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Trial record 44 of 80 for:    "phenylketonuria"

Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria (PKU)

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ClinicalTrials.gov Identifier: NCT01428258
Recruitment Status : Completed
First Posted : September 2, 2011
Results First Posted : March 3, 2017
Last Update Posted : November 17, 2017
Sponsor:
Collaborator:
Boston Children’s Hospital
Information provided by (Responsible Party):
University of Wisconsin, Madison

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Phenylketonuria
Interventions: Other: GMP Diet/GMP Medical Foods
Other: AA Diet/AA Medical Foods

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
GMP Diet-AA Diet

In this randomized crossover study, half of subjects will be assigned to an arm that consists of the the GMP diet followed by the AA diet referred to as the Glycomacropeptide (GMP) diet given first intervention.

Glycomacropeptide (GMP) diet given first: The intervention compares a new low-phenylalanine (phe) dietary therapy for PKU, a diet containing foods and beverages made from GMP using Glytactin provided by Cambrooke Foods LLC, with the usual amino acid (AA) low-phe dietary therapy. PKU subjects in the GMP Diet-AA Diet Arm will follow the GMP diet that will replace all of the dietary protein equivalents provided by AA formula with foods and beverages made from GMP for 3 weeks followed by a 3 wk wash out period. They will then follow the usual AA diet for 3 weeks. Each dietary treatment period will maintain constant intake of total protein and phe and last for 3 weeks in subjects living at home.

AA Diet - GMP Diet

In this randomized crossover study, half of subjects will be assigned to an arm that consists of the AA diet followed by the GMP diet referred to as the Amino Acid (AA) Diet given first intervention.

Amino Acid (AA) Diet Given First: The intervention compares the usual amino acid (AA) low-phenylalanine (phe) dietary therapy with a new dietary therapy for PKU, a low-phe diet containing foods and beverages made from glycomacropeptide (GMP). PKU subjects in the AA Diet-GMP Diet Arm will follow their usual AA diet for 3 weeks followed by a 3 wk wash out period. They will then replace all of the protein equivalents provided in their diet by AA formula with foods and beverages made from GMP using Glytactin as provided by Cambrooke Foods, LLC. Each dietary treatment period will maintain constant intake of total protein and phe and last for 3 weeks in subjects living at home.


Participant Flow for 2 periods

Period 1:   Intervention 1
    GMP Diet-AA Diet   AA Diet - GMP Diet
STARTED   17   15 
COMPLETED   15   15 
NOT COMPLETED   2   0 
Withdrawal by Subject                2                0 

Period 2:   Intervention 2
    GMP Diet-AA Diet   AA Diet - GMP Diet
STARTED   15   15 
COMPLETED   15   15 
NOT COMPLETED   0   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
GMP Diet-AA Diet

In this randomized crossover study, half of subjects will be assigned to an arm that consists of the the GMP diet followed by the AA diet referred to as the Glycomacropeptide (GMP) diet given first intervention.

Glycomacropeptide (GMP) diet given first: The intervention compares a new low-phenylalanine (phe) dietary therapy for PKU, a diet containing foods and beverages made from GMP using Glytactin provided by Cambrooke Foods LLC, with the usual amino acid (AA) low-phe dietary therapy. PKU subjects in the GMP Diet-AA Diet Arm will follow the GMP diet that will replace all of the dietary protein equivalents provided by AA formula with foods and beverages made from GMP for 3 weeks followed by a 3 wk wash out period. They will then follow the usual AA diet for 3 weeks. Each dietary treatment period will maintain constant intake of total protein and phe and last for 3 weeks in subjects living at home.

AA Diet - GMP Diet

In this randomized crossover study, half of subjects will be assigned to an arm that consists of the AA diet followed by the GMP diet referred to as the Amino Acid (AA) Diet given first intervention.

Amino Acid (AA) Diet Given First: The intervention compares the usual amino acid (AA) low-phenylalanine (phe) dietary therapy with a new dietary therapy for PKU, a low-phe diet containing foods and beverages made from glycomacropeptide (GMP). PKU subjects in the AA Diet-GMP Diet Arm will follow their usual AA diet for 3 weeks followed by a 3 wk wash out period. They will then replace all of the protein equivalents provided in their diet by AA formula with foods and beverages made from GMP using Glytactin as provided by Cambrooke Foods, LLC. Each dietary treatment period will maintain constant intake of total protein and phe and last for 3 weeks in subjects living at home.

Total Total of all reporting groups

Baseline Measures
   GMP Diet-AA Diet   AA Diet - GMP Diet   Total 
Overall Participants Analyzed 
[Units: Participants]
 17   15   32 
Age 
[Units: Participants]
Count of Participants
     
<=18 years      3  17.6%      2  13.3%      5  15.6% 
Between 18 and 65 years      14  82.4%      13  86.7%      27  84.4% 
>=65 years      0   0.0%      0   0.0%      0   0.0% 
Age 
[Units: Years]
Mean (Full Range)
 28 
 (15 to 56) 
 28 
 (15 to 56) 
 28 
 (15 to 56) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      14  82.4%      5  33.3%      19  59.4% 
Male      3  17.6%      10  66.7%      13  40.6% 
Region of Enrollment 
[Units: Participants]
Count of Participants
     
United States   17   15   32 


  Outcome Measures

1.  Primary:   Change in the Plasma Phenylalanine Concentration of PKU Subjects Fed the Glycomacropeptide Diet Compared With the Change When Fed the Amino Acid Diet   [ Time Frame: baseline to day 22 on each diet ]

2.  Secondary:   Dietary Compliance   [ Time Frame: 3 week dietary treatment ]

3.  Secondary:   Executive Function Assessed by BRIEF   [ Time Frame: day 22 of each dietary treatment ]

4.  Secondary:   Vitamin D (25-OH) Plasma Concentration at Day 22   [ Time Frame: day 22 of each dietary treatment ]

5.  Secondary:   Comparison of Phe Concentrations in Plasma With Concentrations in Dried Blood Spots   [ Time Frame: 4 times total, 2 per treatment ]

6.  Secondary:   Bone-specific Alkaline Phosphatase (BSAP) Plasma Concentration at Day 22   [ Time Frame: day 22 of each dietary treatment ]

7.  Secondary:   N-terminal Telopeptide (NTX) Plasma Concentration at Day 22   [ Time Frame: day 22 of each dietary treatment ]

8.  Other Pre-specified:   Bone Mineral Density Determined by Dual-energy X-ray Absorptiometry (DXA) Scan   [ Time Frame: once during first 3 week dietary treatment ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The trial was completed as planned with an adequate number of subjects and there were no limitations.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Denise M Ney
Organization: University of Wisconsin-Madison
phone: 608-262-4386
e-mail: ney@nutrisci.wisc.edu


Publications of Results:

Other Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: University of Wisconsin, Madison
ClinicalTrials.gov Identifier: NCT01428258     History of Changes
Other Study ID Numbers: H-2010-0165
1R01FD003711-01A1 ( U.S. FDA Grant/Contract )
First Submitted: August 31, 2011
First Posted: September 2, 2011
Results First Submitted: October 11, 2016
Results First Posted: March 3, 2017
Last Update Posted: November 17, 2017