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

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ClinicalTrials.gov Identifier: NCT01656590
Recruitment Status : Withdrawn (PI left the institution)
First Posted : August 3, 2012
Last Update Posted : April 13, 2015
Sponsor:
Information provided by (Responsible Party):
Columbia University

Tracking Information
First Submitted Date  ICMJE August 1, 2012
First Posted Date  ICMJE August 3, 2012
Last Update Posted Date April 13, 2015
Study Start Date  ICMJE October 2012
Actual Primary Completion Date August 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 2, 2012)
Change in muscle function [ Time Frame: Baseline, 12 months ]
Gross muscle function will be measured by the Walton Scale, the Timed Muscle Function Test and the Six-Minute Walk. Muscle strength will be measured by hand held dynamometer. Functional ability will be assessed by Rotterdam 9-item Handicap Scale.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01656590 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 2, 2012)
Change in pulmonary function (Vital capacity, forced expiration volume) [ Time Frame: Baseline, 12 months ]
The Pulmonary Function Test will be used to measure vital capacity (VC) and forced expiration volume (FEV1) to assess pulmonary function in sitting and supine positions.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease
Official Title  ICMJE High Protein Nutrition and Exercise Therapy (HPET) Plus Nocturnal Enteral Feeding (NEF) in Juvenile-onset Pompe Disease.
Brief 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
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Glycogen Storage Disease Type II
Intervention  ICMJE Other: High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding
  1. High Protein and Moderate Carbohydrate Nutrition designed by our nutritionist for every patient based on his/her nutrition requirements, age & gender
  2. 500 cc Formula - Nutren Replete with Fiber overnight [8 hours] via gastrostomy tube
  3. Conditioning Exercise once daily
Study Arms  ICMJE High Protein and Exercise therapy along-with Nocturnal Enteral
Intervention: Other: High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: April 9, 2015)
0
Original Estimated Enrollment  ICMJE
 (submitted: August 2, 2012)
6
Actual Study Completion Date  ICMJE August 2013
Actual Primary Completion Date August 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Male or female, 8 to 17 years of age.
  2. Diagnosis of Pompe disease; either by enzyme deficiency of muscle biopsy specimen or skin fibroblast culture, or homozygous or compound heterozygous for GAA mutation.
  3. Muscle Function < grade 7 on Walton Scale.
  4. Women of reproductive age (> 15 years) agree to use reliable methods of contraception during the study, if sexually active
  5. Subject or legal representative is willing and able to provide written informed consent.

Exclusion Criteria:

  1. Any intercurrent condition that may preclude accurate interpretation of study data
  2. Obstructive pulmonary disease
  3. Invasive ventilatory support
  4. Noninvasive ventilatory support while awake and in an upright position
  5. History of QTc prolongation > 450 msec for males and > 470 msec for females
  6. Life expectancy < 1 year
  7. History of allergy, sensitivity or any serious adverse reaction to rhGAA drug
  8. Pregnancy
  9. Current or recent drug or alcohol abuse.
  10. Treatment with another investigational drug within 60 days of study start
  11. Use of prohibited medication < 3 months prior to randomization
  12. Otherwise unsuitable for the study in the opinion of investigator
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 8 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01656590
Other Study ID Numbers  ICMJE AAAJ7305
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Columbia University
Study Sponsor  ICMJE Columbia University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Alfred E Slonim, MD Columbia University
PRS Account Columbia University
Verification Date April 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP