A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05431127` - High Dose Inspiratory Muscle Training in LOPD	N/A
Terminated	`NCT03347253` - STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
Terminated	`NCT01924845` - BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)	Phase 3

See also

Status

Clinical Trial

Phase

Recruiting

NCT05431127 - High Dose Inspiratory Muscle Training in LOPD

N/A

Terminated

NCT03347253 - STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

Terminated

NCT01924845 - BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Phase 3

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT02191917
Study type	Observational
Source	BioMarin Pharmaceutical
Contact
Status	Terminated
Phase
Start date	October 21, 2014
Completion date	June 22, 2016

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms