Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy

This study has been terminated.
Sponsor:
Collaborator:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
Heather Saavedra, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier:
NCT01541397
First received: July 11, 2011
Last updated: June 11, 2015
Last verified: June 2015
Results First Received: June 11, 2015  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Hyperphenylalaninemia
Phenylketonuria
Intervention: Drug: Sapropterin

  Participant Flow
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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
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Reporting Groups
  Description
Non-Kuvan Treated Adults with hyperphenylalaninemia who have are not receiving Kuvan therapy.
Kuvan Treated

Adults with hyperphenylalaninemia who are treated with Kuvan (sapropterin).

Sapropterin: 20 mg/kg, orally, daily, 1 year or patient chooses to discontinue therapy


Participant Flow:   Overall Study
    Non-Kuvan Treated     Kuvan Treated  
STARTED     4     2  
COMPLETED     0     0  
NOT COMPLETED     4     2  
Study ended prior to data collection                 4                 2  



  Baseline Characteristics
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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.
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Reporting Groups
  Description
Non-Kuvan Treated Adults with hyperphenylalaninemia who have are not receiving Kuvan therapy.
Kuvan Treated

Adults with hyperphenylalaninemia who are treated with Kuvan (sapropterin).

Sapropterin: 20 mg/kg, orally, daily, 1 year or patient chooses to discontinue therapy

Total Total of all reporting groups

Baseline Measures
    Non-Kuvan Treated     Kuvan Treated     Total  
Number of Participants  
[units: participants]
  4     2     6  
Age  
[units: participants]
     
<=18 years     0     0     0  
Between 18 and 65 years     4     2     6  
>=65 years     0     0     0  
Gender  
[units: participants]
     
Female     2     1     3  
Male     2     1     3  



  Outcome Measures
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1.  Primary:   Bone Mineral Density   [ Time Frame: 1 year after initiation of Kuvan therapy ]

2.  Secondary:   Plasma Amino Acid Profile   [ Time Frame: every three months up to 1 year ]

3.  Secondary:   Diet Analysis   [ Time Frame: every 3 months up to 1 year ]

4.  Secondary:   Plasma Phenylalanine Levels   [ Time Frame: weekly for 6 weeks, then at least every three months up to 1 year ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
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  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Heather Saavedra
Organization: The University of Texas Health Science Center at Houston
phone: 713-500-7098
e-mail: Heather.Saavedra@uth.tmc.edu



Responsible Party: Heather Saavedra, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier: NCT01541397     History of Changes
Other Study ID Numbers: HSC-MS-11-0119
Study First Received: July 11, 2011
Results First Received: June 11, 2015
Last Updated: June 11, 2015
Health Authority: United States: Institutional Review Board