1 |
NCT02014415 |
Recruiting |
Alpha-1 Antitrypsin Deficiency Adult Liver Study |
- Alpha-1 Antitrypsin Deficiency
|
- Procedure: Liver Biopsy (Biopsy Group Only)
|
Observational |
|
- St. Louis University
- Alpha-1 Foundation
- University of California
- (and 3 more...)
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- The risk and rate of histologic liver injury progression, as measured by liver biopsy, over a 5-year period.
- Calculated Model for End-stage Liver Disease score (MELD)
- Liver synthetic dysfunction defined by international normalized ratio (INR) > 1.3 or serum albumin < 3.2 gm/dL
- (and 8 more...)
|
120 |
All |
18 Years and older (Adult, Senior) |
NCT02014415 |
A1F-SLU-7113 Alpha-1 Foundation 22609 |
|
December 2013 |
August 2020 |
August 2021 |
December 18, 2013 |
January 18, 2018 |
|
- University of California
San Diego, California, United States - Boston University School of Medicine
Boston, Massachusetts, United States - Saint Louis University
Saint Louis, Missouri, United States
|
2 |
NCT03039335 |
Recruiting |
The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency |
- Alpha 1-Antitrypsin Deficiency
|
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Retrospective
|
|
90 |
All |
18 Years and older (Adult, Senior) |
NCT03039335 |
16001 Pro00019289 |
|
December 15, 2016 |
December 2018 |
December 2019 |
February 1, 2017 |
February 1, 2017 |
|
- Biocerna LLC
Fulton, Maryland, United States
|
3 |
NCT02691611 |
Recruiting |
Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency |
- Alpha 1-Antitrypsin Deficiency
|
|
Observational |
|
- National Jewish Health
- Medical University of South Carolina
|
Other |
- Observational Model: Case-Control
- Time Perspective: Prospective
|
- Epigenetic signature of specific inflammation-associated histone modifications from CD14+ macrophages
- Epigenetically regulated genomic profile of AATD in AM
- Epigenetic mechanisms to regulate gene expression and cell function
|
19 |
All |
18 Years to 85 Years (Adult, Senior) |
NCT02691611 |
HS-2939 |
AATD_Epi |
December 2015 |
November 2018 |
November 2020 |
February 25, 2016 |
April 5, 2018 |
|
- National Jewish Health
Denver, Colorado, United States - Medical University of South Carolina
Charleston, South Carolina, United States
|
4 |
NCT02929940 |
Recruiting |
Liver Disease in Patients With alpha1-antitrypsin Deficiency |
- alpha1-antitrypsin Deficiency
|
|
Observational |
|
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Number of participants with Liver fibrosis, Hepatic decompensation, Liver cancer, Death or Liver transplantation
|
500 |
All |
18 Years to 99 Years (Adult, Senior) |
NCT02929940 |
Alpha1-Liver |
|
April 2015 |
April 2020 |
December 2020 |
October 11, 2016 |
May 31, 2017 |
|
- RWTH Aachen University
Aachen, North Rhine Westphalia, Germany
|
5 |
NCT02168686 |
Recruiting |
Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency |
- Alpha 1-Antitrypsin Deficiency
|
|
Interventional |
Phase 1 Phase 2 |
- Adverum Biotechnologies, Inc.
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Sequential Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number and proportion of subjects experiencing adverse events
- Number and proportion of subjects experiencing abnormal changes in clinical laboratory parameters
- Changes in plasma concentrations of M-specific A1AT up to 52 weeks
- Changes in total plasma concentrations of A1AT up to 52 weeks
|
20 |
All |
18 Years and older (Adult, Senior) |
NCT02168686 |
ADVM-043-01 |
ADVANCE |
November 28, 2017 |
December 2018 |
August 2019 |
June 20, 2014 |
March 1, 2018 |
|
- Ronald Reagan UCLA Medical Center
Santa Monica, California, United States - University of Florida
Gainesville, Florida, United States - University of Chicago Medical Center
Chicago, Illinois, United States - (and 2 more...)
|
6 |
NCT01379469 |
Recruiting |
Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency |
- Alpha-1-antitrypsin Deficiency
- Liver Cirrhosis
|
- Drug: Drug-Carbamazepine (Tegretol XR)
- Drug: Carbamazepine (Tegretol XR) Placebo
|
Interventional |
Phase 2 |
- Washington University School of Medicine
- Novartis
- National Institutes of Health (NIH)
- (and 2 more...)
|
Other / Industry / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- The primary outcome will be to determine the effect of Carbamazepine on hepatic ATZ load.
- For the secondary outcomes we will determine the effect of Carbamazepine treatment on hepatic fibrosis.
- For the secondary outcome we will determine if Carbamazepine treatment reduces the MELD score.
|
30 |
All |
14 Years to 80 Years (Child, Adult, Senior) |
NCT01379469 |
201510060-PRO09070279 1R21DK092567-01 |
CBZ |
January 2012 |
January 2019 |
January 2019 |
June 23, 2011 |
May 11, 2017 |
|
- Washington University in St. Louis School of Medicine
Saint Louis, Missouri, United States - Children's Hospital of Pittsburgh, UPMC
Pittsburgh, Pennsylvania, United States - University of Pittsburgh Medical Center, Presbyterian Hospital
Pittsburgh, Pennsylvania, United States
|
7 |
NCT02525861 |
Recruiting |
PH 3/4 GLASSIA SAFETY, IMMUNOGENICITY, AND BRONCHOALVEOLAR LAVAGE STUDY |
- Alpha1-antitrypsin Deficiency
|
|
Interventional |
Phase 3 |
- Baxalta now part of Shire
- Shire
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Prevention
|
- Number (proportion) of adverse events (AEs) considered potentially related to the presence of particle load in the GLASSIA solution
- Incidence of treatment-emergent adverse reactions (ARs) plus suspected ARs
- Number (proportion) of infusions that are discontinued, slowed, or interrupted due to an adverse event (AE)
- (and 6 more...)
|
36 |
All |
18 Years and older (Adult, Senior) |
NCT02525861 |
471101 |
|
March 8, 2016 |
May 15, 2019 |
May 15, 2019 |
August 18, 2015 |
February 23, 2018 |
|
- St. Joseph's Hospital and Medical Center
Phoenix, Arizona, United States - UCLA Medical Center
Los Angeles, California, United States - University of California Davis Health System
Sacramento, California, United States - (and 11 more...)
|
8 |
NCT00499941 |
Recruiting |
Alpha-1 Foundation Research Registry |
- Alpha 1-Antitrypsin Deficiency
|
|
Observational |
|
- Medical University of South Carolina
- Alpha-1 Foundation
|
Other |
- Observational Model: Other
- Time Perspective: Other
|
- To study the natural history of PiZZ, PiSZ, and PiMZ alpha-1 antitrypsin deficiency
|
10000 |
All |
Child, Adult, Senior |
NCT00499941 |
HR 9059 |
|
April 2000 |
June 2050 |
June 2050 |
July 12, 2007 |
January 23, 2018 |
|
- Medical University of South Carolina. Division of Pulmonary and Critical Care Medicine
Charleston, South Carolina, United States
|
9 |
NCT03114020 |
Recruiting |
Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency |
- Emphysema
- Alpha 1-Antitrypsin Deficiency
|
- Drug: Hyaluronic Acid Inhalation Solution
- Drug: Placebo Inhalation Solution
|
Interventional |
Phase 2 |
- Gerard Turino
- Turino, Gerard, M.D.
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine
- Assessment of pulmonary function tests
- Assessment of St. George Respiratory Questionnaire
|
40 |
All |
18 Years to 80 Years (Adult, Senior) |
NCT03114020 |
C100-008 |
|
March 22, 2017 |
July 2018 |
July 2018 |
April 14, 2017 |
November 29, 2017 |
|
- National Jewish Health
Denver, Colorado, United States - University of Miami Hospital
Miami, Florida, United States - Pulmonary Health Physicians, PC
Fayetteville, New York, United States - (and 2 more...)
|
10 |
NCT03362242 |
Recruiting |
Study of ARO-AAT in Normal Adult Volunteers |
- Alpha 1-Antitrypsin Deficiency
|
- Drug: ARO-AAT Injection
- Other: Sterile Normal Saline (0.9% NaCl)
|
Interventional |
Phase 1 |
- Arrowhead Pharmaceuticals
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Number of Participants With Adverse Events (AEs) Possibly or Probably Related to Treatment
- Pharmacokinetics (PK) of ARO-AAT: Maximum Observed Plasma Concentration (Cmax)
- PK of ARO-AAT: Time to Maximum Plasma Concentration (Tmax)
- (and 5 more...)
|
40 |
All |
18 Years to 55 Years (Adult) |
NCT03362242 |
AROAAT1001 |
|
March 12, 2018 |
November 30, 2018 |
February 28, 2019 |
December 5, 2017 |
March 9, 2018 |
|
- Auckland Clinical Studies Limited
Grafton, Auckland, New Zealand
|
11 |
NCT00500123 |
Recruiting |
Alpha-1 Coded Testing(ACT) Study |
- Alpha-1 Antitrypsin Deficiency
|
- Procedure: Alpha-1 Antitrypsin Genotype
|
Observational |
|
- Medical University of South Carolina
- University of Florida
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Structured questionnaire responses on the risks and benefits of testing.
|
50000 |
All |
Child, Adult, Senior |
NCT00500123 |
HR 9556 |
ACT |
January 2001 |
January 2050 |
January 2050 |
July 12, 2007 |
January 23, 2018 |
|
- Medical University of South Carolina. Division of Pulmonary and Critical Care Medicine
Charleston, South Carolina, United States
|
12 |
NCT01974830 |
Recruiting |
AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry |
- Alpha-1 Anti-trypsin Deficiency
|
|
Observational |
|
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change in Pulmonary Function
- Change in Quality of Life
- Pulmonary Infections and Exacerbation
|
400 |
All |
18 Years and older (Adult, Senior) |
NCT01974830 |
CT-10-04 |
AL1TER |
October 2013 |
October 2020 |
October 2025 |
November 4, 2013 |
April 25, 2017 |
|
- Coram Clinical Trials
Denver, Colorado, United States
|
13 |
NCT02502201 |
Recruiting |
Environment Effect on Six-Minute Walk Test Performance |
- Chronic Obstructive Pulmonary Disease
- Alpha-1 Antitrypsin Deficiency
|
- Other: six-minute walk study indoors
- Other: six-minute walk study outdoors
|
Interventional |
Not Applicable |
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- Change in the meters walked during the six-minute walk test between the two groups.
- Change in Borg Scale for shortness of breath between the two groups
- Change in Borg Scale for fatigue between the two groups
|
65 |
All |
18 Years and older (Adult, Senior) |
NCT02502201 |
IRB201500220 |
6MWTAATD |
October 2015 |
September 2018 |
September 2018 |
July 20, 2015 |
October 6, 2017 |
|
- Uf Ctsi Crc
Gainesville, Florida, United States
|
14 |
NCT01862211 |
Recruiting |
Polygen Defi-Alpha: Genetic Polymorphisms Study in Children With Alpha-1 Antitrypsin Deficiency, Included in the DEFI-ALPHA Cohort |
- Children With a Deficiency of Alpha-1 Antitrypsin
|
|
Interventional |
Not Applicable |
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- presence of mutations / polymorphisms in genes SERPINA1, Ermani and KPNB1 (with haplotype interpretation) in children with hepatic complications
- measurement and interpretation of serum IL-8
- Identification of modifier genes or mutations responsible of hepatic complications
|
420 |
All |
7 Years and older (Child, Adult, Senior) |
NCT01862211 |
2011.663 |
Polygen |
May 2013 |
December 2017 |
December 2018 |
May 24, 2013 |
February 9, 2017 |
|
- CHU d'Amiens - Hopital Nord
Amiens, France - CHU de BESANCON
Besancon, France - Hôpital Pellegrin
Bordeaux, France - (and 17 more...)
|
15 |
NCT02722304 |
Recruiting |
Stage 1 Study of ARALAST NP and GLASSIA in A1PI |
- Chronic Obstructive Pulmonary Disease
- Alpha1-antitrypsin Deficiency
|
- Biological: ARALAST NP 60 mg/kg
- Biological: ARALAST NP 120 mg/kg
- Biological: GLASSIA 60 mg/kg
- (and 2 more...)
|
Interventional |
Phase 4 |
- Baxalta now part of Shire
- Shire
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Rate of change in lung density for (1) all ARALAST NP recipients versus placebo recipients; and (2) all GLASSIA recipients versus placebo recipients
- Rate of change in lung density for each treatment group
- Mean steady state trough concentration of antigenic and functional Alpha1-Proteinase Inhibitor (A1PI) for ARALAST NP and GLASSIA at each dose level
- (and 9 more...)
|
138 |
All |
18 Years and older (Adult, Senior) |
NCT02722304 |
460503 |
|
June 22, 2016 |
July 30, 2021 |
July 30, 2021 |
March 30, 2016 |
January 29, 2018 |
|
- Phoenix Medical Research Institute, LLC
Peoria, Arizona, United States - Newport Native MD, Inc
Newport Beach, California, United States - Pulmonary Disease Specialists, P.A., / PDS Research
Kissimmee, Florida, United States - (and 15 more...)
|
16 |
NCT01983241 |
Recruiting |
Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD) |
- Pulmonary Emphysema in Alpha-1 PI Deficiency
|
- Biological: Alpha-1 MP
- Other: 0.9% Sodium Chloride for Injection, USP
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change from Baseline in Whole lung PD15 (15th percentile point)
- Adverse Events (AEs)
- Serious Adverse Events (SAEs)
- (and 3 more...)
|
339 |
All |
18 Years to 70 Years (Adult, Senior) |
NCT01983241 |
GTi1201 |
SPARTA |
November 2013 |
August 2021 |
August 2021 |
November 13, 2013 |
March 5, 2018 |
|
- St. Joseph's Hospital and Medical Center
Phoenix, Arizona, United States - University of Florida
Gainesville, Florida, United States - University of Miami Miller School of Medicine
Miami, Florida, United States - (and 41 more...)
|
17 |
NCT01851642 |
Recruiting |
Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs |
- Alpha-1 Antitrypsin Deficiency
- AAT Deficiency
- AATD
- Cystic Fibrosis (CF)
|
- Procedure: History and physical exam.
- Procedure: Blood draw.
- Procedure: Pulmonary function testing.
- Drug: Albuterol inhaler.
|
Observational |
|
|
Other |
- Observational Model: Case-Control
- Time Perspective: Prospective
|
- Evaluation of macrophage function.
- Comparison of the amount of alpha-1 antitrypsin in the blood.
- Comparison of the amount of an inflammatory marker in the blood, called C-reactive protein.
- Evaluation of lung function.
|
220 |
All |
18 Years and older (Adult, Senior) |
NCT01851642 |
IRB201501051 699 08-2007 |
|
September 2007 |
October 2018 |
December 2018 |
May 10, 2013 |
October 24, 2017 |
|
- Shands at the University of Florida
Gainesville, Florida, United States
|
18 |
NCT00571272 |
Recruiting |
Evaluating the Genetic Causes and Progression of Cholestatic Liver Diseases (LOGIC) |
- Liver Diseases
- Alagille Syndrome
- Alpha 1-Antitrypsin Deficiency
|
|
Observational |
|
- Arbor Research Collaborative for Health
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Demonstration of disease progression for each of the four cholestatic liver diseases of the study, including liver transplantation, death, growth failure, worsening liver function, and developmental complications of portal high blood pressure
- Jaundice (total serum bilirubin of greater than 2.0 mg/dl)
- Listing for liver transplantation
- (and 5 more...)
|
1150 |
All |
up to 25 Years (Child, Adult) |
NCT00571272 |
LOGIC - ChiLDReN Network U01DK103149 U01DK103140 U01DK103135 U01DK084575 U01DK084538 U01DK084536 U01DK062503 U01DK062500 U01DK062497 U01DK062481 U01DK062470 U01DK062466 U01DK062456 U01DK062453 U01DK062452 U01DK062445 U01DK062436 |
LOGIC |
November 2007 |
June 2019 |
June 2019 |
December 11, 2007 |
February 19, 2018 |
|
- Children's Hospital of Los Angeles
Los Angeles, California, United States - University of California at San Francisco (UCSF)
San Francisco, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 14 more...)
|
19 |
NCT03172455 |
Available |
Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT) |
- SR aGvHD
- Acute-graft-versus-host Disease
- Steroid Refractory Acute Graft Versus Host Disease
- (and 6 more...)
|
|
Expanded Access |
|
- Impatients N.V. trading as myTomorrows
- Kamada, Ltd.
|
Industry |
|
|
|
All |
Child, Adult, Senior |
NCT03172455 |
2017-AATGLAKAM-EU |
|
|
|
|
June 1, 2017 |
June 1, 2017 |
|
|
20 |
NCT03285100 |
Recruiting |
The Effects of Discontinuation of Vitamin K Antagonists on the Rate of Elastin Degradation |
- Emphysema or COPD
- Aneurysm
- Cystic Fibrosis
- Alpha 1-Antitrypsin Deficiency
|
- Diagnostic Test: Venipuncture
|
Observational |
|
- Canisius-Wilhelmina Hospital
- Maastricht University Medical Center
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Difference in elastin degradation rate
- Difference in vitamin K status
- Association between desmosine and dp-ucMGP
- Differences in desmosine and dp-ucMGP levels between different VKORC1 polymorphisms
|
30 |
All |
18 Years and older (Adult, Senior) |
NCT03285100 |
NL60536.091.17 |
|
October 31, 2017 |
October 1, 2018 |
October 1, 2018 |
September 15, 2017 |
January 25, 2018 |
|
- Canisius Wilhelmina Hospital
Nijmegen, Netherlands
|
21 |
NCT03455686 |
Not yet recruiting |
Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung Disease |
- Asthma
- Chronic Obstructive Pulmonary Disease
- Bronchiectasis
- (and 5 more...)
|
- Other: Hyperpolarized 129Xe MRI
|
Interventional |
Not Applicable |
- McMaster University
- St. Joseph's Healthcare Hamilton
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Ventilation Defect Percent (VDP)
- Apparent Diffusion Coefficient (ADC)
- Signal-to-noise ratio (SNR)
|
60 |
All |
18 Years to 85 Years (Adult, Senior) |
NCT03455686 |
FIRH_Xe0001 |
|
March 2018 |
March 2023 |
March 2023 |
March 6, 2018 |
March 6, 2018 |
|
- Firestone Institute for Respiratory Health, St. Joseph's Healthcare
Hamilton, Ontario, Canada
|
22 |
NCT02915614 |
Recruiting |
Effects of Pulmonary Rehabilitation on Skeletal Muscle in COPD Patients |
- Chronic Obstructive Pulmonary Disease
|
- Procedure: Pulmonary rehabilitation
|
Interventional |
Not Applicable |
- Klinikum Berchtesgadener Land der Schön-Kliniken
- CSL Behring
|
Other / Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Endurance time
- Myofibre type I Distribution
- Capillary to fibre ratio
- muscle strength
|
40 |
All |
25 Years to 75 Years (Adult, Senior) |
NCT02915614 |
AAT 2016 |
|
March 2, 2017 |
December 2019 |
June 2020 |
September 27, 2016 |
March 14, 2018 |
|
- Schoen Klinik Berchtesgadener Land
Schönau Am Königssee, Germany
|