DDrare: Database of Drug Development for Rare Diseases

Searched query = "Alpha-1-antitrypsin deficiency", "AATD"
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 = (4S)-4-[4-cyano-2-(methylsulfonyl)phenyl]-3,6-dimethyl-2-oxo-1-[3-(trifluoromethyl)phenyl]-1,2,3,4-tetrahydropyrimidine-5-carbonitrile; 4-PBA; ADS-001; ALN-61444; ALN-77412; ALN-AAT; ALN-AAT02; ALPHA-1-ANTITRYPSIN; ALVELESTAT; API; API-AAT; API-AAT, AD00370, ARC-AAT, API drug Substance; ARC-AAT; ARC-AAT Injection; ARO-AAT; ARO-AAT Injection; Acetaminophen; Aerosolized human Alpha-1 Antitrypsin; Albumin (Human) 20%, United States Pharmacopeia (USP); Albuterol; Albuterol inhaler.; Alpha 1-Antitrypsin; Alpha 1-Proteinase Inhibitor; Alpha 1-antitrypsin; Alpha 1-proteinase inhibitor (human); Alpha-1 Antitrypsin (AAT); Alpha-1 Antitrypsin (human); Alpha-1 MP; Alpha-1 proteinase inhibitor (human); Alpha-1-antitrypsin; Alpha1-Proteinase Inhibitor; Alpha1-Proteinase Inhibitor (Human); Alpha1-proteinase Inhibitor; Alvelestat; Alvelestat (MPH966); Alvelestat oral tablet - dose 1; Alvelestat oral tablet - dose 2; Alvelestat tosylate; Aspirin; Carbamazepine; DCR-A1AT; Device: Abdominal ultrasound; Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor; Dose of 60 mg/kg alpha1-proteinase inhibitor; Drug-Carbamazepine (Tegretol XR); Fentanyl; Glassia; Hyaluronic Acid Inhalation Solution; Hyaluronic acid; INBRX-101/rhAAT-Fc; Kamada -AAT for Inhalation or inhaled AAT (AAT); Kamada-AAT for Inhalation, 160mg; Kamada-AAT for Inhalation, 80mg; Kamada-AAT for inhalation; Kamada-API; Lidocaine; Lorazepam; MPH966; Midazolam; None; None assigned; OctaAlpha1; Ondansetron; Other: Liver questionnaire; Other: Sterile Normal Saline (0.9% NaCl); Other: Withdrawal from alpha1 antitrypsin replacement therapy; Oxycodone; Oxycodone/Acetaminophen; PBA; PHP-303; Procedure: Blood draw; Procedure: Blood draw.; Procedure: History and physical; Procedure: History and physical exam.; Procedure: Intravenous catheter; Procedure: Liver Biopsy; Procedure: Pulmonary function testing.; Prolastin; Prolastin-C; Prolastin-C, 120 mg/kg; Prolastin-C, 60 mg/kg; RAAV1-CB-hAAT; RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR EXPRESSING THE HUMAN ALPHA-1-ANTITRYPSIN GENE; RO3300074; Retinoic Acid Receptor gamma Agonist; Retinoic acid; VX-814; VX-864; VX864

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	NCT02796937 (ClinicalTrials.gov)	July 2016	3/6/2016	Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency	An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency	Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency	Biological: Alpha-1 MP	Grifols Therapeutics LLC	NULL	Enrolling by invitation	20 Years	72 Years	All	250	Phase 3	United States;Australia;Canada;Denmark;Estonia;New Zealand;Poland;Sweden
3	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
4	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
5	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
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	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
7	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
8	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

NCT02796937
(ClinicalTrials.gov)

July 2016

3/6/2016

Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency

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

Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency

Biological: Alpha-1 MP

Grifols Therapeutics LLC

NULL

Enrolling by invitation

20 Years

72 Years

All

250

Phase 3

United States;Australia;Canada;Denmark;Estonia;New Zealand;Poland;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

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

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

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

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