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Trial Outcomes & Findings for Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy (NCT NCT01541397)

NCT ID: NCT01541397

Last Updated: 2015-07-07

Results Overview

A DXA scan will be conducted one year after Kuvan therapy is initiated.

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

6 participants

Primary outcome timeframe

1 year after initiation of Kuvan therapy

Results posted on

2015-07-07

Participant Flow

Participant milestones

Participant milestones
Measure
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
Overall Study
STARTED
4
2
Overall Study
COMPLETED
0
0
Overall Study
NOT COMPLETED
4
2

Reasons for withdrawal

Reasons for withdrawal
Measure
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
Overall Study
Study ended prior to data collection
4
2

Baseline Characteristics

Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Non-Kuvan Treated
n=4 Participants
Adults with hyperphenylalaninemia who have are not receiving Kuvan therapy.
Kuvan Treated
n=2 Participants
Adults with hyperphenylalaninemia who are treated with Kuvan (sapropterin). Sapropterin: 20 mg/kg, orally, daily, 1 year or patient chooses to discontinue therapy
Total
n=6 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
4 Participants
n=5 Participants
2 Participants
n=7 Participants
6 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Sex: Female, Male
Female
2 Participants
n=5 Participants
1 Participants
n=7 Participants
3 Participants
n=5 Participants
Sex: Female, Male
Male
2 Participants
n=5 Participants
1 Participants
n=7 Participants
3 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 1 year after initiation of Kuvan therapy

Population: Zero participants were analyzed because the study was ended early (due to an insufficient number of enrolled participants).

A DXA scan will be conducted one year after Kuvan therapy is initiated.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: every three months up to 1 year

Population: Zero participants were analyzed because the study was ended early (due to an insufficient number of enrolled participants).

Evaluation of levels of plasma amino acids.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: every 3 months up to 1 year

Population: Zero participants were analyzed because the study was ended early (due to an insufficient number of enrolled participants).

Subjects will provide a 3 day diet record for every plasma amino acid evaluation. Diets will be analyzed to determine phenylalanine, protein, calories, fat, vitamins and minerals.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: weekly for 6 weeks, then at least every three months up to 1 year

Population: Zero participants were analyzed because the study was ended early (due to an insufficient number of enrolled participants).

Plasma phenylalanine levels will be monitored to determine effectiveness of Kuvan therapy.

Outcome measures

Outcome data not reported

Adverse Events

Non-Kuvan Treated

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Kuvan Treated

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Heather Saavedra

The University of Texas Health Science Center at Houston

Phone: 713-500-7098

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place