Phase
Condition
Pompe Disease
Treatment
Miglustat
Cipaglucosidase Alfa
Clinical Study ID
Ages < 17 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Male or female subjects (ERT-naïve [have never received a dose of rhGAA] orERT-experienced [have received rhGAA every 2 weeks for at least 6 months immediatelybefore enrollment, and if ERT dosage has been modified, must have been on themodified dosage for at least 3 months before enrollment]) diagnosed with LOPD whoare aged 12 to <18 years at screening (Cohort 1 only) or aged 0 months to < 12 yearsat screening (Cohort 2 only)
Subject weighs ≤ 115 kg. (Cohort 1 Only)
Subject must have a diagnosis of LOPD based on documentation as defined in studyprotocol
If of reproductive potential and if sexually active, female and male subjects agreeto use a highly effective method of contraception throughout the duration of thestudy and for up to 90 days after their last dose of Cipaglucosidase Alfa/Miglustat
Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value forhealthy Adolescents at screening (Cohort 1 only)
Subject (aged 12 to <18 years; Cohort 1) performs one 6-Minute Walk Test (6MWT) (≥ 75 meters) at screening that is valid, as determined by the clinical evaluator, orsubject (aged ≥ 5 to < 12 years; Cohort 2) performs one 6MWT (≥ 40 meters) atscreening that is valid, as determined by the clinical evaluator
Exclusion
Exclusion Criteria:
Subject has received any investigational/experimental drug, oral anabolic steroid orderivative, biologic, or device within 30 days or 5 half-lives of the therapy ortreatment, whichever is longer, before screening
Subject has received treatment with prohibited medications within 30 days ofscreening
Subject has received any gene therapy at any time
Subject has any intercurrent illness or condition at screening or baseline that maypreclude the subject from fulfilling the protocol requirements or suggests to theinvestigator and/or the medical monitor that the potential subject may have anunacceptable risk by participating in this study
Subject has a hypersensitivity to any of the excipients in ATB200, approved rhGAA,or AT2221
Female subject is pregnant or breast-feeding at screening
Subject requires the use of ventilation support for > 6 hours per day while awake
Subject has evidence of moderate to severe hypertrophic cardiomyopathy aligning withclassic IOPD
In the opinion of the investigator, the parent or legally authorized representativeis unlikely or unable to comply with the study requirements
Subject has any prior history of illness or condition known to affect motorfunction, such as, but not limited to, Guillain-Barre syndrome, cerebral palsy, etc
Subject who is diagnosed with Pompe disease via newborn screening and isasymptomatic (ie, showing no signs and symptoms of Pompe disease (Cohort 2 Only)
Study Design
Connect with a study center
Women's and Children's Hospital
North Adelaide, South Australia 5006
AustraliaActive - Recruiting
University of Calgary
Calgary, Alberta T3B 6A8
CanadaCompleted
SphinCS GmbH Clinical Science for LSD
Hochheim, Hessen 65239
GermanyActive - Recruiting
San Gerardo Hospital
Monza, 20900
ItalyActive - Recruiting
Izumi City General Hospital
Osaka, Izumi-Shi 594-0073
JapanActive - Recruiting
Gunma University Hospital
Gunma, 371-8511
JapanActive - Recruiting
Tohoku University Hospital
Miyagi, 980-8574
JapanActive - Recruiting
Tokyo Women's Medical University
Tokyo, 162-8666
JapanActive - Recruiting
National Taiwan University Hospital
Taipei, 100
TaiwanSite Not Available
Neuromuscular Research Center
Phoenix, Arizona 85028
United StatesSite Not Available
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United StatesSite Not Available
UCSF Benioff Children's Hospital
Oakland, California 94609
United StatesSite Not Available
University of Florida Clinical Research Center
Gainesville, Florida 32610
United StatesActive - Recruiting
Wolfson Children's Hospital
Jacksonville, Florida 32207
United StatesActive - Recruiting
Woodruff Memorial Research Building
Atlanta, Georgia 30322
United StatesSite Not Available
Infusion Associates
Grand Rapids, Michigan 49525
United StatesSite Not Available
Mayo Clinic
Rochester, Minnesota 55905
United StatesSite Not Available
St. Louis Children's Hospital
Saint Louis, Missouri 63110
United StatesActive - Recruiting
Duke University Medical Center
Durham, North Carolina 27710
United StatesActive - Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United StatesActive - Recruiting
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania 15224
United StatesActive - Recruiting
University of Utah, Clinical and Translational Sciences Institute
Salt Lake City, Utah 84108
United StatesActive - Recruiting
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia 22030
United StatesActive - Recruiting
Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.