DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
231	α1-アンチトリプシン欠乏症	9
298	遺伝性膵炎	1
299	嚢胞性線維症	2

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	EUCTR2015-004110-23-PL (EUCTR)	19/04/2018	16/02/2018	A Study with an active treatment to Assess the Long-term Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin Deficiency	An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), ModifiedProcess 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency - SPARTA-OLE	Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 20.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]	Trade Name: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN	Grifols Therapeutics LLC	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	250	Phase 3	United States;Estonia;Canada;Poland;Denmark;Australia;Russian Federation;New Zealand;Sweden
2	EUCTR2013-001870-38-DE (EUCTR)	21/09/2015	27/03/2015	A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency	A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA	Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 19.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]	Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN	Grifols Therapeutics Inc.	NULL	Not Recruiting			Female: yes Male: yes	339	Phase 3	United States;Estonia;Finland;Spain;Ireland;Russian Federation;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;Germany;New Zealand;Sweden
3	EUCTR2013-001870-38-PL (EUCTR)	30/10/2014	29/08/2014	A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency	A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA	Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 17.1;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]	Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN	Grifols Therapeutics Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	339		United States;Estonia;Slovakia;Finland;Spain;Ireland;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;New Zealand;Sweden
4	EUCTR2013-001870-38-IE (EUCTR)	21/03/2014	27/09/2013	A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency	A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA	Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 16.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]	Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN	Grifols Therapeutics Inc.	NULL	Not Recruiting			Female: yes Male: yes	339	Phase 3	Serbia;United States;Estonia;Slovakia;Finland;Spain;Ireland;Lithuania;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Brazil;Denmark;Australia;Germany;Netherlands;Latvia;Norway;New Zealand;Sweden
5	EUCTR2013-001870-38-SE (EUCTR)	09/01/2014	23/09/2013	A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency	A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA	Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency MedDRA version: 20.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]	Trade Name: Prolastin-C Product Name: Alpha1-Proteinase Inhibitor (Human) Product Code: Prolastin-C INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human) Other descriptive name: ALPHA-1-ANTITRYPSIN	Grifols Therapeutics LLC	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	339	Phase 3	United States;Serbia;Estonia;Finland;Spain;Ireland;Russian Federation;United Kingdom;France;Canada;Brazil;Poland;Romania;Australia;Denmark;Germany;Moldova, Republic of;New Zealand;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
6	NCT01213043 (ClinicalTrials.gov)	November 2010	29/9/2010	Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency	A Randomized Double-blind Crossover Study to Assess the Safety and Pharmacokinetics of Two Different Doses of Weekly Intravenous Administration of Alpha1-Proteinase Inhibitor (Human) Prolastin®-C in Subjects With Alpha1-Antitrypsin Deficiency	Emphysema;Alpha 1-antitrypsin Deficiency (AATD)	Biological: Prolastin-C, 60 mg/kg;Biological: Prolastin-C, 120 mg/kg	Grifols Therapeutics Inc.	NULL	Completed	18 Years	70 Years	All	30	Phase 2	United States
7	EUCTR2007-004869-18-GB (EUCTR)	03/07/2008	04/04/2008	Evaluation of the relative severity of pulmonary neutrophilic inflammation and therapeutic modification with IV Prolastin by means of 18 FDG PET/CT scanning in subjects with alpha1-antitrypsin deficiency. - ECLIPSE AATD	Evaluation of the relative severity of pulmonary neutrophilic inflammation and therapeutic modification with IV Prolastin by means of 18 FDG PET/CT scanning in subjects with alpha1-antitrypsin deficiency. - ECLIPSE AATD	The medical condition is emphysema and will be investigated in subjects with alpha 1-antitrypsin deficiency. MedDRA version: 9.1;Level: LLT;Classification code 10001811;Term: Alpha-1 proteinase inhibitor deficiency MedDRA version: 9.1;Classification code 10014563;Term: Emphysema pulmonary MedDRA version: 9.1;Classification code 10010952;Term: COPD MedDRA version: 9.1;Classification code 10006458;Term: Bronchitis chronic		UHB NHS Foundation Trust	NULL	Not Recruiting			Female: yes Male: yes	26	Phase 2	United Kingdom
8	NCT00295061 (ClinicalTrials.gov)	May 2006	20/2/2006	Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults	Multi-center, Randomized, Double-blind, Crossover Trial to Evaluate the Pharmacokinetic Comparability of Alpha-1 MP to Prolastin in Subjects With Alpha1-antitrypsin Deficiency.	Alpha 1-Antitrypsin Deficiency	Drug: Alpha-1 MP;Drug: alpha-1 proteinase inhibitor (human)	Grifols Therapeutics Inc.	NULL	Completed	18 Years	N/A	All	24	Phase 3	United States
9	NCT00263887 (ClinicalTrials.gov)	December 2003	12/9/2005	Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)	Multi-center, Randomized Trial With I.V. Prolastin® to Evaluate Frequency of Exacerbations and Progression of Emphysema by Means of Multi-slice CT Scans in Patients With Congenital Alpha-1-antitrypsin Deficiency.	Alpha 1-Antitrypsin Deficiency	Drug: Alpha1-Proteinase Inhibitor (Human);Drug: Albumin (Human) 20%, United States Pharmacopeia (USP)	Grifols Therapeutics Inc.	NULL	Completed	18 Years	N/A	All	77	Phase 2	Denmark;Sweden;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

EUCTR2015-004110-23-PL
(EUCTR)

19/04/2018

16/02/2018

A Study with an active treatment to Assess the Long-term Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha1-Antitrypsin Deficiency

An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), ModifiedProcess 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency - SPARTA-OLE

Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency
MedDRA version: 20.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Trade Name: Prolastin-C
INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human)
Other descriptive name: ALPHA-1-ANTITRYPSIN

Grifols Therapeutics LLC

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

250

Phase 3

United States;Estonia;Canada;Poland;Denmark;Australia;Russian Federation;New Zealand;Sweden

EUCTR2013-001870-38-DE
(EUCTR)

21/09/2015

27/03/2015

A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency

A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency - SPARTA

Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency
MedDRA version: 19.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Trade Name: Prolastin-C
Product Name: Alpha1-Proteinase Inhibitor (Human)
Product Code: Prolastin-C
INN or Proposed INN: Alpha1-Proteinase Inhibitor (Human)
Other descriptive name: ALPHA-1-ANTITRYPSIN

Grifols Therapeutics Inc.

NULL

Not Recruiting

Female: yes
Male: yes

339

Phase 3

United States;Estonia;Finland;Spain;Ireland;Russian Federation;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;Germany;New Zealand;Sweden

EUCTR2013-001870-38-PL
(EUCTR)

30/10/2014

29/08/2014

A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency

Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency
MedDRA version: 17.1;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Grifols Therapeutics Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

339

United States;Estonia;Slovakia;Finland;Spain;Ireland;France;Canada;Argentina;Poland;Brazil;Romania;Australia;Denmark;New Zealand;Sweden

EUCTR2013-001870-38-IE
(EUCTR)

21/03/2014

27/09/2013

A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency

Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency
MedDRA version: 16.0;Level: LLT;Classification code 10014563;Term: Emphysema pulmonary;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Grifols Therapeutics Inc.

NULL

Not Recruiting

Female: yes
Male: yes

339

Phase 3

Serbia;United States;Estonia;Slovakia;Finland;Spain;Ireland;Lithuania;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Brazil;Denmark;Australia;Germany;Netherlands;Latvia;Norway;New Zealand;Sweden

EUCTR2013-001870-38-SE
(EUCTR)

09/01/2014

23/09/2013

A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency

Grifols Therapeutics LLC

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

339

Phase 3

United States;Serbia;Estonia;Finland;Spain;Ireland;Russian Federation;United Kingdom;France;Canada;Brazil;Poland;Romania;Australia;Denmark;Germany;Moldova, Republic of;New Zealand;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

NCT01213043
(ClinicalTrials.gov)

November 2010

29/9/2010

Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency

A Randomized Double-blind Crossover Study to Assess the Safety and Pharmacokinetics of Two Different Doses of Weekly Intravenous Administration of Alpha1-Proteinase Inhibitor (Human) Prolastin®-C in Subjects With Alpha1-Antitrypsin Deficiency

Emphysema;Alpha 1-antitrypsin Deficiency (AATD)

Biological: Prolastin-C, 60 mg/kg;Biological: Prolastin-C, 120 mg/kg

Grifols Therapeutics Inc.

NULL

Completed

18 Years

70 Years

All

Phase 2

United States

EUCTR2007-004869-18-GB
(EUCTR)

03/07/2008

04/04/2008

Evaluation of the relative severity of pulmonary neutrophilic inflammation and therapeutic modification with IV Prolastin by means of 18 FDG PET/CT scanning in subjects with alpha1-antitrypsin deficiency. - ECLIPSE AATD

The medical condition is emphysema and will be investigated in subjects with alpha 1-antitrypsin deficiency.
MedDRA version: 9.1;Level: LLT;Classification code 10001811;Term: Alpha-1 proteinase inhibitor deficiency
MedDRA version: 9.1;Classification code 10014563;Term: Emphysema pulmonary
MedDRA version: 9.1;Classification code 10010952;Term: COPD
MedDRA version: 9.1;Classification code 10006458;Term: Bronchitis chronic

UHB NHS Foundation Trust

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United Kingdom

NCT00295061
(ClinicalTrials.gov)

May 2006

20/2/2006

Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults

Multi-center, Randomized, Double-blind, Crossover Trial to Evaluate the Pharmacokinetic Comparability of Alpha-1 MP to Prolastin in Subjects With Alpha1-antitrypsin Deficiency.

Alpha 1-Antitrypsin Deficiency

Drug: Alpha-1 MP;Drug: alpha-1 proteinase inhibitor (human)

Grifols Therapeutics Inc.

NULL

Completed

18 Years

N/A

All

Phase 3

United States

NCT00263887
(ClinicalTrials.gov)

December 2003

12/9/2005

Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)

Multi-center, Randomized Trial With I.V. Prolastin® to Evaluate Frequency of Exacerbations and Progression of Emphysema by Means of Multi-slice CT Scans in Patients With Congenital Alpha-1-antitrypsin Deficiency.

Alpha 1-Antitrypsin Deficiency

Drug: Alpha1-Proteinase Inhibitor (Human);Drug: Albumin (Human) 20%, United States Pharmacopeia (USP)

Grifols Therapeutics Inc.

NULL

Completed

18 Years

N/A

All

Phase 2

Denmark;Sweden;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
1	NCT02010411 (ClinicalTrials.gov)	June 2004	9/12/2013	Alpha1 Antitrypsin Aerosol Therapy in Cystic Fibrosis	Effects of Prolastin Aerosol Therapy on Bacterial Density in the Airways of Patients With Cystic Fibrosis	Cystic Fibrosis	Drug: Prolastin (drug)	Université de Sherbrooke	NULL	Terminated	14 Years	N/A	Both	17	Phase 2	Canada
2	NCT00486837 (ClinicalTrials.gov)	December 2003	13/6/2007	Deposition of Inhaled Prolastin in Cystic Fibrosis Patients	Multicenter, Randomized, Parallel Group Study to Investigate the Optimal Deposition Site for Inhaled Prolastin® in Patients With Cystic Fibrosis (CF)	Cystic Fibrosis	Drug: Alpha1-Proteinase Inhibitor (Human)	Grifols Therapeutics Inc.	NULL	Completed	8 Years	N/A	All	72	Phase 2	NULL