DDrare: Database of Drug Development for Rare Diseases

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	EUCTR2016-004664-18-DE (EUCTR)	09/02/2018	24/05/2017	A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201	A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)	The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201 MedDRA version: 20.0;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: NEOD001 INN or Proposed INN: N/A Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody	Prothena Therapeutics Limited	NULL	Not Recruiting			Female: yes Male: yes	100	Phase 2	France;United States;Greece;Spain;Austria;Australia;Israel;Germany;Italy;United Kingdom
2	EUCTR2016-004664-18-AT (EUCTR)	07/11/2017	20/09/2017	A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201	A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)	The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201 MedDRA version: 20.0;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: NEOD001 INN or Proposed INN: N/A Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody	Prothena Therapeutics Limited	NULL	Not Recruiting			Female: yes Male: yes	100	Phase 2	France;Australia;Austria;Israel;Germany;Italy;United Kingdom;United States;Greece;Spain
3	EUCTR2016-004664-18-GR (EUCTR)	27/09/2017	02/06/2017	A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201	A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)	The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201 MedDRA version: 20.0;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: NEOD001 INN or Proposed INN: N/A Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody	Prothena Therapeutics Limited	NULL	Not Recruiting			Female: yes Male: yes	100	Phase 2	France;United States;Greece;Spain;Austria;Australia;Israel;Germany;Italy;United Kingdom
4	EUCTR2016-004664-18-GB (EUCTR)	08/08/2017	09/05/2017	A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201	A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)	The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201 MedDRA version: 20.0;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: NEOD001 INN or Proposed INN: N/A Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody	Prothena Therapeutics Limited	NULL	Not Recruiting			Female: yes Male: yes	100	Phase 2	France;United States;Greece;Spain;Austria;Australia;Israel;Germany;Italy;United Kingdom
5	EUCTR2016-004664-18-ES (EUCTR)	31/07/2017	29/05/2017	A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201	A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)	The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201 MedDRA version: 20.0;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: NEOD001 INN or Proposed INN: N/A Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody	Prothena Therapeutics Limited	NULL	Not Recruiting			Female: yes Male: yes	100	Phase 2	France;United States;Greece;Spain;Austria;Australia;Israel;Germany;Italy;United Kingdom
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
6	NCT03154047 (ClinicalTrials.gov)	June 14, 2017	28/4/2017	Study in Subjects With Light Chain (AL) Amyloidosis	A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects With Light Chain (AL) Amyloidosis Who Were Previously Enrolled in Study NEOD001-201 (PRONTO)	AL Amyloidosis	Drug: NEOD001	Prothena Therapeutics Ltd.	NULL	Terminated	18 Years	N/A	All	80	Phase 2	United States;Australia;Austria;France;Germany;Greece;Israel;Italy;Spain;United Kingdom
7	EUCTR2015-004318-14-AT (EUCTR)	07/07/2016	22/08/2016	A study to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.	A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction	Light chain (AL) amyloidosis involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains results in both soluble, aggregated forms of light chains and insoluble,fibrillar deposits of abnormal AL protein (amyloid),in the tissues and organs. This can cause a range of symptoms and organ dysfunction including cardiac,renal,and hepatic dysfunction,gastrointestinal involvement and neuropathy and macroglossia MedDRA version: 20.0;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: NEOD001 INN or Proposed INN: N/A Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody	Prothena Therapeutics Limited	NULL	Not Recruiting			Female: yes Male: yes	129	Phase 2	France;United States;Greece;Spain;Australia;Austria;Israel;Germany;Italy;United Kingdom
8	EUCTR2015-004318-14-DE (EUCTR)	13/06/2016	26/11/2015	A study to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.	A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction	Light chain (AL) amyloidosis involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains results in both soluble, aggregated forms of light chains and insoluble,fibrillar deposits of abnormal AL protein (amyloid),in the tissues and organs. This can cause a range of symptoms and organ dysfunction including cardiac,renal,and hepatic dysfunction,gastrointestinal involvement and neuropathy and macroglossia MedDRA version: 20.0;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: NEOD001 INN or Proposed INN: N/A Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody	Prothena Therapeutics Limited	NULL	Not Recruiting			Female: yes Male: yes	129	Phase 2	United States;France;Greece;Spain;Austria;Australia;Israel;Germany;United Kingdom;Italy
9	EUCTR2015-004318-14-GR (EUCTR)	21/04/2016	11/02/2016	A study to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.	A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction	Light chain (AL) amyloidosis involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains results in both soluble, aggregated forms of light chains and insoluble,fibrillar deposits of abnormal AL protein (amyloid),in the tissues and organs. This can cause a range of symptoms and organ dysfunction including cardiac,renal,and hepatic dysfunction,gastrointestinal involvement and neuropathy and macroglossia MedDRA version: 18.1;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: NEOD001 INN or Proposed INN: N/A Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody	Prothena Therapeutics Limited	NULL	Not Recruiting			Female: yes Male: yes	100	Phase 2	France;United States;Greece;Spain;Austria;Germany;Italy;United Kingdom
10	EUCTR2015-004318-14-ES (EUCTR)	10/03/2016	18/03/2016	A study is to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.	A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction	Light chain (AL) amyloidosis or primary systemic amyloidosis, involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains by plasma cells results in both soluble, aggregated forms of light chains and insoluble, fibrillar deposits of abnormal AL protein (amyloid), in the tissues and organs of individuals with AL amyloidosis. MedDRA version: 18.1;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: NEOD001 INN or Proposed INN: N/A Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody	Prothena Therapeutics Limited	NULL	Not Recruiting			Female: yes Male: yes	100	Phase 2	France;United States;Greece;Spain;Austria;Germany;Italy;United Kingdom
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
11	NCT02632786 (ClinicalTrials.gov)	March 2016	9/12/2015	The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis	A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis Who Have Persistent Cardiac Dysfunction	AL Amyloidosis	Drug: NEOD001;Drug: Placebo	Prothena Therapeutics Ltd.	NULL	Completed	18 Years	N/A	All	129	Phase 2	United States;Australia;Austria;France;Germany;Greece;Israel;Italy;Spain;United Kingdom
12	EUCTR2015-004318-14-GB (EUCTR)	05/02/2016	27/11/2015	A study to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.	A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction	Light chain (AL) amyloidosis involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains results in both soluble, aggregated forms of light chains and insoluble,fibrillar deposits of abnormal AL protein (amyloid),in the tissues and organs. This can cause a range of symptoms and organ dysfunction including cardiac,renal,and hepatic dysfunction,gastrointestinal involvement and neuropathy and macroglossia MedDRA version: 20.0;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: NEOD001 INN or Proposed INN: N/A Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody	Prothena Therapeutics Limited	NULL	Not Recruiting			Female: yes Male: yes	129	Phase 2	United States;France;Greece;Spain;Austria;Australia;Israel;Germany;Italy;United Kingdom
13	NCT02613182 (ClinicalTrials.gov)	February 2016	18/11/2015	Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis	Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of NEOD001 in Subjects With Light Chain (AL) Amyloidosis	AL Amyloidosis	Drug: NEOD001	Prothena Therapeutics Ltd.	NULL	Terminated	N/A	N/A	All	34	Phase 2	United States
14	NCT02312206 (ClinicalTrials.gov)	February 2015	2/12/2014	The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis	A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, 2-Arm, Efficacy and Safety Study of NEOD001 Plus Standard of Care Versus Placebo Plus Standard of Care in Subjects With Light Chain (AL) Amyloidosis	Primary Systemic (AL) Amyloidosis	Drug: NEOD001;Other: Placebo	Prothena Therapeutics Ltd.	NULL	Terminated	18 Years	N/A	All	260	Phase 3	United States;Australia;Austria;Belgium;Canada;Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Poland;Spain;United Kingdom

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

EUCTR2016-004664-18-DE
(EUCTR)

09/02/2018

24/05/2017

A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201

A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)

The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201
MedDRA version: 20.0;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: NEOD001
INN or Proposed INN: N/A
Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody

Prothena Therapeutics Limited

NULL

Not Recruiting

Female: yes
Male: yes

100

Phase 2

France;United States;Greece;Spain;Austria;Australia;Israel;Germany;Italy;United Kingdom

EUCTR2016-004664-18-AT
(EUCTR)

07/11/2017

20/09/2017

A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201

A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)

Product Name: NEOD001
INN or Proposed INN: N/A
Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody

Prothena Therapeutics Limited

NULL

Not Recruiting

Female: yes
Male: yes

100

Phase 2

France;Australia;Austria;Israel;Germany;Italy;United Kingdom;United States;Greece;Spain

EUCTR2016-004664-18-GR
(EUCTR)

27/09/2017

02/06/2017

A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201

A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)

Product Name: NEOD001
INN or Proposed INN: N/A
Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody

Prothena Therapeutics Limited

NULL

Not Recruiting

Female: yes
Male: yes

100

Phase 2

France;United States;Greece;Spain;Austria;Australia;Israel;Germany;Italy;United Kingdom

EUCTR2016-004664-18-GB
(EUCTR)

08/08/2017

09/05/2017

A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201

A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)

Product Name: NEOD001
INN or Proposed INN: N/A
Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody

Prothena Therapeutics Limited

NULL

Not Recruiting

Female: yes
Male: yes

100

Phase 2

France;United States;Greece;Spain;Austria;Australia;Israel;Germany;Italy;United Kingdom

EUCTR2016-004664-18-ES
(EUCTR)

31/07/2017

29/05/2017

A study to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who completed Study NEOD001 201

A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects with Light Chain (AL) Amyloidosis who were previously enrolled in Study NEOD001-201 (PRONTO)

Product Name: NEOD001
INN or Proposed INN: N/A
Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody

Prothena Therapeutics Limited

NULL

Not Recruiting

Female: yes
Male: yes

100

Phase 2

France;United States;Greece;Spain;Austria;Australia;Israel;Germany;Italy;United Kingdom

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

NCT03154047
(ClinicalTrials.gov)

June 14, 2017

28/4/2017

Study in Subjects With Light Chain (AL) Amyloidosis

A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects With Light Chain (AL) Amyloidosis Who Were Previously Enrolled in Study NEOD001-201 (PRONTO)

AL Amyloidosis

Drug: NEOD001

Prothena Therapeutics Ltd.

NULL

Terminated

18 Years

N/A

All

Phase 2

United States;Australia;Austria;France;Germany;Greece;Israel;Italy;Spain;United Kingdom

EUCTR2015-004318-14-AT
(EUCTR)

07/07/2016

22/08/2016

A study to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.

A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction

Light chain (AL) amyloidosis involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains results in both soluble, aggregated forms of light chains and insoluble,fibrillar deposits of abnormal AL protein (amyloid),in the tissues and organs. This can cause a range of symptoms and organ dysfunction including cardiac,renal,and hepatic dysfunction,gastrointestinal involvement and neuropathy and macroglossia
MedDRA version: 20.0;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: NEOD001
INN or Proposed INN: N/A
Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody

Prothena Therapeutics Limited

NULL

Not Recruiting

Female: yes
Male: yes

129

Phase 2

France;United States;Greece;Spain;Australia;Austria;Israel;Germany;Italy;United Kingdom

EUCTR2015-004318-14-DE
(EUCTR)

13/06/2016

26/11/2015

A study to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.

A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction

Product Name: NEOD001
INN or Proposed INN: N/A
Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody

Prothena Therapeutics Limited

NULL

Not Recruiting

Female: yes
Male: yes

129

Phase 2

United States;France;Greece;Spain;Austria;Australia;Israel;Germany;United Kingdom;Italy

EUCTR2015-004318-14-GR
(EUCTR)

21/04/2016

11/02/2016

A study to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.

A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction

Light chain (AL) amyloidosis involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains results in both soluble, aggregated forms of light chains and insoluble,fibrillar deposits of abnormal AL protein (amyloid),in the tissues and organs. This can cause a range of symptoms and organ dysfunction including cardiac,renal,and hepatic dysfunction,gastrointestinal involvement and neuropathy and macroglossia
MedDRA version: 18.1;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: NEOD001
INN or Proposed INN: N/A
Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody

Prothena Therapeutics Limited

NULL

Not Recruiting

Female: yes
Male: yes

100

Phase 2

France;United States;Greece;Spain;Austria;Germany;Italy;United Kingdom

EUCTR2015-004318-14-ES
(EUCTR)

10/03/2016

18/03/2016

A study is to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.

A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction

Light chain (AL) amyloidosis or primary systemic amyloidosis, involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains by plasma cells results in both soluble, aggregated forms of light chains and insoluble, fibrillar deposits of abnormal AL protein (amyloid), in the tissues and organs of individuals with AL amyloidosis.
MedDRA version: 18.1;Level: PT;Classification code 10036673;Term: Primary amyloidosis;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: NEOD001
INN or Proposed INN: N/A
Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody

Prothena Therapeutics Limited

NULL

Not Recruiting

Female: yes
Male: yes

100

Phase 2

France;United States;Greece;Spain;Austria;Germany;Italy;United Kingdom

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

NCT02632786
(ClinicalTrials.gov)

March 2016

9/12/2015

The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis

A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis Who Have Persistent Cardiac Dysfunction

AL Amyloidosis

Drug: NEOD001;Drug: Placebo

Prothena Therapeutics Ltd.

NULL

Completed

18 Years

N/A

All

129

Phase 2

United States;Australia;Austria;France;Germany;Greece;Israel;Italy;Spain;United Kingdom

EUCTR2015-004318-14-GB
(EUCTR)

05/02/2016

27/11/2015

A study to evaluate whether NEOD001 is safe and effective in subjects with light chain AL amyloidosis affecting the heart.

A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction

Product Name: NEOD001
INN or Proposed INN: N/A
Other descriptive name: Humanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody

Prothena Therapeutics Limited

NULL

Not Recruiting

Female: yes
Male: yes

129

Phase 2

United States;France;Greece;Spain;Austria;Australia;Israel;Germany;Italy;United Kingdom

NCT02613182
(ClinicalTrials.gov)

February 2016

18/11/2015

Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis

Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of NEOD001 in Subjects With Light Chain (AL) Amyloidosis

AL Amyloidosis

Drug: NEOD001

Prothena Therapeutics Ltd.

NULL

Terminated

N/A

All

Phase 2

United States

NCT02312206
(ClinicalTrials.gov)

February 2015

2/12/2014

The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis

A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, 2-Arm, Efficacy and Safety Study of NEOD001 Plus Standard of Care Versus Placebo Plus Standard of Care in Subjects With Light Chain (AL) Amyloidosis

Primary Systemic (AL) Amyloidosis

Drug: NEOD001;Other: Placebo

Prothena Therapeutics Ltd.

NULL

Terminated

18 Years

N/A

All

260

Phase 3

United States;Australia;Austria;Belgium;Canada;Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Poland;Spain;United Kingdom