Clinical Studies

Examples of Cystic Fibrosis Clinical Studies Recently Conducted at UAB

L= lead site, P = participating site, M = multicenter site, S = single site

Years

Study Title

Status

Sponsor

Role

Comments

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

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

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

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

2007-2010

Detection of airway CFTR expression, localization, and activity in CF patients

Enrollment complete

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

Inspire

P,M

5 complete

2008-2012

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

2008-2010

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- 2013

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)

Complete

CF Foundation

S

42 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

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- 2012

An Open-Label, Rollover Study to Evaluate the Long-Term Safety and Efficacy of VX-770 in Subjects with Cystic Fibrosis 770-105

Complete

Vertex Pharma

P,M

5 enrolled

2011- 2013

B aby Observational and Nutritional Study (BONUS)

Complete

CF Foundation

P,M

9 enrolled

2011-2013

A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis

Complete

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-tooST aph Aureus Resistance – treat or observe)

Ongoing

CF Foundation

P,M

5 enrolled

2011- 2013

Systemic CFTR Dysfunction in COPD (STACI-Rowe)

Complete

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

Longitudinal Evaluation of TGF-B1 as a Biomarker in Cystic Fibrosis Lung Disease

Ongoing

Departmental Funds

S

currently enrolling

2011- Closed

VX-770 Expanded Access Program (EAP)

Complete

Vertex Pharma

P, M

2 enrolled

2010-Ongoing

Development of new procedures for treating CF sinusitis

Ongoing

Department of Surgery

S

50 enrolled

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

2014- Ongoing

661-103 : A Phase 2, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and

Pharmacodynamics of VX-661 in Combination With Ivacaftor for 12 Weeks in Subjects With Cystic Fibrosis, Homozygous for the

F508del-CFTR Mutation

Ongoing

Vertex Pharma

P, M

2 Enrolled

2014- Ongoing

STOP: Pilot Observational Study to Determine Feasibility of a Standardized Treatment of Pulmonary Exacerbation in Patients with Cystic Fibrosis

Ongoing

CF Foundation

P, M

12 Enrolled

2014

Goal Extension: G 551D Observational Study – Expanded to Additional Genotypes and Extended for Long Term Follow-up ( GOAL-e²)

Ongoing

CF Foundation

L, M

6 Enrolled

2013-2014

809-103: A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With

Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

Finalizing

Vertex Pharma

P, M

9 Enrolled

2014-Ongoing

809-105: A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With

Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

Complete

Vertex Pharma

P, M

9 Enrolled

2013-Ongoing

770-109: A Phase 3, 2-Arm, Roll-Over Study to Evaluate the Long-term Safety and Pharmacodynamics of Ivacaftor Treatment in Pediatric

Subjects With Cystic Fibrosis and a CFTR Gating Mutation

Ongoing

Vertex Pharma

P, M

3 Enrolled

2013-2014

CAT: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Aztreonam for Inhalation Solution (AZLI) in a

Continuous Alternating Therapy (CAT) Regimen of Inhaled Antibiotics for the Treatment of Chronic Pulmonary Pseudomonas

aeruginosa Infection in Subjects with Cystic Fibrosis

Complete

Gilead Sciences

P, M

3 Enrolled

2013- Ongoing

EICE: Early Intervention in cystic Fibrosis Exacerbation (eICE)

Ongoing

CF Foundation

P, M

13 Enrolled

2012-2014

A Three Week Dose Escalation, Randomized, Double-Blind, Placebo-Controlled Trial, to Assess the Safety and Tolerability of 100 mg or 200 mg of Inhaled Alpha-1 HC, Once a Day in Subjects with Cystic Fibrosis

Complete

Griffols Pharma

P,M

2 Enrolled

2013- Ongoing

112: A Phase 3, Two-Arm, Rollover Study to Evaluate the Safety of Long-Term Ivacaftor Treatment in Subjects 6 Years of Age and Older

with Cystic Fibrosis and a Non-G551D CFTR Mutation

Ongoing

Vertex Pharma

P, M

4 Enrolled

2012-Ongoing

N30: A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study of N6022 to Evaluate Safety and

Pharmacokinetics in Subjects with Cystic Fibrosis Homozygous for the F508del-CFTR Mutation

Ongoing

N30 Pharmaceuticals

P, M

2 Enrolled

Pending Approval

Bronchoalveolar Lavage Fluid (BALF) Specimen Bank and Lower Airway Microbiome Analysis in Children and Adults with and without Cystic Fibrosis (CF).

-

CF Foundation

P, M

Pending approval

Pending Approval

Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Assess the Efficacy, Safety and Tolerability of Arikace™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa (TR02-109/110)

-

Transave

P, M

Pending Approval

Pending Approval

ICM002: Centralized Intestinal Current Measurement (ICM) testing, and comparison of suction and forceps-based biopsy performance.

-

CF Foundation

L, M

Pending Approval