloid mAb 11-1F4: 2 mCi (1 mg); Sodium sulfate; Standard of care therapy; Sulfate; Sunitinib; Sunitinib malate; TOLCAPONE; Tafamidis; Tafamidis Meglumine 20mg Soft Gelatin Capsule (Clinical trial image); Tafamidis Meglumine 20mg Soft Gelatin Capsule (Commerical image); Tafamidis meglumine; Tafamidis meglumine 20 mg soft gelatin capsules; Tasmar; Tea Extract; Thalidomide; Thiotepa; Tolcapone; VELCADE; VELCADE*1FL 3,5MG; Valganciclovir; Velcade; Venetoclax; Vyndaqel; Vyndaqel 20mg soft capsule; Yttrium-90; Yttrium-90 radiolabelled anti-CD66 antibody; Yttrium90 radiolabelled antiCD66; [18F]Florbetaben; [d-glucitol, 1-deoxy-1-(methylamino)-, 2-(3,5-dichlorophenyl)-6-benzoxazolecarboxylate (1:1)]; [d-glucitol, 1-deoxy-1-(methylamino)-,2-(3,5-dichlorophenyl)-6-benzoxazolecarboxylate (1:1)]

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	EUCTR2014-001586-27-ES (EUCTR)	25/07/2014	02/06/2014	Study of SOM0226 in familial amyloid polyneuropathy (FAP) patients and asymptomatic carriers to evaluate protein stabilization activity	Study of SOM0226 in familial amyloid polyneuropathy (FAP) patients and asymptomatic carriers to evaluate protein stabilization activity	Patients with familial amyloid polyneuropathy (FAP), asymptomatic carriers and healthy volunteers MedDRA version: 17.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Trade Name: Tasmar INN or Proposed INN: TOLCAPONE	SOM Innovation Biotech SL (SOM Biotech)	NULL	Not Recruiting			Female: yes Male: yes			Spain

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EUCTR2014-001586-27-ES
(EUCTR)

25/07/2014

02/06/2014

Study of SOM0226 in familial amyloid polyneuropathy (FAP) patients and asymptomatic carriers to evaluate protein stabilization activity

Patients with familial amyloid polyneuropathy (FAP), asymptomatic carriers and healthy volunteers
MedDRA version: 17.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Trade Name: Tasmar
INN or Proposed INN: TOLCAPONE

SOM Innovation Biotech SL (SOM Biotech)

NULL

Not Recruiting

Female: yes
Male: yes

Spain