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	EUCTR2017-005115-13-ES (EUCTR)	25/04/2018	19/02/2018	A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3	A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3	Familial Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915	Ionis Pharmaceuticals, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	164	Phase 3	United Kingdom;New Zealand;Italy;Portugal;France;United States;Argentina;Brazil;Spain;Germany
2	EUCTR2017-005115-13-PT (EUCTR)	23/04/2018	06/03/2018	A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3	A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3	Familial Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915 Other descriptive name: Inotersen	Ionis Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	164	Phase 3	France;United States;Portugal;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom
3	EUCTR2013-004561-13-ES (EUCTR)	28/12/2015	30/10/2015	Not available	An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3	Familial Amyloid Polyneuropathy MedDRA version: 18.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915	Isis Pharmaceuticals, Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	195		Portugal;France;United States;Argentina;Brazil;Spain;Bulgaria;Germany;New Zealand;Italy;United Kingdom
4	EUCTR2013-004561-13-DE (EUCTR)	22/09/2015	15/06/2015	Not available	An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3	Familial Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915	Ionis Pharmaceuticals, Inc	NULL	Not Recruiting			Female: yes Male: yes	135	Phase 3	Portugal;France;United States;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom
5	EUCTR2013-004561-13-GB (EUCTR)	23/07/2015	09/02/2015	Not available	An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3	Familial Amyloid Polyneuropathy MedDRA version: 19.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915	Ionis Pharmaceuticals, Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	135	Phase 3	Portugal;France;United States;Argentina;Brazil;Spain;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	EUCTR2012-001831-30-BG (EUCTR)	23/04/2015	23/02/2015	A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy	A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2	Familial Amyloid Polyneuropathy MedDRA version: 18.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915	Isis Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	195	Phase 2;Phase 3	Portugal;France;United States;Mexico;Argentina;Brazil;Spain;Australia;Bulgaria;Germany;Italy;United Kingdom
7	EUCTR2012-001831-30-ES (EUCTR)	25/04/2014	07/03/2014	A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy	A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2	Familial Amyloid Polyneuropathy MedDRA version: 16.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915	Isis Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	195	Phase 2;Phase 3	United States;France;Portugal;Argentina;Brazil;Spain;Bulgaria;Germany;United Kingdom;Italy
8	EUCTR2013-004561-13-FR (EUCTR)	16/04/2014	17/06/2015	Not available	An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3	Familial Amyloid Polyneuropathy MedDRA version: 18.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915	Isis Pharmaceuticals, Inc	NULL	Not Recruiting			Female: yes Male: yes	195	Phase 3	United States;Portugal;France;Argentina;Spain;Brazil;Germany;United Kingdom;Italy
9	EUCTR2012-001831-30-IT (EUCTR)	04/07/2013	19/06/2013	A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy	A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2	Familial Amyloid Polyneuropathy MedDRA version: 14.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915	Isis Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	195	Phase 2;Phase 3	France;United States;Portugal;Spain;Brazil;Bulgaria;Germany;United Kingdom;Italy
10	EUCTR2012-001831-30-GB (EUCTR)	22/04/2013	16/11/2012	A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy	A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2	Familial Amyloid Polyneuropathy MedDRA version: 19.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915	Ionis Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	135	Phase 2;Phase 3	Portugal;France;United States;Argentina;Brazil;Spain;Bulgaria;Germany;New Zealand;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	EUCTR2012-001831-30-PT (EUCTR)	21/02/2013	20/11/2012	A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy	A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2	Familial Amyloid Polyneuropathy MedDRA version: 19.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915	Ionis Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	135	Phase 2;Phase 3	France;United States;Portugal;Argentina;Brazil;Spain;Bulgaria;Germany;New Zealand;Italy;United Kingdom
12	EUCTR2017-005115-13-FR (EUCTR)		27/02/2018	A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3	A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3	Familial Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915 Other descriptive name: Inotersen	Ionis Pharmaceuticals, Inc.	NULL	NA			Female: yes Male: yes	164	Phase 3	Portugal;United States;France;Argentina;Brazil;Spain;Germany;New Zealand;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

EUCTR2017-005115-13-ES
(EUCTR)

25/04/2018

19/02/2018

A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3

Familial Amyloid Polyneuropathy
MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915

Ionis Pharmaceuticals, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

164

Phase 3

United Kingdom;New Zealand;Italy;Portugal;France;United States;Argentina;Brazil;Spain;Germany

EUCTR2017-005115-13-PT
(EUCTR)

23/04/2018

06/03/2018

A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915
Other descriptive name: Inotersen

Ionis Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

164

Phase 3

France;United States;Portugal;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom

EUCTR2013-004561-13-ES
(EUCTR)

28/12/2015

30/10/2015

Not available

An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3

Familial Amyloid Polyneuropathy
MedDRA version: 18.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915

Isis Pharmaceuticals, Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

195

Portugal;France;United States;Argentina;Brazil;Spain;Bulgaria;Germany;New Zealand;Italy;United Kingdom

EUCTR2013-004561-13-DE
(EUCTR)

22/09/2015

15/06/2015

Not available

An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915

Ionis Pharmaceuticals, Inc

NULL

Not Recruiting

Female: yes
Male: yes

135

Phase 3

Portugal;France;United States;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom

EUCTR2013-004561-13-GB
(EUCTR)

23/07/2015

09/02/2015

Not available

An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3

Familial Amyloid Polyneuropathy
MedDRA version: 19.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915

Ionis Pharmaceuticals, Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

135

Phase 3

Portugal;France;United States;Argentina;Brazil;Spain;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

EUCTR2012-001831-30-BG
(EUCTR)

23/04/2015

23/02/2015

A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy

A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2

Familial Amyloid Polyneuropathy
MedDRA version: 18.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915

Isis Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

195

Phase 2;Phase 3

Portugal;France;United States;Mexico;Argentina;Brazil;Spain;Australia;Bulgaria;Germany;Italy;United Kingdom

EUCTR2012-001831-30-ES
(EUCTR)

25/04/2014

07/03/2014

A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy

A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2

Familial Amyloid Polyneuropathy
MedDRA version: 16.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915

Isis Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

195

Phase 2;Phase 3

United States;France;Portugal;Argentina;Brazil;Spain;Bulgaria;Germany;United Kingdom;Italy

EUCTR2013-004561-13-FR
(EUCTR)

16/04/2014

17/06/2015

Not available

An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915

Isis Pharmaceuticals, Inc

NULL

Not Recruiting

Female: yes
Male: yes

195

Phase 3

United States;Portugal;France;Argentina;Spain;Brazil;Germany;United Kingdom;Italy

EUCTR2012-001831-30-IT
(EUCTR)

04/07/2013

19/06/2013

A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy

A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2

Familial Amyloid Polyneuropathy
MedDRA version: 14.1;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915

Isis Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

195

Phase 2;Phase 3

France;United States;Portugal;Spain;Brazil;Bulgaria;Germany;United Kingdom;Italy

EUCTR2012-001831-30-GB
(EUCTR)

22/04/2013

16/11/2012

A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy

A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2

Familial Amyloid Polyneuropathy
MedDRA version: 19.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915

Ionis Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

135

Phase 2;Phase 3

Portugal;France;United States;Argentina;Brazil;Spain;Bulgaria;Germany;New Zealand;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

EUCTR2012-001831-30-PT
(EUCTR)

21/02/2013

20/11/2012

A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with Familial Amyloid Polyneuropathy

A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy - ISIS 420915-CS2

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915

Ionis Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

135

Phase 2;Phase 3

France;United States;Portugal;Argentina;Brazil;Spain;Bulgaria;Germany;New Zealand;Italy;United Kingdom

EUCTR2017-005115-13-FR
(EUCTR)

27/02/2018

A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3

Product Name: Human Transthyretin Antisense Oligonucleotide
Product Code: ISIS 420915
INN or Proposed INN: ISIS 420915
Other descriptive name: Inotersen

Ionis Pharmaceuticals, Inc.

NULL

Female: yes
Male: yes

164

Phase 3

Portugal;United States;France;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom