Clinical Trial Finder
Observational Enrolling
Study to evaluate the effects of CFTR modulators in infants and young children (BEGIN Part A) (BEGIN-OB-19 Part A)
This two-part observational study will look at the effects of CFTR modulators on growth in young children with CF. These drugs are intended to help CFTR protein function closer to normal.
Part A of this study will enroll young children with CF who are not yet taking CFTR modulators. Researchers will collect information on growth factors in these young children by measuring weight and height for up to 3 years. Researchers will also collect information on gastrointestinal (GI) symptoms and lung function. Children who enroll in Part A of this study may also choose to enroll in Part B of this study once they begin taking CFTR modulator treatment with elexacaftor/tezacaftor/ivacaftor to observe how that drug impacts their growth and health.
This study will require physical exams, lung function tests, and/or other additional procedures.
Email a research coordinator to express interest in participating in this study.
Eligibility
See other primary eligibility criteria for more information.
-
Age:
Less than 5 Years -
Mutation(s):
See Other Primary Eligibility Criteria -
FEV1% Predicted:
No FEV1 Limit
For a more comprehensive list of eligibility criteria and details on this study, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Part A of this study is for children with CF who are less than 5 years old and not on ivacaftor or the elexacaftor/tezacaftor/ivacaftor triple-combination therapy. Additionally, participants in Part A of this study cannot have mutations that are currently treatable with ivacaftor.
Study Design
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Study Type: ?more info
Observational -
Randomized Study: ?more info
No -
Placebo Controlled: ?more info
No -
Length of Participation:
3 years -
Number of Study Visits:
6
Additional Information
-
Phase: ?more info
Not Applicable -
Study Sponsor: ?more info
Ramsey, Bonnie -
Study Drugs:
N/A
Study Sites
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Enrolling
Alabama
The Children's Hospital Alabama, University of Alabama at Birmingham, Birmingham, AL 35233
Contact
Heather Hathorne
Phone: +1 (205) 638-9568
Email: hhathorne@peds.uab.edu
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Enrolling
Colorado
Children's Hospital Colorado, Aurora, CO 80045
Contact
Meg Anthony
Phone: +1 (720) 777-2945
Email: Meg.Anthony@childrenscolorado.org
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Enrolling Soon
Florida
University of Miami, Miami, FL 33136
Contact
Alejandra Weisman
Phone: +1 (305) 243-1425
Email: a.weisman1@miami.edu
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Enrolling
Florida
The Nemours Children's Clinic - Orlando, Orlando, FL 32827
Contact
Amanda Darling
Phone: +1 (407) 650-7966
Email: amanda.darling@nemours.org
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Enrolling
Florida
Nemours Children's Clinic, Jacksonville, FL 32207
Contact
Jennifer (Jenn) Gafford
Phone: +1 (904) 697-3600
Email: Jennifer.gafford@nemours.org
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Enrolling Soon
Indiana
Riley Hospital for Children, Indianapolis, IN 46202
Contact
Lisa Bendy
Phone: +1 (317) 278-7152
Email: lbendy@iu.edu
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Enrolling
Iowa
University of Iowa, Iowa City, IA 52242
Contact
Mary Teresi
Phone: +1 (319) 384-7546
Email: mary-teresi@uiowa.edu
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Enrolling Soon
Kansas
University of Kansas Medical Center, Kansas City, KS 66160
Contact
Lawrence Scott
Phone: +1 (913) 588-4020
Email: lscott2@kumc.edu
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Enrolling Soon
Massachusetts
Boston Children's Hospital, Boston, MA 02115
Contact
Robert Fowler
Phone: +1 (617) 355-1834
Email: Robert.fowler@childrens.harvard.edu
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Enrolling Soon
Michigan
University of Michigan, Michigan Medicine, Ann Arbor, MI 48109
Contact
Dawn Kruse
Phone: +1 (734) 615-3266
Email: dmkruse@med.umich.edu
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Enrolling Soon
Michigan
Helen DeVos Children's Hospital, Grand Rapids , MI 49503
Contact
Cynthia Gile
Phone: +1 (616) 391-6148
Email: Cynthia.Gile@spectrumhealth.org
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Enrolling Soon
Minnesota
The Minnesota Cystic Fibrosis Center, Minneapolis, MN 55455
Contact
Participant Contact University of Minnesota
Phone: +1
Email: cftrials@umn.edu
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Enrolling Soon
Minnesota
Children’s Hospitals and Clinics of Minnesota, Minneapolis, MN 55404
Contact
Camerone Bey
Phone: +1 (612) 813-7628
Email: Camerone.bey@childrensmn.org
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Enrolling Soon
Missouri
St. Louis Children's Hospital, St. Louis, MO 63110
Contact
Tina Hicks
Phone: +1 (314) 747-2707
Email: hickst@wustl.edu
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Enrolling Soon
Missouri
Children's Mercy Kansas City, KCMO, MO 64108
Contact
Candy Schmoll
Phone: +1 (816) 302-3538
Email: cschmoll@cmh.edu
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Enrolling
New York
The Cystic Fibrosis Center of Western New York, Buffalo, NY 14203
Contact
Christine Roach
Phone: +1 (716) 323-0072
Email: croach@upa.chob.edu
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Enrolling Soon
New York
SUNY Upstate Medical University, Syracuse, NY 13210
Contact
Mary Forell
Phone: +1 (315) 464-9209
Email: forellm@upstate.edu
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Enrolling
New York
New York Medical College at Westchester Medical Center, Valhalla, NY 10595
Contact
Zachary Messer
Phone: +1 (914) 594-2352
Email: Zachary_Messer@nymc.edu
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Enrolling Soon
New York
Children's Hospital of New York, New York, NY 10032
Contact
Carmen Liriano
Phone: +1 (212) 305-6265
Email: crl58@cumc.columbia.edu
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Enrolling Soon
Ohio
Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229
Contact
Kelly Thornton
Phone: +1 (513) 636-0604
Email: Kelly.Thornton@cchmc.org
-
Enrolling
Ohio
Nationwide Children's Hospital, Columbus, OH 43205
Contact
Diana Gilmore
Phone: +1 (614) 722-4752
Email: diana.gilmore@nationwidechildrens.org
-
Enrolling
Oklahoma
Oklahoma Cystic Fibrosis Center, OKC, OK 73104
Contact
Participant Contact Oklahoma Cystic Fibrosis Center
Phone: +1
Email: cfresearchteam@ouhsc.edu
-
Enrolling
Oregon
Oregon Health Sciences University, Portland, OR 97239
Contact
Brendan Klein
Phone: +1 (503) 418-8108
Email: kleinb@ohsu.edu
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Enrolling Soon
Pennsylvania
Children's Hospital of Philadelphia, Philadelphia, PA 19104
Contact
Erin Donnelly
Phone: +1 (267) 426-9626
Email: Donnellye4@email.chop.edu
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Enrolling Soon
Pennsylvania
Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA 15224
Contact
Elizabeth Hartigan
Phone: +1 (412) 692-7060
Email: elizabeth.hartigan@chp.edu
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Enrolling Soon
Pennsylvania
Hershey Medical Center Pennsylvania State University, Hershey, PA 17033
Contact
Diane Kitch
Phone: +1 (717) 531-5646
Email: dkitch@pennstatehealth.psu.edu
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Enrolling Soon
Texas
University of Texas Southwestern / Children’s Health, Dallas, TX 75207
Contact
Daniyal Kamal
Phone: +1 (214) 456-5489
Email: Daniyal.Kamal@childrens.com
-
Enrolling
Texas
Cook Children's Medical Center, Fort Worth, TX 76104
Contact
Heather Urbanek
Phone: +1 (682) 885-1244
Email: Heather.Urbanek@cookchildrens.org
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Enrolling Soon
Texas
Baylor College of Medicine, Houston, TX 77030
Contact
Katie Howe
Phone: +1 (832) 822-4252
Email: klhowe@texaschildrens.org
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Enrolling Soon
Utah
Primary Children's Cystic Fibrosis Center, Salt Lake City, UT 84113
Contact
Jane Vroom
Phone: +1 (801) 587-7458
Email: jane.vroom@hsc.utah.edu
-
Enrolling
Vermont
Vermont Children's Hospital, Burlington, VT 05401
Contact
Julie Sweet
Phone: +1 (802) 847-7958
Email: julie.sweet@uvmhealth.org
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Enrolling
Virginia
University of Virginia, Charlottesville, VA 22903
Contact
Christie Aderholt
Phone: +1 (434) 297-7773
Email: cla6e@virginia.edu
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Enrolling Soon
Washington
Seattle Children's Hospital, Seattle, WA 98105
Contact
Sharon McNamara
Phone: +1 (206) 987-3921
Email: sharon.mcnamara@seattlechildrens.org
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Enrolling Soon
Wisconsin
University of Wisconsin, Madison, WI 53792
Contact
Melanie Nelson
Phone: +1 (608) 265-4617
Email: mnelson@pediatrics.wisc.edu
Email a research coordinator to express interest in participating in this study.
Eligibility
See other primary eligibility criteria for more information.
-
Age:
Less than 5 Years -
Mutation(s):
See Other Primary Eligibility Criteria -
FEV1% Predicted:
No FEV1 Limit
For a more comprehensive list of eligibility criteria and details on this study, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Part A of this study is for children with CF who are less than 5 years old and not on ivacaftor or the elexacaftor/tezacaftor/ivacaftor triple-combination therapy. Additionally, participants in Part A of this study cannot have mutations that are currently treatable with ivacaftor.
Related Topics
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Questions to Ask When Considering a Specific Trial
Clinical trials that test potential drugs and therapies in people with cystic fibrosis are a major part of CF research. They take place at Cystic Fibrosis Foundation-accredited care centers all over the United States and enroll people with CF of all ages.
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Patient Safety Is a Priority
Nothing is more important than patient safety in developing new treatments for cystic fibrosis. The U.S. government requires that all clinical trials are supervised for safety, and the Cystic Fibrosis Foundation adds additional measures to protect study volunteers.
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Trikafta™
The following is a collection of information and news about Trikafta™, the triple-combination modulator therapy that was approved by the U.S. Food and Drug Administration on Oct. 21, 2019.
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