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Results for “LOPD”

22 trials

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Showing 20 of 22 results

Not applicableUnknownNCT05272969
What this trial is testing

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more
LMU Klinikum 95
Large-scale testing (Phase 3)Active Not RecruitingNCT03911505
What this trial is testing

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 21
Not applicableLooking for participantsNCT05989620
What this trial is testing

Long-Term Development of Muscular Dystrophy Outcome Assessments

Who this might be right for
LGMD1BLGMD1CLGMD1D+26 more
Virginia Commonwealth University 1,000
Testing effectiveness (Phase 2)WithdrawnNCT04094948
What this trial is testing

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Duke University
Testing effectiveness (Phase 2)Looking for participantsNCT07282847
What this trial is testing

Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)

Who this might be right for
Pompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD
AskBio Inc 12
Early research (Phase 1)Not Yet RecruitingNCT07354724
What this trial is testing

Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Who this might be right for
Late-onset Pompe Disease
Denali Therapeutics Inc. 32
Testing effectiveness (Phase 2)Active Not RecruitingNCT04093349
What this trial is testing

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+4 more
Spark Therapeutics, Inc. 4
Not applicableUnknownNCT05951790
What this trial is testing

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease (Late-onset)
Fondazione Don Carlo Gnocchi Onlus 20
Not applicableStudy completedNCT03255213
What this trial is testing

Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)

Who this might be right for
Glycogen Storage Disease
Duke University 5
Early research (Phase 1)Study completedNCT01859624
What this trial is testing

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Duke University 8
Testing effectiveness (Phase 2)Active Not RecruitingNCT04174105
What this trial is testing

Gene Transfer Study in Patients With Late Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Astellas Gene Therapies 11
Not applicableEnded earlyNCT02191917
What this trial is testing

Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

Who this might be right for
Late-onset Pompe Disease
BioMarin Pharmaceutical 8
Very early researchNot Yet RecruitingNCT06178432
What this trial is testing

Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease (Late-onset)
Huashan Hospital 6
Not applicableStudy completedNCT02765828
What this trial is testing

Identification of Tongue Involvement in Late-Onset Pompe Disease

Who this might be right for
MyopathyNeuropathyGlycogen Storage Disease Type II (Late-onset Pompe Disease)
Duke University 73
Early research (Phase 1)Study completedNCT02240407
What this trial is testing

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 2
Not applicableStudy completedNCT03893240
What this trial is testing

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+3 more
Spark Therapeutics, Inc. 61
Large-scale testing (Phase 3)Study completedNCT04138277
What this trial is testing

Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 119
Not applicableEnded earlyNCT03347253
What this trial is testing

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

Who this might be right for
Late-onset Pompe Disease
Amicus Therapeutics 12
Testing effectiveness (Phase 2)Looking for participantsNCT07123155
What this trial is testing

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 45
Testing effectiveness (Phase 2)Study completedNCT01885936
What this trial is testing

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

Who this might be right for
Pompe Disease
Duke University 16
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