A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT)



Status:Completed
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:12 - Any
Updated:8/29/2018
Start Date:June 2015
End Date:July 27, 2018

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identify and validate biomarkers that might reflect partial restoration of CFTR function and
can be used to monitor disease progression, and ii) evaluate the mechanistic effects of CFTR
modulators and other relevant therapies in individuals with CF

Cystic fibrosis (CF) is a genetic disorder caused by mutations in the gene encoding the
cystic fibrosis transmembrane conductance regulator (CFTR) protein. Over 1,900 mutations,
categorized into five genotypic or functional classes are implicated in causing CF. Severity
of disease varies widely in CF based on CFTR-dependent and independent factors. Progressive
obstructive lung disease is the main determinant of morbidity and mortality in CF; therefore
it is critical to identify biomarker profiles that reflect and predict this phenotypic
variability, and understand their relationship to residual CFTR activity. Emerging CFTR
modulator therapies that directly target defective CFTR are being evaluated in pivotal
clinical trials and may become available in the next few years. It is not known how partial
restoration of CFTR function might impact CF disease progression and disease-related
biomarkers. Thus there is urgent need to i) identify and validate biomarkers that might
reflect partial restoration of CFTR function and can be used to monitor disease progression,
and ii) evaluate the mechanistic effects of CFTR modulators and other relevanttherapies in
individuals with CF

Inclusion Criteria Part A COHORT 1:

- 1. Written informed consent (and assent when applicable) obtained from subject or
subject's legal representative.

2. Be willing and able to adhere to the study visit schedul and other protocol
requirements 3. Male or female ≥ 12 years of age at Visit 1. 4. Have a body mass index
(BMI) of:

- For subjects ≥ 18 years of age: ≤ 30 kg/m2

- For subjects 12 - 17 years of age: ≤ 95th percentile 5. Be a non-smoker for ≥ 1
year at screening and have ≤ 10 pack-year history of smoking.

6. To participate in the optional DNA banking component of this study, subject
must have signed the informed consent indicating willingness to participate in
the genomic component of the study. Refusal to give consent for this component
does not exclude a subject from participation in the study.

Inclusion Cohorts 2-3

1. Written informed consent (and assent when applicable) obtained from subject or
subject's legal representative.

2. Male or female ≥ 12 years of age at Visit 1.

3. Documentation of a CF diagnosis as evidenced by one or more clinical features
consistent with the CF phenotype and the following criteria: Cohort 2: (Partial
Function CFTR CF)

- Two mutations in the CFTR gene:

- At least one allele must be a Class IV or V mutation

- The second allele can be within any CFTR mutation class.

- Pancreatic sufficient (based on the absence of daily PERT use)

- At least one historic sweat chloride ≥60 mEq/L by quantitative pilocarpine
iontophoresis test (QPIT) OR sweat chloride results ≥ 40, but < 60mEQ/L upon
permission of the PROSPECT Investigator-Sponsors.

Cohort 3: (Absent Function CF)

• Two class I or II CFTR mutations

4. Enrolled in the Cystic Fibrosis Foundation Patient Registry. Patients may enroll in
the Registry at Visit 1 if not previously enrolled.

5. Clinically stable with no signifIcant changes in health status within 2 weeks prior to
Visit 1.

6. Be a non-smoker for ≥ 1 year at screening and have ≤ 10 pack-year history of smoking.

7. To participate in the optional DNA banking component of this study, subject must have
signed the informed consent indicating willingness to participate in the genomic
component of the study. Refusal to give consent for this component does not exclude a
subject from participation in the study

Part B Inclusion

1. Written informed consent (and assent when applicable) obtained from subject or
subject's legal representative.

2. Physician decision to treat with ivacaftor/lumicaftor.

3. Completion of at least Visit 1 and Visit 2 of Part A

Exclusion Criteria PART A COHORT 1

1. Presence of a condition or abnormality that in the opinion of the Investigator would
compromise the safety of the patient or the quality of the data.

2. A history of any clinically significant medical illness or medical disorder that
requires ongoing systemic medical therapy, including (but not limited to)
cardiovascular disease, neuromuscular disease, hematological disease including
bleeding disorders, chronic respiratory disease (including persistent asthma), hepatic
or gastrointestinal (GI) disease, neurological disease, neoplastic disease, renal
diseases, or endocrine disorders including diabetes.

3. Acute illness requiring any new prescription or over-the-counter treatment within 14
days prior to Visit 1.

4. Major or traumatic surgery within 12 weeks prior to Visit 1.

5. For females of child-bearing potential: a positive pregnancy test at Visit 1.

6. Initiation of any new chronic therapy within 28 days prior to Visit 1.

7. Use of an investigational agent within 28 days prior to Visit 1.

Exclusion Part A COHORTS 2-3

1. Presence of a condition or abnormality that in the opinion of the Investigator would
compromise the safety of the patient or the quality of the data.

2. Initiation of newly prescribed antibiotics [oral, intravenous (IV), and/or inhaled]
for acute respiratory symptoms within 2 weeks of Visit 1.

3. Major or traumatic surgery within 12 weeks prior to Visit 1.

4. For females of child-bearing potential: a positive pregnancy test at Visit 1.

5. Initiation of any new chronic therapy (e.g., ibuprofen Pulmozyme®, hypertonic saline,
azithromycin, TOBI®, Cayston®) within 4 weeks prior to Visit 1.

6. Use of an investigational agent within 28 days prior to Visit 1.

7. Use of oral corticosteroids in doses exceeding 10 mg prednisone/day or 20 mg
prednisone/every other day (subjects on oral steroids will be on stable doses for > 12
weeks prior to visit 1).

8. Active treatment for nontuberculous mycobacterial (NTM) infection, consisting of ≥ two
antibiotics (oral, IV, and/or inhaled).

9. Use of CFTR modulator therapy such as ivacaftor (Kalydeco®) within 28 days prior to
Visit 1.

10. History of lung or liver transplantation, or listing for organ transplantation.

Exclusion PART B

1. Presence of a condition or abnormality that in the opinion of the Investigator would
compromise the safety of the patient or the quality of the data.

2. Initiation of newly prescribed antibiotics [oral, intravenous (IV), and/or inhaled]
for acute respiratory symptoms within 2 weeks of Visit 4.

3. Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline,
azithromycin, TOBI®, Cayston®) within 4 weeks prior to Visit 4.

4. Use of an investigational agent within 28 days prior to Visit 4.

5. Use of oral corticosteroids in doses exceeding 10 mg prednisone/day or 20 mg
prednisone/every other day (subjects on oral steroids will be on stable doses for > 12
weeks prior to Visit 4).

6. Active treatment for nontuberculous mycobacterial (NTM) infection, consisting of ≥ two
antibiotics (oral, IV, and/or inhaled).

7. Use of CFTR modulator therapy such as ivacaftor (Kalydeco®) within 28 days prior to
Visit 4.
We found this trial at
38
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2401 Gillham Rd
Kansas City, Missouri 64108
(816) 234-3000
Phone: 816-701-1339
Children's Mercy Hospital Children's Mercy Hospitals and Clinics continues redefining pediatric medicine throughout the Midwest...
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1 Perkins Square
Akron, Ohio 44308
(330) 543-1000
Phone: 330-543-3089
Akron Children's Hospital From humble beginnings as a day nursery in 1890, Akron Children
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1720 2nd Ave S
Birmingham, Alabama 35233
(205) 934-4011 
Phone: 205-639-5970
University of Alabama at Birmingham The University of Alabama at Birmingham (UAB) traces its roots...
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
Phone: 617-726-3719
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Chapel Hill, North Carolina 27599
(919) 962-2211
Phone: 919-966-9198
University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Phone: 513-803-4325
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Phone: 323-361-3255
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Minneapolis, Minnesota 55455
(612) 625-5000
Phone: 612-626-7425
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Phone: 267-426-5135
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Phone: 412-692-7060
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Aurora, Colorado 80045
Phone: 720-777-6162
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1101 East 33rd Street
Baltimore, Maryland 21287
Phone: 443-287-8983
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Boston, Massachusetts 02115
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Buffalo, New York 14222
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171 Ashley Avenue
Charleston, South Carolina 29425
843-792-1414
Phone: 843-792-4349
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Phone: 312-227-6861
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Cleveland, Ohio 44106
Phone: 216-844-5733
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Columbus, Ohio 43205
Phone: 614-722-4752
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1400 Jackson St
Denver, Colorado 80206
(303) 388-4461
Phone: 303-398-1265
National Jewish Health National Jewish Health is known worldwide for treatment of patients with respiratory,...
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3901 Beaubien St
Detroit, Michigan 48201
(313) 745-5437
Phone: 313-745-4737
Children's Hospital of Michigan Since 1886, the Children's Hospital of Michigan has been dedicated to...
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Grand Rapids, Michigan 49503
Phone: 616-391-6148
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Hershey, Pennsylvania 17033
Phone: 717-531-5646
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Houston, Texas
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Kansas City, Kansas 66160
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1 Medical Center Dr
Lebanon, New Hampshire 03756
 (603) 650-5000
Phone: 603-650-8178
Dartmouth Hitchcock Medical Center Dartmouth-Hitchcock is a national leader in patient-centered health care and building...
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Milwaukee, Wisconsin 53226
Phone: 414-955-7030
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9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
(414) 266-2000
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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Nashville, Tennessee 37232
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630 W 168th St
New York, New York
212-305-2862
Phone: 212-305-0290
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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Palo Alto, California 94394
Phone: 650-736-0388
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Portland, Oregon 97227
Phone: 503-494-7807
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Saint Louis, Missouri 63104
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Saint Louis, Missouri 63110
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Salt Lake City, Utah 84143
Phone: 801-587-7458
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Phone: 206-987-3921
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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1959 NE Pacific St
Seattle, Washington 98195
(206) 598-3300
University of Washington Medical Center University of Washington Medical Center is one of the nation's...
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Valhalla, New York 10595
Phone: 914-594-2348
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