|
2005-2009
|
|
Phase IB Study of SLITTM Amikacin (TR02) in CF Patients Infected with P. aeruginosa
|
|
Complete
|
Transave
|
L
|
3 enrolled and completed
|
|
2004- 2011
|
|
Phase 2 study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated CF
|
|
Complete
|
PTC Therapeutics
|
L
|
5 enrolled, 5 completed, 7 screened,
4 sites
|
|
2003-2005
|
|
A Multicenter, Double-Blind, Placebo-Controlled, Phase II Study of Aerosolized tgAAVCF for the Treatment of CF
|
|
Complete
|
Targeted Genetics Corporation
|
P, M
|
10 enrolled and completed, exceeded enrollment goals
|
|
2004-2005
|
|
A Phase 2, Randomized, Double Blind, Parallel Dose Ranging Study of Oral TheraCLECTM-Total In CF Subjects with Exocrine Pancreatic Insufficiency
|
|
Complete
|
Altus Biologics, Inc
|
P, M
|
4 enrolled and completed
|
|
2004- 2006
|
|
Assessment of Induced Sputum as a Tool to Evaluate Anti Inflammatory Agents in Patients with CF
|
|
Complete
|
CFF Therapeutics, Inc.
|
P, M
|
7 enrolled and completed
|
|
2004- Ongoing
|
|
Effectiveness and safety of Intermittent Antimicrobial Therapy for the Treatment of New Onset Pseudomonas aeruginosa Airway Infection in Young Patients with CF
|
|
Observational
Ongoing. Clinical closed.
|
CFF
|
P, M
|
39 enrolled in the observational EPIC trial, 6 enrolled in the clinical EPIC trial
|
|
|
|
|
|
|
|
|
|
2004-2005
|
|
Long-term Safety and Efficacy of Azithromycin in CF: Observational Study of AZ0001 Participants
|
|
Complete
|
CF Services, Inc.
|
P, M
|
8 enrolled and completed
|
|
1995-Ongoing
|
|
Measurement of Nasal Potential Difference in Patients With and Without CF
|
|
Ongoing
|
NIH
|
S
|
172 enrolled and completed
|
|
1999-2010
|
|
Regulation of CFTR by Adenosine, A2 receptors, and PLA2
|
|
Complete
|
NIH
|
S
|
70 enrolled and completed
|
|
2002-2010
|
|
Matrix Metalloproteinases in CF Lung Disease
|
|
Complete
|
Pediatric Departmental Funds
|
S
|
142 enrolled and completed
|
|
2003-Ongoing
|
|
Genetic Modifiers of CF Liver Disease
|
|
Ongoing
|
NIH
|
P, M
|
5 enrolled and completed
|
|
2004-2010
|
|
Effect of hypertonic aerosols on motility and flagellar function of P. aeruginosa
|
|
Complete
|
Institutional Funds
|
S
|
20 enrolled and completed
|
|
2004-Ongoing
|
|
Genetic Modifiers of CF Lung Disease
|
|
Ongoing
|
CFF and UNC
|
P, M
|
29 enrolled and completed
|
|
2006-2009
|
|
Activation of non-CFTR chloride secretion by zinc
|
|
Complete
|
Institutional Funds
|
S
|
3 enrolled and completed
|
|
2006-2007
|
|
A Phase 3, Double-Blind, Multicenter, Multinational, Randomized, Placebo-Controlled Trial Evaluating Aztreonam Lysinate for Inhalation in CF Patients with Pulmonary P. aeruginosa (AIR CF1)
|
|
Complete
|
Corus Pharma
|
P, M
|
3 enrolled, 5 screened
|
|
|
|
|
|
|
|
|
|
2006
|
|
A Randomized, Double-Blind, Placebo-Controlled Two-Treatment, Crossover Study to Evaluate the Safety and Efficacy of Eurand Pancreatic Enzyme Product (PEP) in Patients with CF and Exocrine Pancreatic Insufficiency
|
|
Completed
|
Eurand S.P.A.
|
P, M
|
2 enrolled and completed in EUR-1008.
2 enrolled and completed in EUR-1009.
|
|
2006- 2008
|
|
A Multi-Center, Double Blind Placebo Controlled Randomized Efficacy and Safety Study of Denufosol Tetrasodium Inhalation Solution in Patients with Mild CF Lung Disease
|
|
Complete
|
Inspire
|
P,M
|
9 enrolled, 12 screened. 8 completed
|
|
|
|
|
|
|
|
|
|
2007-
Ongoing
|
|
A Phase 3, Open-label, Follow-On Study of Multiple Courses of Aztreonam Lysinate for Inhalation (AI) in Cystic Fibrosis Patients (AIR-CF3)
|
|
Ongoing
|
Corus
|
P. M
|
2 enrolled
|
|
|
|
|
|
|
|
|
|
2007-2009
|
|
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study of VX-770 to Evaluate Safety, Pharmacokinetics, and Biomarkers of CFTR Activity in CF Subjects with Genotype G551D
|
|
Complete
|
Vertex
|
L, M
|
5 complete
|
|
2007-2010
|
|
Detection of airway CFTR expression, localization, and activity in CF patients
|
|
Enrollment complete, In data analysis only
|
CFF
|
S
|
39 complete
|
|
2007-Ongoing
|
|
Detection of CFTR in nasal and rectal tissue from human subjects.
|
|
Ongoing
|
CFFT/Vertex Pharmaceuticals
|
S
|
111 (rectal tissues)
3 (nasal tissues)
|
|
2008-Ongoing
|
|
Antimicrobial Resistance in sputum obtained from patients with CF.
|
|
Ongoing
|
CFFT
|
P,M
|
4 enrolled
|
|
2008-2010
|
|
A Phase 3, International Multi-Center, Randomized, Double Blind Placebo Controlled Parallel Group, Efficacy and Safety Study of Denufosol Tetrasodium Inhalation Solution in Patients with Cystic Fibrosis Lung Disease and FEV1 > or equal to 75% predicted.
|
|
Enrollment complete, In Data Analysis Only
|
Inspire
|
P,M
|
5 complete
|
|
|
|
|
|
|
|
|
|
2008-2009
|
|
A Phase I/II Randomized, Double-Blind, Placebo-Controlled, Single-Dose, Dose Escalation Study of KB001 in Cystic Fibrosis Patients infected with Pseudomonas aeruginosa
|
|
Complete
|
KaloBios
|
P,M
|
3 screen failures
1 withdrew
0 complete
|
|
|
|
|
|
|
|
|
|
2008-Closed
|
|
Expanded Access Program for Aztreonam Lysine for Inhalation in Patients with CF and Pseudomonas aeruginosa Airway Infection Who Have Limited Treatment Options and are at Risk for Disease Progression
|
|
Complete
|
Gilead Sciences
|
P,M
|
screened 12
enrolled 10
|
|
|
|
|
|
|
|
|
|
2008-2009
|
|
Detection of CFTR with Quercetin in Nasal Potential Differences from Human Subjects (Center for CFTR Detection)
|
|
Closed to Enrollment
|
CF Foundation
|
S
|
11 complete
|
|
|
|
|
|
|
|
|
|
2008-2010
|
|
Genetic modifiers of mild CF-related phenotypes such as CBAVD
|
|
Complete
|
CF Foundation & Departmental Funds
|
L,M
|
131 enrolled
|
|
2008- Ongoing
|
|
Airway Tissue Procurement for the Study of Lung Disease (UAB CF Research and Translation Core Center)
|
|
Ongoing
|
NIH
|
S
|
100 complete
|
|
2008-Onging
|
|
The role of HMGB1 as a biomarker of CF disease severity
|
|
Ongoing
|
NIH
|
S
|
13 enrolled
|
|
2009- Ongoing
|
|
ICM001: Detection of CFTR Activity in Rectal Tissues from Human Subjects (Therapeutic Development Center - UAB Center for CFTR Detection)
|
|
Closed Data analysis ongoing
|
CF Foundation
|
L, M
|
19 normal
3 CF complete
|
|
2009- Ongoing
|
|
Implications of Cross-Protease Dysregulation in Cystic Fibrosis: A randomized, placebo controlled pilot trial of doxycycline to reduce sputum MMP-9 activity in adult CF patients hospitalized for pulmonary exacerbations (DOXY)
|
|
Ongoing
|
CF Foundation
|
S
|
30 enrolled
|
|
2009-Ongoing
|
|
Characterization of MRSA infection/colonization in CF
|
|
Ongoing
|
CF Foundation
|
P,M
|
AIM 2 closed at 59
AIM 3: enrolling
|
|
2009-2011
|
|
A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
|
|
Enrollment Complete
|
PTC Therapeutics
|
P,M
|
5 enrolled
2 withdrawn
2 screen Fails
3 completed
|
|
2009-2011
|
|
Infant Study of Inhaled Saline in Cystic Fibrosis (ISIS002)
|
|
Closed to Enrollment
|
CF Foundation
|
P,M
|
7 completed
|
|
2009-2011
|
|
A Multi-Center IIB, Randomized, Placebo-Controlled, Double-Blind Study of the Effects of N-Acetylcysteine on Redox Changes and Lung Inflammation in Cystic Fibrosis Patients.
|
|
Closed to Enrollment
|
CF Foundation
|
P,M
|
7 complete
|
|
2009-2010
|
|
A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics, and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the ΔF508-CFTR Gene Mutation (809-101)
|
|
Complete
|
Vertex Pharma
|
L,M
|
7 complete
|
|
2010-2011
|
|
Open Label Extension of Study 08-110. A Multicenter Study of Denufosol Tetrasodium Inhalation Solution in Patients with Cystic Fibrosis Lung Disease
|
|
Complete- terminated by Sponsor
|
Inspire Pharma
|
P,M
|
3 enrolled
|
|
2010-2011
|
|
Nasal Potential Studies Utilizing CFTR Modulators (UAB Center for Clinical and Translational Science) Quercetin- Rowe
|
|
Closed to Enrollment
|
NIH
|
L, P, 3 sites
|
16 complete
|
|
2010-2011
|
|
A Phase 2, Multicenter, Double-Blinded, Placebo-Controlled, Multiple-Dose Study to Evaluate Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of VX-809 Alone and in Combination with VX-770 in Subjects with Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation (809-102)
|
|
Complete
|
Vertex Pharma
|
P,M
|
6 complete
|
|
|
|
|
|
|
|
|
|
2010-2011
|
|
A Phase 3, Randomized, Double-Blind,Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects with Cystic Fibrosis and the G551D Mutation Vertex Study Number: VX08-770-102
|
|
Complete
|
Vertex Pharma
|
P,M
|
4 complete
|
|
|
|
|
|
|
|
|
|
2010-2011
|
|
A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Pharmacokinetics, Efficacy and Safety of VX-770 in Subjects Aged 6 to 11 years with Cystic Fibrosis and the G551D Mutation 770-103
|
|
Complete
|
Vertex Pharma
|
P,M
|
1 complete
|
|
|
|
|
|
|
|
|
|
2010-2011
|
|
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of VX‑770 in Subjects Aged 12 Years and Older With Cystic Fibrosis who are Homozygous for the F508del-CFTR Mutation 770-104
|
|
Complete
|
Vertex Pharma
|
P,M
|
1 complete
|
|
|
|
|
|
|
|
|
|
2010-2011
|
|
A Randomized, Double Blind, Parallel Group, Placebo Controlled 28 Day Study to Investigate the Safety, Tolerability and Pharmacodynamics of SB-656933 in Patients with Cystic Fibrosis (GLAXO)
|
|
Complete
|
Glaxo Pharma
|
P, M
|
3 completed
|
|
|
|
|
|
|
|
|
|
2011- Ongoing
|
|
An Open-Label, Rollover Study to Evaluate the Long-Term Safety and Efficacy of VX-770 in Subjects with Cystic Fibrosis 770-105
|
|
Ongoing
|
Vertex Pharma
|
P,M
|
5 enrolled
|
|
2011- Ongoing
|
|
Baby Observational and Nutritional Study (BONUS)
|
|
Document Preparation
|
CF Foundation
|
P,M
|
goal 5
|
|
2011-Ongoing
|
|
A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis
|
|
Ongoing
|
PTC Therapeutics
|
P,M
|
3 enrolled,
2 withdrawn
1 on Treatment
|
|
2011-Ongoing
|
|
Early MRSA therapy in CF - culture based vs. observant therapy (treat or observe) (STAR-too - STaph Aureus Resistance - treat or observe)
|
|
Pending Contract
|
CF Foundation
|
P,M
|
pending contract
|
|
|
|
|
|
|
|
|
|
2011- Ongoing
|
|
Systemic CFTR Dysfunction in COPD (STACI-Rowe)
|
|
Ongoing
|
UAB Lung Health Center
|
S
|
53 enrolled
|
|
2009- Ongoing
|
|
TGF-β1 activity in CF lung disease: Measurement of BALF TGF-β1 activity in CF and non-CF pediatric patients
|
|
Ongoing
|
Department Funds
|
S
|
69 enrolled
|
|
2011- Ongoing
|
|
Bronchoalveolar Lavage Fluid (BALF) Specimen Bank and Lower Airway Microbiome Analysis in Children and Adults with and without Cystic Fibrosis (CF).
|
|
Pending Approval
|
CF Foundation
|
P, M
|
pending approval
|
|
|
|
|
|
|
|
|
|
2011- Ongoing
|
|
Longitudinal Evaluation of TGF-B1 as a Biomarker in Cystic Fibrosis Lung Disease
|
|
Ongoing
|
Departmental Funds
|
S
|
currently enrolling
|
|
2011- Ongoing
|
|
Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Assess the Efficacy, Safety and Tolerability of ArikaceTM in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa (TR02-109/110)
|
|
Document prep
|
Transave
|
P, M
|
pending approval
|
|
|
|
|
|
|
|
|
|
2011- Ongoing
|
|
ICM002: Centralized Intestinal Current Measurement (ICM) testing, and comparison of suction and forceps-based biopsy performance.
|
|
Pending approval
|
CF Foundation
|
L, M
|
pending approval
|
|
2011- Ongoing
|
|
VX-770 Expanded Access Program (EAP)
|
|
Recruiting
|
Vertex Pharma
|
P, M
|
recruiting
|
|
2010-Ongoing
|
|
Development of new procedures for treating CF sinusitis
|
|
Ongoing
|
Department of Surgery
|
S
|
50 enrolled
|
|
2011-Ongoing
|
|
Probiotic treatment of CF infants identified by newborn screening
|
|
Pending approval
|
Maternal and Child Health/
Pulmonary Care Center
|
S
|
pending approval
goal 30
|
|
2009-2010
|
|
Vitamin D interventions to enhance CF nutrition
|
|
Complete
|
Maternal and Child Health/
Pulmonary Care Center
|
S
|
47 enrolled
|
|
2007-Ongoing
|
|
Salivary proteomic abnormalities resulting from absent CFTR
|
|
Ongoing
|
Dixon Foundation
|
S
|
68 enrolled
|
|
2009-Ongoing
|
|
Home pulmonary function monitoring in CF
|
|
Ongoing
|
NIH
|
S
|
39 enrolled
|
|
2010-Ongoing
|
|
In vivo testing of acquired forms of CFTR deficiency
|
|
Ongoing
|
NIH
|
S
|
25 enrolled
|
Current Research