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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05099640
Registration number
NCT05099640
Ethics application status
Date submitted
6/10/2021
Date registered
29/10/2021
Date last updated
10/01/2024
Titles & IDs
Public title
A Study of PTC923 in Participants With Phenylketonuria
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Scientific title
A Phase 3 Study of PTC923 in Subjects With Phenylketonuria
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Secondary ID [1]
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2021-000474-29
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Secondary ID [2]
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PTC923-MD-003-PKU
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Phenylketonuria
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Condition category
Condition code
Metabolic and Endocrine
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Other metabolic disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - PTC923
Treatment: Drugs - Placebo
Experimental: Part 1: PTC923 - Participants will receive PTC923 7.5 milligrams (mg)/kilogram (kg) (participants 0 to <6 months of age), 15 mg/kg (participants 6 to <12 months of age), 30 mg/kg (participants 12 months to <2 years of age), or 60 mg/kg (participants =2 years of age) orally once daily for 14 days.
Experimental: Part 2: PTC923 - Participants will receive PTC923 20 mg/kg daily for Weeks 1 and 2, then PTC923 40 mg/kg daily for Weeks 3 and 4, then PTC923 60 mg/kg daily for Weeks 5 and 6.
Placebo Comparator: Part 2: Placebo - Participants will receive equivalent quantities of placebo to match the 20 to 40 to 60 mg/kg dose escalation of the PTC923 treatment arm.
Treatment: Drugs: PTC923
PTC923 powder for oral use will be suspended in water or apple juice prior to administration.
Treatment: Drugs: Placebo
Placebo matching to PTC923
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phenylketonuria (Phe) Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline =30% During Part 1
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Assessment method [1]
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Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean level at Weeks 5 and 6 was calculated as the average of blood Phe levels collected during the Week 5-6 analysis visit window. Least square (LS) mean and standard error (SE) were calculated using mixed model repeated measures (MMRM) method.
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Timepoint [1]
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Baseline, Weeks 5 and 6 (average of the 2-week period)
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Primary outcome [2]
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Part 2 Double-blind Phase: Percent Change From Baseline in Blood Phe Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline =30% During Part 1
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Assessment method [2]
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Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean level at Weeks 5 and 6 was calculated as the average of blood Phe levels collected during the Week 5-6 analysis visit window. LS mean and SE were calculated using MMRM method.
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Timepoint [2]
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Baseline, Weeks 5 and 6 (average of the 2-week period)
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Secondary outcome [1]
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Part 2 Double-blind Phase: Percentage of Participants With Baseline Phe Levels =600 µmol/L Who Achieved Phe Levels <600 µmol/L in Participants With Phe Reduction From Baseline =30% During Part 1
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Assessment method [1]
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Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean level at Weeks 5 and 6 was calculated as the average of blood Phe levels collected during the Week 5-6 analysis visit window.
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Timepoint [1]
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Weeks 5 and 6 (average of the 2-week period)
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Secondary outcome [2]
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Part 2 Double-blind Phase: Percentage of Participants With Baseline Phe Levels =360 µmol/L Who Achieved Phe Levels <360 µmol/L in Participants With Phe Reduction From Baseline =30% During Part 1
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Assessment method [2]
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Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean level at Weeks 5 and 6 was calculated as the average of blood Phe levels collected during the Week 5-6 analysis visit window.
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Timepoint [2]
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Weeks 5 and 6 (average of the 2-week period)
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Secondary outcome [3]
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Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phe Level at Each 2-Week Period (Averaged Over Each 2-Week Period) in Participants With Phe Reduction From Baseline =30% During Part 1
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Assessment method [3]
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Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean levels at Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6 were calculated as the average of blood Phe levels collected during the Week 1-2, Week 3-4, and Week 5-6 analysis visit windows, respectively.
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Timepoint [3]
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Baseline, Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6 (average of each 2-week period)
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Secondary outcome [4]
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Part 2 Double-blind Phase: Percent Change From Baseline in Blood Phe Level at Each 2-Week Period (Averaged Over Each 2-Week Period) in Participants With Phe Reduction From Baseline =30% During Part 1
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Assessment method [4]
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Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean levels at Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6 were calculated as the average of blood Phe levels collected during the Week 1-2, Week 3-4, and Week 5-6 analysis visit windows, respectively.
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Timepoint [4]
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Baseline, Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6 (average of each 2-week period)
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Secondary outcome [5]
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Part 1 Open-label Run-in Phase: Plasma Concentration of Tetrahydrobiopterin (BH4) and Sepiapterin
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Assessment method [5]
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Timepoint [5]
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Predose, 0.5, 1, 2, 4, 6, 8, and 24 hours postdose at Day 1; 2 and 6 hours postdose at Day 14
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Secondary outcome [6]
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Part 2 Double-blind Phase: Plasma Concentration of BH4 and Sepiapterin
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Assessment method [6]
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Timepoint [6]
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Predose and 4 hours postdose at Days 1, 14, 28, and 42
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Secondary outcome [7]
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Part 1 Open-label Run-in Phase: Area Under the Concentration-time Curve From Time 0 to 24 Hours Postdose (AUC0-24h) of Sepiapterin and BH4 Following the First Dose of Sepiapterin at 60 mg/kg
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Assessment method [7]
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Timepoint [7]
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0 to 24 hours postdose at Day 1
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Secondary outcome [8]
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Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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Assessment method [8]
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An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. TEAEs were considered:
Part 1 TEAEs, which included all AEs occurring after first dose in Part 1 but before first dose in Part 2;
Part 2 TEAEs, which included all AEs after first randomized dose in Part 2. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
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Timepoint [8]
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Baseline up to Day 42
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Eligibility
Key inclusion criteria
- Uncontrolled blood Phe level =360 µmol/L on current therapy anytime during screening
and uncontrolled blood Phe level =360 µmol/L on current therapy when taking the
average of the 3 most recent Phe levels from the participant's medical history
(inclusive of the screening value).
- Clinical diagnosis of phenylketonuria with hyperphenylalaninemia (HPA) documented by
past medical history of at least 2 blood Phe measurements =600 µmol/L.
- Women of childbearing potential must have a negative pregnancy test at screening and
agree to abstinence or the use of at least one highly effective form of contraception
for the duration of the study, and for up to 90 days after the last dose of study
drug.
- Males who are sexually active with women of childbearing potential who have not had a
vasectomy must agree to use a barrier method of birth control during the study and for
up to 90 days after the last dose of study drug. Males must also refrain from sperm
donations during this time period.
- Willing to continue current diet unchanged while participating in the study.
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Gastrointestinal disease (such as irritable bowel syndrome, inflammatory bowel
disease, chronic gastritis, and peptic ulcer disease, etc.) that could affect the
absorption of study drug.
- History of gastric surgery, including Roux-en-Y gastric bypass surgery or an
antrectomy with vagotomy, or gastrectomy.
- History of allergies or adverse reactions to synthetic tetrahydrobiopterin (BH4) or
sepiapterin.
- Current participation in any other investigational drug study or use of any
investigational agent within 30 days prior to screening.
- Any clinically significant laboratory abnormality as determined by the investigator.
- A female who is pregnant or breastfeeding, or considering pregnancy.
- Serious neuropsychiatric illness (for example, major depression) not currently under
medical control, that in the opinion of the investigator or sponsor, would interfere
with the participant's ability to participate in the study or increase the risk of
participation for that participant.
- Past medical history and/or evidence of renal impairment and/or condition including
moderate/severe renal insufficiency (glomerular filtration rate [GFR] <60 milliliters
[mL]/minute [min]) and/or under care of a nephrologist.
- Any abnormal physical examination and/or laboratory findings indicative of signs or
symptoms of renal disease, including calculated GFR <60 mL/min/1.73 square meter
(m^2).
- Requirement for concomitant treatment with any drug known to inhibit folate synthesis
(for example, methotrexate).
- Confirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic pathogenic
mutations in 6-pyruvoyltetrahydropterin synthase, recessive guanosine-5'-triphosphate
(GTP) cyclohydrolase I, sepiapterin reductase, quinoid dihydropteridine reductase, or
pterin-4-alpha-carbinolamine dehydratase genes.
- Major surgery within the prior 90 days of screening.
- Concomitant treatment with BH4 supplementation (for example, sapropterin
dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ).
- Unwillingness to washout from BH4 supplementation (for example, sapropterin
dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ)
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
30/09/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
3/05/2023
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Sample size
Target
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Accrual to date
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Final
157
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Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC
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Recruitment hospital [1]
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Westmead Hospital - Westmead
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Recruitment hospital [2]
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PARC Clinical Research - Adelaide
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Recruitment hospital [3]
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Royal Melbourne Hospital - Melbourne
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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SA 5000 - Adelaide
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Recruitment postcode(s) [3]
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3050 - Melbourne
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Recruitment outside Australia
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United States of America
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California
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United States of America
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Colorado
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United States of America
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Florida
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United States of America
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Indiana
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United States of America
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Massachusetts
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United States of America
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New York
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United States of America
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Pennsylvania
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Texas
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Utah
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Wisconsin
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Brazil
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Rio Grande Do Sul
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Brazil
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São Paulo
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Canada
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Alberta
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Canada
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Ontario
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Denmark
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Copenhagen
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France
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Centre-Val De Loire
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Georgia
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Tbilisi
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Germany
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Hamburg
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Germany
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Heidelberg
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Germany
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Münster
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Italy
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Lazio
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Italy
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Veneto
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Mexico
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Jalisco
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Mexico
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Mexico City
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Netherlands
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Groningen
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Portugal
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Douro Litoral
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Portugal
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Estremadura
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Spain
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Esplugues De Llobregat
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Spain
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Madrid
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Turkey
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Ankara
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Turkey
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Istanbul
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Turkey
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Izmir
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Turkey
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Adana
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United Kingdom
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Birmingham
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
PTC Therapeutics
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood
phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in
blood Phe levels from baseline to Weeks 5 and 6 (that is, the average of each respective
treatment dose 2-week period of double-blind treatment).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05099640
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05099640
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