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_ gender	Target_ size	Phase	Countries
1	EUCTR2019-004881-16-IE (EUCTR)	29/10/2020	09/07/2020	Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype	A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects	Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype MedDRA version: 23.0;Level: PT;Classification code 10083869;Term: Alpha-1 antitrypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864	Vertex Pharmaceuticals Incorporated	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	40	Phase 2	United States;Canada;Ireland;Netherlands;Germany;United Kingdom;Sweden
2	EUCTR2019-004881-16-DE (EUCTR)	17/09/2020	10/07/2020	Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype	A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects	Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype MedDRA version: 23.0;Level: PT;Classification code 10083869;Term: Alpha-1 antitrypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX864 Other descriptive name: VX-864	Vertex Pharmaceuticals Incorporated	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	40	Phase 2	United States;Canada;Ireland;Netherlands;Germany;United Kingdom;Sweden
3	EUCTR2019-004881-16-GB (EUCTR)	14/09/2020	21/07/2020	Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype	A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects - VX19-864-101 Efficacy and Safety of VX-864	Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype MedDRA version: 23.0;Level: PT;Classification code 10083869;Term: Alpha-1 antitrypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: VX864 Product Code: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 Other descriptive name: VX-864	Vertex Pharmaceuticals Incorporated	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	40	Phase 2	United States;Canada;Ireland;Netherlands;Germany;United Kingdom;Sweden
4	EUCTR2019-004881-16-SE (EUCTR)	11/09/2020	14/07/2020	Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype	A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects	Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype MedDRA version: 23.0;Level: PT;Classification code 10083869;Term: Alpha-1 antitrypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX-864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX-864 Other descriptive name: VX-864 Product Name: VX864 Product Code: VX864 INN or Proposed INN: VX-864 Other descriptive name: VX-864	Vertex Pharmaceuticals Incorporated	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	40	Phase 2	United States;Canada;Ireland;Netherlands;Germany;United Kingdom;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

EUCTR2019-004881-16-IE
(EUCTR)

29/10/2020

09/07/2020

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects

Alpha 1 antitrypsin deficiency in Subjects With the PiZZ Genotype
MedDRA version: 23.0;Level: PT;Classification code 10083869;Term: Alpha-1 antitrypsin deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Product Name: VX864
Product Code: VX864
INN or Proposed INN: VX864
Other descriptive name: VX-864
Product Name: VX864
Product Code: VX864
INN or Proposed INN: VX864
Other descriptive name: VX-864
Product Name: VX864
Product Code: VX864
INN or Proposed INN: VX864
Other descriptive name: VX-864

Vertex Pharmaceuticals Incorporated

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;Canada;Ireland;Netherlands;Germany;United Kingdom;Sweden

EUCTR2019-004881-16-DE
(EUCTR)

17/09/2020

10/07/2020

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects

Product Name: VX864
Product Code: VX864
INN or Proposed INN: VX864
Other descriptive name: VX-864
Product Name: VX864
Product Code: VX864
INN or Proposed INN: VX864
Other descriptive name: VX-864

Vertex Pharmaceuticals Incorporated

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;Canada;Ireland;Netherlands;Germany;United Kingdom;Sweden

EUCTR2019-004881-16-GB
(EUCTR)

14/09/2020

21/07/2020

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects - VX19-864-101 Efficacy and Safety of VX-864

Product Name: VX864
Product Code: VX864
Other descriptive name: VX-864
Product Name: VX864
Product Code: VX864
Other descriptive name: VX-864
Product Name: VX864
Product Code: VX864
Other descriptive name: VX-864

Vertex Pharmaceuticals Incorporated

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;Canada;Ireland;Netherlands;Germany;United Kingdom;Sweden

EUCTR2019-004881-16-SE
(EUCTR)

11/09/2020

14/07/2020

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects

Product Name: VX864
Product Code: VX864
INN or Proposed INN: VX-864
Other descriptive name: VX-864
Product Name: VX864
Product Code: VX864
INN or Proposed INN: VX-864
Other descriptive name: VX-864
Product Name: VX864
Product Code: VX864
INN or Proposed INN: VX-864
Other descriptive name: VX-864

Vertex Pharmaceuticals Incorporated

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;Canada;Ireland;Netherlands;Germany;United Kingdom;Sweden