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-JapicCTI-111678	07/11/2011	02/11/2011	The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin	The Effect Of Transthyretin Stabilization, Safety, Tolerability, Efficacy And Pharmacokinetics Of Orally Administered Tafamidis In Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin: A Phase 3, Open-Label(B3461010)	Transthyretin Familial Amyloid Polyneuropathy	Intervention name : tafamidis meglumine INN of the intervention : tafamidis meglumine Dosage And administration of the intervention : 20 mg QD Control intervention name : null	Pfizer Japan Inc.	NULL		20	75	BOTH	10	Phase 3	NULL
2	EUCTR2009-011535-12-PT (EUCTR)	09/09/2009	15/07/2009	Open-Label Safety and Efficacy Evaluation of Fx-1006A in Patients with Transthyretin Amyloidosis	Open-Label Safety and Efficacy Evaluation of Fx-1006A in Patients with Transthyretin Amyloidosis	Transthyretin Amyloidosis (ATTR) MedDRA version: 14.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]	Product Name: Tafamidis meglumine Product Code: Fx-1006A INN or Proposed INN: tafamidis Other descriptive name: [d-glucitol, 1-deoxy-1-(methylamino)-,2-(3,5-dichlorophenyl)-6-benzoxazolecarboxylate (1:1)]	Pfizer Inc.	NULL	Not Recruiting			Female: yes Male: yes	110		France;United States;Portugal;Argentina;Brazil;Germany;Italy;Sweden
3	EUCTR2009-011535-12-DE (EUCTR)	26/08/2009	18/05/2009	In preparation	Open-Label Safety and Efficacy Evaluation of Fx-1006A in Patients with Transthyretin Amyloidosis	Transthyretin Amyloidosis (ATTR) MedDRA version: 14.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]	Product Name: Tafamidis meglumine Product Code: Fx-1006A INN or Proposed INN: tafamidis Other descriptive name: [d-glucitol, 1-deoxy-1-(methylamino)-, 2-(3,5-dichlorophenyl)-6-benzoxazolecarboxylate (1:1)]	Pfizer Inc.	NULL	Not Recruiting			Female: yes Male: yes	110		Portugal;Germany;Argentina;France;Italy;United States;Sweden;Brazil
4	EUCTR2009-011535-12-SE (EUCTR)	24/06/2009	10/06/2009	To obtain additional long-term, safety and efficacy data for tafamidis in subjects with transthyretin (TTR) familial amyloid polyneuropathy (TTR-FAP) and to continue to provide the Tafamidis to subjects with TTR-FAP who have completed Protocol Fx-006 or Protocol Fx1A-201	Open-Label Safety and Efficacy Evaluation of Fx-1006A in Subjects with Transthyretin (TTR) Amyloidosis	Transthyretin Amyloidosis (ATTR) MedDRA version: 16.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]	Trade Name: Vyndaqel 20mg soft capsule Product Name: Tafamidis Meglumine 20mg Soft Gelatin Capsule (Clinical trial image) Product Code: PF-06291826-83/Fx-1006A INN or Proposed INN: Tafamidis meglumine Other descriptive name: tafamidis meglumine 20 mg soft gelatin capsules Trade Name: Vyndaqel 20mg soft capsule Product Name: Tafamidis Meglumine 20mg Soft Gelatin Capsule (Commerical image) Product Code: PF-06291826-83/Fx-1006A INN or Proposed INN: Tafamidis meglumine Other descriptive name: tafamidis meglumine 20 mg soft gelatin capsules	Pfizer Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	110		Portugal;France;United States;Argentina;Brazil;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

JPRN-JapicCTI-111678

07/11/2011

02/11/2011

The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin

The Effect Of Transthyretin Stabilization, Safety, Tolerability, Efficacy And Pharmacokinetics Of Orally Administered Tafamidis In Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin: A Phase 3, Open-Label(B3461010)

Transthyretin Familial Amyloid Polyneuropathy

Intervention name : tafamidis meglumine
INN of the intervention : tafamidis meglumine
Dosage And administration of the intervention : 20 mg QD
Control intervention name : null

Pfizer Japan Inc.

NULL

BOTH

Phase 3

NULL

EUCTR2009-011535-12-PT
(EUCTR)

09/09/2009

15/07/2009

Open-Label Safety and Efficacy Evaluation of Fx-1006A in Patients with Transthyretin Amyloidosis

Transthyretin Amyloidosis (ATTR)
MedDRA version: 14.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Product Name: Tafamidis meglumine
Product Code: Fx-1006A
INN or Proposed INN: tafamidis
Other descriptive name: [d-glucitol, 1-deoxy-1-(methylamino)-,2-(3,5-dichlorophenyl)-6-benzoxazolecarboxylate (1:1)]

Pfizer Inc.

NULL

Not Recruiting

Female: yes
Male: yes

110

France;United States;Portugal;Argentina;Brazil;Germany;Italy;Sweden

EUCTR2009-011535-12-DE
(EUCTR)

26/08/2009

18/05/2009

In preparation

Open-Label Safety and Efficacy Evaluation of Fx-1006A in Patients with Transthyretin Amyloidosis

Transthyretin Amyloidosis (ATTR)
MedDRA version: 14.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Product Name: Tafamidis meglumine
Product Code: Fx-1006A
INN or Proposed INN: tafamidis
Other descriptive name: [d-glucitol, 1-deoxy-1-(methylamino)-, 2-(3,5-dichlorophenyl)-6-benzoxazolecarboxylate (1:1)]

Pfizer Inc.

NULL

Not Recruiting

Female: yes
Male: yes

110

Portugal;Germany;Argentina;France;Italy;United States;Sweden;Brazil

EUCTR2009-011535-12-SE
(EUCTR)

24/06/2009

10/06/2009

To obtain additional long-term, safety and efficacy data for tafamidis in subjects with transthyretin (TTR) familial amyloid polyneuropathy (TTR-FAP) and to continue to provide the Tafamidis to subjects with TTR-FAP who have completed Protocol Fx-006 or Protocol Fx1A-201

Open-Label Safety and Efficacy Evaluation of Fx-1006A in Subjects with Transthyretin (TTR) Amyloidosis

Transthyretin Amyloidosis (ATTR)
MedDRA version: 16.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Trade Name: Vyndaqel 20mg soft capsule
Product Name: Tafamidis Meglumine 20mg Soft Gelatin Capsule (Clinical trial image)
Product Code: PF-06291826-83/Fx-1006A
INN or Proposed INN: Tafamidis meglumine
Other descriptive name: tafamidis meglumine 20 mg soft gelatin capsules
Trade Name: Vyndaqel 20mg soft capsule
Product Name: Tafamidis Meglumine 20mg Soft Gelatin Capsule (Commerical image)
Product Code: PF-06291826-83/Fx-1006A
INN or Proposed INN: Tafamidis meglumine
Other descriptive name: tafamidis meglumine 20 mg soft gelatin capsules

Pfizer Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

110

Portugal;France;United States;Argentina;Brazil;Germany;Italy;Sweden