DDrare: Database of Drug Development for Rare Diseases

Searched query = "Polycystic kidney disease", "PKD", "PCKD", "Polycystic kidney", "ADPKD", "ARPKD"
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 = 15 or 45 mg; 30 or 60 mg; 30 or 90 mg; AL; AMIAS; AMLODIPINE; APRINOX; Acetate; Amias; Aminohippurate; Aminohippurate Sodium Inj 20%; Amlodipine; Anti-hypertensive medications; Antihypertensive drugs; Aprinox; Arginine; BARDOXOLONE METHYL; BENDROFLUMETHIAZIDE; BIM 23014; BISOPROLOL; BOSUTINIB; Bardoxolone; Bardoxolone Methyl; Bardoxolone methyl; Bardoxolone methyl 10 mg; Bardoxolone methyl 15 mg; Bardoxolone methyl 20 mg; Bardoxolone methyl 5 mg; Bardoxolone methyl capsules; Bardoxolone methyl oral capsule; Behavioral: Diet and water adjustment; Bendroflumethiazide; Bisoprolol; Bosutinib; Bosutinib (anhydrous); C03XA01; CANDESARTAN; CARDURA; Calcineurin inhibitors maintenance; Candesartan; Candesartan and Cilnidipine; Candesartan plus non-CCB agents; Certican; Certican Tabletten; Cilnidipine; Conventional therapy; Curcumin; DOXAZOSIN; Doxazosin; Emodin; Equal; Everolimus; G1117337; GENZ-682452; GLPG2737; GZ402671; Genz-682452-AU; HCT; High water intake; Hydralazine; Hydrochlorothiazide; ISTIN; Imidapril; Intravenous injection autologous mesenchymal stem cells; Iohexol; Iohexol Inj 300 milligrams per milliliter (MG/ML); Istin; JINARC - 15 MG +45 MG - COMPRESSA - USO ORALE - BLISTER(ALU/PVC) IN CONFEZIONE A PORTAFOGLIO - 56 COMPRESSE (28X15 MG + 28 X 45 MG); JINARC - 15 MG - COMPRESSA - USO ORALE - BLISTER (ALU/PVC) - 28 COMPRESSE; JINARC - 30 MG + 60 MG - COMPRESSA - USO ORALE - BLISTER(ALU/PVC) IN CONFEZIONE A PORTAFOGLIO - 56 COMPRESSE (28 X 30 MG + 28 X 60 MG); JINARC - 30 MG +90 MG - COMPRESSA - USO ORALE - BLISTER(ALU/PVC) IN CONFEZIONE A PORTAFOGLIO - 56 COMPRESSE (28 X 30 MG + 28 X 90 MG); JINARC - 30 MG - COMPRESSA - USO ORALE - BLISTER (ALU/PVC) - 28 COMPRESSE; Jinarc; Jinarc - Tolvaptan tablets; Jinarc 15 mg tablets, Jinarc 45 mg tablets; Jinarc 30 mg tablets, 90 mg tablets; Jinarc 30 mg tablets, Jinarc 60 mg tablets; Jinarc® 15mg Tablet; Jinarc® 30 Tablet; KD019 (tesevatinib); LANREOTIDE ACETATE; LISINOPRIL; Lanreotide; Lisinopril; Lixivaptan; Long-acting somatostatin; Losartan; METFORMIN; METFORMINA; MONOCOR; Metformin; Metformina; Monocor; NG-Monomethyl-L-arginine; Ng-monomethyl-L-arginine (drug); Niacinamide; OPC-41061; OPC-41061 (tolvaptan); Octeotride; Octreotide; Octreotide LAR; Octreotide-LAR; Other: Ad libitum water intake; Other: High Water Intake; Other: Low Blood Pressure Control; Other: Saline solution; Other: Saline solution.; Other: Standard; Other: Standard Blood Pressure Control; Other: Water; Other: Water prescription; Other: water; PF-05208763; Pasireotide; Pasireotide LAR; Pioglitazone; Placebo; Potassium; Pravastatin; Procedure: renal sympathetic denervation; Protein; RAD001; RAPAMUNE*100CPR RIV 1MG; RAPAMUNE*30CPR RIV 1MG; RAPAMUNE*30CPR RIV 2MG; RGLS4326; RTA 402; Radiation: PET/CT Scan; Ramipril; Rapamune; Rapamune 1mg tablets; Rapamycin; SANDOSTATINA - LAR 20 MG/2.5 ML POLVERE E SOLVENTE PER SOSPENSIONE INIETTABILE FLACONE POLVERE + SIRINGA PRERIEMPITA 2.5 ML + 2 AGHI; SANDOSTATINA LAR; SAR402671, GZ402671 or GZ/SAR402671; SIROLIMUS; SKI-606; Saline; Samsca; Samsca 15 mg tablets; Samsca 30 mg tablets; Sandostatin LAR; Sandostatina; Sandostatina LAR; Sandostatina Lar IM Fl 20mg +2F; Sirolimus; Sodium citrate; Sodiumchloride; Somatostatin; Somatuline; Somatuline Autogel; Somatuline Autosolution; Spironolactone; Sugar pill; TOLVAPTAN; TOLVAPTAN (OPC-41061); TOLVAPTAN 15; TOLVAPTAN 30; TOLVAPTAN 45/15; TOLVAPTAN 60/30; Telmisartan; Tesevatinib; Tetracosactin; Tolvaptan; Tolvaptan (OPC-41061); Tolvaptan 15 mg Tablet; Tolvaptan 15 mg tablets; Tolvaptan 15mg; Tolvaptan 30 mg Tablet; Tolvaptan 30 mg tablets; Tolvaptan 30mg; Tolvaptan 7.5 mg Tablet; Tolvaptan IR; Tolvaptan MR; Tripterygium; Tripterygium Wilfordii; Tripterygium wilfordii; Triptolide; Triptolide-Containing Formulation; UDCA; URSOCHOL 300; Unknown; Ursochol; Ursodeoxycholic Acid; Ursodeoxycholic acid; Venglustat; Venglustat GZ/SAR402671; Venglustat GZ402671; Water; ZESTRIL; ZUGLIMET - 500 MG COMPRESSE RIVESTITE CON FILM 30 COMPRESSE IN BLISTER PVC/AL; [METFORMINA]; [TOL; [TOLVAPTAN 15]; [TOLVAPTAN 30]; [TOLVAPTAN 45/15]

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	EUCTR2017-004084-12-AT (EUCTR)	11/04/2019	14/01/2019	A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients	Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD	Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU	Genzyme Corporation	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	982	Phase 2	Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Japan;Korea, Republic of;United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada
2	EUCTR2017-004084-12-GB (EUCTR)	28/01/2019	24/05/2018	A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients	Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD	Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU	Genzyme Corporation	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	982	Phase 2	United States;Portugal;Czechia;Taiwan;Spain;Austria;Switzerland;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of
3	EUCTR2017-004084-12-DK (EUCTR)	07/11/2018	23/05/2018	A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients	Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD	Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU	Genzyme Corporation	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	982	Phase 2	United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Denmark;Australia;Germany;Netherlands;China;Japan;Korea, Republic of
4	EUCTR2017-004084-12-BE (EUCTR)	31/08/2018	31/05/2018	A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients	Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD	Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU	Genzyme Corporation	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	982	Phase 2	United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Japan;Korea, Republic of
5	EUCTR2017-004084-12-PT (EUCTR)	06/08/2018	11/06/2018	A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients	Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD	Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU	Genzyme Corporation	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	982	Phase 2	United States;Portugal;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of
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	EUCTR2017-004084-12-DE (EUCTR)	10/07/2018	22/05/2018	A Medical Research Study Designed to Determine if Venglustat can be aFuture Treatment for ADPKD Patients	Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD	Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU	Genzyme Corporation	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	982	Phase 2	United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Japan;Korea, Republic of
7	EUCTR2017-004084-12-CZ (EUCTR)	03/07/2018	13/06/2018	A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients	Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD	Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452 -AU	Genzyme Corporation	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	982	Phase 2	United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of

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

EUCTR2017-004084-12-AT
(EUCTR)

11/04/2019

14/01/2019

A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients

Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD

Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452 -AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452 -AU

Genzyme Corporation

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

982

Phase 2

Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Japan;Korea, Republic of;United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada

EUCTR2017-004084-12-GB
(EUCTR)

28/01/2019

24/05/2018

A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients

Genzyme Corporation

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

982

Phase 2

United States;Portugal;Czechia;Taiwan;Spain;Austria;Switzerland;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of

EUCTR2017-004084-12-DK
(EUCTR)

07/11/2018

23/05/2018

A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients

Genzyme Corporation

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

982

Phase 2

United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Denmark;Australia;Germany;Netherlands;China;Japan;Korea, Republic of

EUCTR2017-004084-12-BE
(EUCTR)

31/08/2018

31/05/2018

A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients

Genzyme Corporation

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

982

Phase 2

United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Japan;Korea, Republic of

EUCTR2017-004084-12-PT
(EUCTR)

06/08/2018

11/06/2018

A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients

Genzyme Corporation

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

982

Phase 2

United States;Portugal;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of

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

EUCTR2017-004084-12-DE
(EUCTR)

10/07/2018

22/05/2018

A Medical Research Study Designed to Determine if Venglustat can be aFuture Treatment for ADPKD Patients

Genzyme Corporation

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

982

Phase 2

United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Japan;Korea, Republic of

EUCTR2017-004084-12-CZ
(EUCTR)

03/07/2018

13/06/2018

A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients

Genzyme Corporation

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

982

Phase 2

United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of