Trial · NCT01376908
Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
NCT01376908, Phase 3, Completed, BioMarin Pharmaceutical; results first posted 2016-05-12
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Kuvan® in Phenylketonuria Patients Less Than 4 Years Old. Trials, record NCT01376908. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01376908
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
- NCT number
- NCT01376908
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 56 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Phenylketonuria
- Interventions
- Kuvan®, Phenylalanine (Phe)-restricted diet
- First posted
- 2011-06-20
- Last updated
- 2017-09-15
- Other registries' IDs
- eudract:2009-015768-33
- Results first posted
- 2016-05-12
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Dietary Phenylalanine (Phe) Tolerance at Week 26 | Week 26 | Phe tolerance was defined as the amount of dietary Phe prescribed (milligram per kilogram per day \[mg/kg/day\]) while maintaining blood Phe levels within the selected therapeutic target range (defined as greater than or equal to \[\>=\] 120 to less than \[\<\] 360 micromoles per liter \[mcmol/L\]). |
| Secondary | Mean Blood Phe Levels | Baseline, Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, and 26 | Mean blood phe levels were defined as the mean of blood phe levels assessed over each 2-week intervals |
| Secondary | Change From Baseline in Dietary Phe Tolerance After 26 Weeks | Baseline and at Week 26 (last observation carried-forward [LOCF]) | Phe tolerance was defined as the amount of dietary Phe ingested (mg/kg/day) while maintaining blood Phe levels within the selected therapeutic target range (defined as \>=120 to \<360 mcmol/L). |
| Secondary | Number of Subjects With Any TEAEs, AEs Related to Kuvan, Serious AEs, AEs Leading to Death, and AEs Leading to Discontinuation | From the first dose of study drug administration up to 31 days after the last dose of study drug administration | An AE was defined as any new untoward medical occurrences/worsening of pre-existing medical condition without regard to possibility of causal relationship. A serious adverse event (SAE) was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect. Treatment-emergent are events between first dose of study treatment and up to 31 days after last dose that were absent before treatment or that worsened relative to pretreatment state. |
| Secondary | Number of Subjects With Normal Neuromotor Developmental Milestones Assessed Using Denver Developmental Scale (DDS) | Baseline, Weeks 12, 26 | Subjects with normal neuromotor development were assessed by standardized developmental milestones using a parent/guardian report form in the following areas: fine motor, gross motor, language, and personal-social using DDS Test. DDS Test is a widely used to examine the developmental progress of 0-6 years of children. The scale reflects what percentage of a certain age group is able to perform a certain task. Tasks are grouped into 4 categories (social contact, fine motor skill, language, and gross motor skill) and include items such as smiles spontaneously (performed by 90% of three-month-olds), knocks 2 building blocks against each other (90% of 13-month-olds), speaks 3 words other than "mom" and "dad" (90% of 21-month-olds), or hops on 1 leg (90% of 5-year-olds). The more items a child fails to perform (passed by 90% of his/her peers), the more likely the child manifests a significant developmental problems. |
| Secondary | Neurodevelopmental Status Assessed Using Bayley III Scales of Infant and Toddler Development | Baseline and Week 26 | Neurodevelopmental assessments was done using the following age-dependent scales: Bayley III for subjects less than (\<) 3.5 years of age and WPPSI III for subjects greater than or equal to (\>=) 3.5 to \<4 years of age, based on following scores: adaptive behavior composite (ABC) score, cognitive composite (CC) score, language composite (LC) score, motor composite (MC) score., and social-emotional composite (SEC) score. Composite scores ranged from 40 (very poor) to 160 (excellent) and are classified as following: \>=115: accelerated performance; 85-114: development within normal limits; 70-84: mildly delayed development; less than or equal to (\<=) 69: significant delayed development. |
| Secondary | Growth Parameters Standard Deviation Scores (SDS) | Baseline, Weeks 4, 8, 12, 16, 20, and 26 | Growth assessment was performed by monitoring body mass index, height (or length), weight, and maximal occipital-frontal head circumference (MOFHC). Supine length was measured up to 2 years of age thereafter standing height was measured unless subject was unable to stand upright, in which case supine length was measured. Respective parameter SDS was calculated as the value of parameter minus reference mean value of parameter divided by standard deviation of the reference population. |
| Secondary | Number of Subjects With Hypophenylalanemia | Week 26 | Hypophenylalanemia is defined as the condition of blood Phe levels \<120 mcmol/L. |
| Secondary | Dietary Phe Tolerance During Extension Period | Every 6 months during 3 year extension period or until product is commercially approved | Phe tolerance was defined as the amount of dietary Phe ingested (mg/kg/day) while maintaining blood Phe levels within the selected therapeutic target range (defined as \>=120 to \<360 mcmol/L). |
| Secondary | Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) | Baseline, Weeks 4, 8, 12, 16, 20, and 26 |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Dietary Phenylalanine (Phe) Tolerance at Week 26 | Week 26 | Intention-to-treat (ITT) population consisted of all the randomized subjects at the start of the study and were analyzed according to the group allocated. | mg/kg/day | MEAN |
| SECONDARY | Mean Blood Phe Levels | Baseline, Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, and 26 | Intention-to-treat (ITT) population consisted of all the randomized subjects at the start of the study and were analyzed according to the group allocated. 'n' signifies number of subjects evaluable for this measure at given time points for each reporting group respectively. | micromol per liter (mmol/L) | MEAN |
| SECONDARY | Change From Baseline in Dietary Phe Tolerance After 26 Weeks | Baseline and at Week 26 (last observation carried-forward [LOCF]) | Intention-to-treat (ITT) population consisted of all the randomized subjects at the start of the study and were analyzed according to the group allocated. 'n' signifies number of subjects evaluable for this measure at given time points for each reporting group respectively. | mg/kg/day | MEAN |
| SECONDARY | Number of Subjects With Any TEAEs, AEs Related to Kuvan, Serious AEs, AEs Leading to Death, and AEs Leading to Discontinuation | From the first dose of study drug administration up to 31 days after the last dose of study drug administration | Safety population included all subjects who either received at least one dose of Kuvan in the study period, or were randomized to Phe-restricted diet alone and who had some safety assessment data available. | subjects | NUMBER |
| SECONDARY | Number of Subjects With Normal Neuromotor Developmental Milestones Assessed Using Denver Developmental Scale (DDS) | Baseline, Weeks 12, 26 | Intention-to-Treat (ITT) population consisted of all subjects who were randomized at the start of the Study Period and analyzed according to the group allocated. n signifies number of evaluable subjects in the specified categories, for each reporting group, respectively. | subjects | NUMBER |
| SECONDARY | Neurodevelopmental Status Assessed Using Bayley III Scales of Infant and Toddler Development | Baseline and Week 26 | Intention-to-Treat (ITT) population consisted of all subjects who were randomized at the start of the Study Period and analyzed according to the group allocated. n signifies number of evaluable subjects in the specified categories, for each reporting group, respectively. | Units on a scale | MEAN |
| SECONDARY | Growth Parameters Standard Deviation Scores (SDS) | Baseline, Weeks 4, 8, 12, 16, 20, and 26 | Intention-to-Treat (ITT) population consisted of all subjects who were randomized at the start of the Study Period and analyzed according to the group allocated. n signifies number of evaluable subjects in the specified categories for each reporting group, respectively. | SDS | MEAN |
| SECONDARY | Number of Subjects With Hypophenylalanemia | Week 26 | Safety population consisted of all subjects who had some safety assessment data available (at least one visit in vital signs, AE or laboratory results) in the Study Period and who received at least one dose of Kuvan in the Study Period, or who were randomized to Phe-restricted diet alone. | Subjects | NUMBER |
| SECONDARY | Dietary Phe Tolerance During Extension Period | Every 6 months during 3 year extension period or until product is commercially approved | The extension period of this study is ongoing. Data will be provided after completion of extension period i.e first quarter 2017 | ||
| SECONDARY | Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) | Baseline, Weeks 4, 8, 12, 16, 20, and 26 | Intention-to-treat (ITT) population consisted of all the randomized subjects at the start of the study and were analyzed according to the group allocated. n signifies number of evaluable subjects in the specified categories for each reporting group, respectively. | mmHg | MEAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group183
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Dietary Phenylalanine (Phe) Tolerance at Week 26 | Kuvan® + Phe-restricted Diet | 80.6 | Standard Error 4.2 | mg/kg/day | |
| Dietary Phenylalanine (Phe) Tolerance at Week 26 | Phe-restricted Diet | 50.1 | Standard Error 4.3 | mg/kg/day | |
| Mean Blood Phe Levels | Kuvan® + Phe-restricted Diet | Baseline (n=27, 29) | 287.3 | Standard Deviation 166.6 | micromol per liter (mmol/L) |
| Mean Blood Phe Levels | Phe-restricted Diet | Baseline (n=27, 29) | 352.9 | Standard Deviation 219.9 | micromol per liter (mmol/L) |
| Mean Blood Phe Levels | Kuvan® + Phe-restricted Diet | Week 2 (n=27, 26) | 214.3 | Standard Deviation 89.3 | micromol per liter (mmol/L) |
| Mean Blood Phe Levels | Phe-restricted Diet | Week 2 (n=27, 26) | 321.3 | Standard Deviation 133.5 | micromol per liter (mmol/L) |
| Mean Blood Phe Levels | Kuvan® + Phe-restricted Diet | Week 4 (n=27, 24) | 202.1 | Standard Deviation 79.3 | micromol per liter (mmol/L) |
| Mean Blood Phe Levels | Phe-restricted Diet | Week 4 (n=27, 24) | 308.0 | Standard Deviation 122.2 | micromol per liter (mmol/L) |
| Mean Blood Phe Levels | Kuvan® + Phe-restricted Diet | Week 6 (n=25, 26) | 248.0 | Standard Deviation 85.4 | micromol per liter (mmol/L) |
| Mean Blood Phe Levels | Phe-restricted Diet | Week 6 (n=25, 26) | 303.8 | Standard Deviation 87.4 | micromol per liter (mmol/L) |
1 to 10 of 183Source ClinicalTrials.gov
See all 183 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Kuvan® + Phe-restricted Diet | 27 | |
| Overall Study | STARTED | Phe-restricted Diet | 29 | |
| Overall Study | COMPLETED | Kuvan® + Phe-restricted Diet | 25 | |
| Overall Study | COMPLETED | Phe-restricted Diet | 26 | |
| Overall Study | NOT COMPLETED | Kuvan® + Phe-restricted Diet | 2 | |
| Overall Study | NOT COMPLETED | Phe-restricted Diet | 3 |
Source ClinicalTrials.gov
Adverse events44
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Nasopharyngitis | Infections and infestations | Kuvan® + Phe-restricted Diet | 13 | 27 | |
| other | Nasopharyngitis | Infections and infestations | Phe-restricted Diet | 11 | 27 | |
| other | Rhinitis | Infections and infestations | Kuvan® + Phe-restricted Diet | 8 | 27 | |
| other | Rhinitis | Infections and infestations | Phe-restricted Diet | 6 | 27 | |
| other | Pharyngitis | Infections and infestations | Kuvan® + Phe-restricted Diet | 7 | 27 | |
| other | Pharyngitis | Infections and infestations | Phe-restricted Diet | 3 | 27 | |
| other | Gastroenteritis | Infections and infestations | Kuvan® + Phe-restricted Diet | 3 | 27 | |
| other | Gastroenteritis | Infections and infestations | Phe-restricted Diet | 3 | 27 | |
| other | Otitis media | Infections and infestations | Kuvan® + Phe-restricted Diet | 2 | 27 | |
| other | Otitis media | Infections and infestations | Phe-restricted Diet | 3 | 27 |
1 to 10 of 44Source ClinicalTrials.gov
See all 44 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Kuvan® + Phe-restricted Diet | ||
| Total, all-cause mortality | Phe-restricted Diet | ||
| Total, other adverse events | Kuvan® + Phe-restricted Diet | 26 | 27 |
| Total, other adverse events | Phe-restricted Diet | 26 | 27 |
| Total, serious adverse events | Kuvan® + Phe-restricted Diet | 3 | 27 |
| Total, serious adverse events | Phe-restricted Diet | 1 | 27 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Kuvan® + Phe-restricted Diet vs Phe-restricted Diet: serious adverse events |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01376908
GET https://conductscience.org/api/trials/v1/NCT01376908/results?page=2