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	JPRN-UMIN000006604	2011/11/01	24/10/2011	Phase I/II study to evaluate the efficacy and safety of the combination treatment of melpharan, dexamethasone and bortezomib for relapsed or refractory systemic AL amyloidosis.		To evaluate the efficacy and safety of the combination treatment melpharan, dexamethasone and bortezomib (BMD treatment) for relapsed or refractory systemic AL amyloidosis.	Patients receive oral melphalan 8 mg/m2 on days 1-4, bortezomib SC (IV) on days 1, 4, 8 and 11, and dexamethasone orally on days 1-2, 4-5 8-9 11 and 12. Treatment repeats every 4 weeks (28 days) for up to 4 cycles in the absence of disease progression or unacceptable toxicity. *The preventive medication on last medication day after the 28th is recommended in principle from acyclovir 200 or 400 mg/the bortezomib medication opening day of a day. Moreover, when the symptoms of herpes are shown, the acyclovir or the rose cyclo building of a therapeutic amount is promptly prescribed for the patient.	Japan Community Health care Organization Kyoto kuramaguchi Medical Center, Department of Hematology	NULL	Complete: follow-up complete	20years-old	65years-old	Male and Female	21	Phase 1;Phase 2	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-UMIN000006604

2011/11/01

24/10/2011

Phase I/II study to evaluate the efficacy and safety of the combination treatment of melpharan, dexamethasone and bortezomib for relapsed or refractory systemic AL amyloidosis.

To evaluate the efficacy and safety of the combination treatment melpharan, dexamethasone and bortezomib (BMD treatment) for relapsed or refractory systemic AL amyloidosis.

Patients receive oral melphalan 8 mg/m2 on days 1-4, bortezomib SC (IV) on days 1, 4, 8 and 11, and dexamethasone orally on days 1-2, 4-5 8-9 11 and 12. Treatment repeats every 4 weeks (28 days) for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
*The preventive medication on last medication day after the 28th is recommended in principle from acyclovir 200 or 400 mg/the bortezomib medication opening day of a day.
Moreover, when the symptoms of herpes are shown, the acyclovir or the rose cyclo building of a therapeutic amount is promptly prescribed for the patient.

Japan Community Health care Organization Kyoto kuramaguchi Medical Center, Department of Hematology

NULL

Complete: follow-up complete

20years-old

65years-old

Male and Female

Phase 1;Phase 2

Japan