High Protein & Exercise Therapy Plus Nocturnal Enteral Feeding in

Status Withdrawn

Clinical Trial Summary

The research protocol will be submitted for approval to the institutional review board of Columbia University Medical Center. An attempt will be made to recruit at least 6 juvenile patients between the ages of 8 and 17, preferably who are still ambulatory.

Subjects meeting all eligibility criteria will undergo a full history and physical examination, including details of age of onset of symptoms, distribution and severity of muscle weakness, muscle function, pulmonary function, and nutritional status. Subjects will undergo an electrocardiogram (ECG), spirometry, muscule strength evaluation, exercise capacity, functional muscle tests, laboratory tests, and muscle biopsy. Quality of life will be assessed via SF 36 questionnaire. Functional ability and level of handicap will be assessed by Rotterdam handicap scale. Written informed consent will be obtained from all subjects.

All patients, who will have received enzyme replacement therapy (ERT) for at least 2 years, will be evaluated prior to institution of high protein nutrition and exercise therapy plus nocturnal enteral feeding (HPET + NEF)(baseline), then again at 3 months, 6 months and 12 months into treatment. The following parameters will be evaluated-

- Skeletal Muscle Function

- Biochemical parameters from collected blood sample Muscle Biopsy will be obtained at baseline and at 12 months. Biopsy specimens, obtained from thigh muscle at baseline and a repeat biopsy of the corresponding area of the other leg at 12 months, will be analyzed as follows:.

- Histology and electron microscopy

- Autophagic and lysosomal function evaluation

- Body composition Body mass index (BMI), body composition, lean body mass, and fat mass will be measured at each visit by bioelectric impedance analysis using BI-101Q RJL Systems, software 3.1b

Clinical Trial Description

n/a

Study Design

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT01656590
Study type	Interventional
Source	Columbia University
Contact
Status	Withdrawn
Phase	Phase 2
Start date	October 2012
Completion date	August 2013

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT00231400` - Pompe Disease Registry Protocol
Recruiting	`NCT04910776` - Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa	Phase 3
Not yet recruiting	`NCT05017402` - Higher Dose of Alglucosidase Alpha for Pompe Disease
Completed	`NCT00701129` - An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease	Phase 4
Active, not recruiting	`NCT04093349` - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)	Phase 1/Phase 2
Completed	`NCT02363153` - Diet and Exercise in Pompe Disease	N/A
Completed	`NCT00074932` - Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease	N/A
Completed	`NCT00025896` - Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease	Phase 2
Completed	`NCT00001331` - Genetic and Family Studies of Inherited Muscle Diseases	N/A
Completed	`NCT00077662` - A Prospective, Observational Study in Patients With Late-Onset Pompe Disease	N/A
Recruiting	`NCT05951790` - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease	N/A
Completed	`NCT00125879` - Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602	Phase 2/Phase 3
Recruiting	`NCT04848779` - A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Active, not recruiting	`NCT05164055` - Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)	Phase 4
Recruiting	`NCT02761421` - Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT	N/A
Active, not recruiting	`NCT02635269` - Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy	N/A
Completed	`NCT00051935` - A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II	Phase 2
Completed	`NCT00053573` - rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)	Phase 1/Phase 2
Completed	`NCT02801539` - Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)	N/A
Completed	`NCT03687333` - Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment	Phase 4

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.

High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms