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    Summary
    EudraCT Number:2021-000497-28
    Sponsor's Protocol Code Number:PTC923-MD-004-PKU
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-12-22
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2021-000497-28
    A.3Full title of the trial
    Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
    Eine offene Phase-III-Studie zu PTC923 (Sepiapterin) bei Phenylketonurie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Study of PTC923 (Sepiapterin) in Subjects with Phenylketonuria
    A.4.1Sponsor's protocol code numberPTC923-MD-004-PKU
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPTC Therapeutics, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPTC Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPTC Therapeutics Inc.
    B.5.2Functional name of contact pointPatient Advocacy
    B.5.3 Address:
    B.5.3.1Street Address100 Corporate Court
    B.5.3.2Town/ citySouth Plainfield
    B.5.3.3Post codeNJ07080
    B.5.3.4CountryUnited States
    B.5.6E-mailMedinfo@ptcbio.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/21/2435
    D.3 Description of the IMP
    D.3.2Product code PTC923
    D.3.4Pharmaceutical form Powder for oral suspension in sachet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot available
    D.3.9.1CAS number 17094-01-8
    D.3.9.2Current sponsor codePTC923
    D.3.9.3Other descriptive name(S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
    D.3.9.4EV Substance CodeSUB193410
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/21/2435
    D.3 Description of the IMP
    D.3.2Product code PTC923
    D.3.4Pharmaceutical form Powder for oral suspension in sachet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot available
    D.3.9.1CAS number 17094-01-8
    D.3.9.2Current sponsor codePTC923
    D.3.9.3Other descriptive name(S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
    D.3.9.4EV Substance CodeSUB193410
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Metabolic Disorders - Phenylketonuria
    E.1.1.1Medical condition in easily understood language
    Phenylketonuria (PKU) is an autosomal-recessive inborn error of metabolism characterized by deficiency of the enzyme phenylalanine hydroxylase (PAH), which metabolizes phenylalanine (Phe)
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10034873
    E.1.2Term Phenylketonuria (PKU)
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To evaluate the long-term safety of PTC923 in subjects with PKU
    • To evaluate changes from baseline in dietary Phe/protein consumption
    E.2.2Secondary objectives of the trial
    • To evaluate PTC923 effect on quality of life (QOL) using the
    Phenylketonuria-quality of life (PKU-QOL) questionnaire in the subset of subjects who are able to complete the PKU-QOL (ie, subjects whose primary language is English [British or American], Turkish, Dutch, German, Spanish, Italian, Portuguese,
    or French) (ages 6 to 8 years Parent PKU-QOL; ages 9 to 11 years Child PKU-QOL; ages 12 to 17 years Adolescent PKU-QOL; ages ≥18 years Adult PKU-QOL)
    • To evaluate PTC923 effect of QOL using the European Quality of Life 5 Dimensions (EQ-5D) (EQ-5D for Youth [EQ-5D-Y] Proxy Version 1 [3 to 7 years]; EQ-5D-Y [8 to 15 years]; EQ-5D- 5 Levels (EQ-5D5L ([≥16 years])
    •To evaluate the pharmacokinetics (PK) of sepiapterin and tetrahydrobiopterin (BH4) following PTC923 dosing
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Informed consent and assent (if necessary, at the investigator’s discretion [ie, for children
    and/or subjects that are mentally impaired secondary to disease]) with parental/legal
    guardian consent
    2. Male or female subjects of any age
    3. Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by past medical history of at least 2 blood Phe measurements ≥600 ╬╝mol/L
    3. Women of childbearing potential, as defined in (CTFG 2020), must have a negative
    pregnancy test at study entry and agree to abstinence or the use of at least one highly
    effective form of contraception (with a failure rate of <1% per year when used
    consistently and correctly):
    • Combined (estrogen and progestogen containing) hormonal contraception associated
    with inhibition of ovulation:
    − Oral
    − Intravaginal
    − Transdermal
    • Progestogen-only hormonal contraception associated with inhibition of ovulation:
    − Oral
    − Injectable
    − Implantable
    • Intrauterine device
    • Intrauterine hormone-releasing system
    • Bilateral tubal occlusion
    • Vasectomized partner with confirmed azoospermia
    Highly effective contraception or abstinence must be continued for the duration of the
    study, and for up to 90 days after the last dose of the study drug.
    All females will be considered of childbearing potential unless they are postmenopausal
    (at least 12 months consecutive amenorrhea in the appropriate age group without other
    known or suspected cause) or have been permanently sterilized surgically (eg, hysterectomy, bilateral
    salpingectomy, bilateral oophorectomy).
    4. 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.
    Males who are abstinent will not be required to use a contraceptive method unless they
    become sexually active. Males who have undergone a vasectomy are not required to use a
    contraceptive method if at least 16 weeks post procedure.
    5. Willing and able to comply with the protocol and study procedure
    6. Willing to continue current diet unchanged while participating in the study (unless
    specifically instructed to change diet during the study by the investigator)
    E.4Principal exclusion criteria
    1. The individual, in the opinion of the investigator, is unwilling or unable to adhere to the
    requirements of the study
    2. Inability to tolerate oral medication
    3. A female who is pregnant or breastfeeding, or considering pregnancy
    4. Serious neuropsychiatric illness (eg, major depression) not currently under medical
    control, that in the opinion of the investigator or sponsor, would interfere with the
    subject’s ability to participate in the study or increase the risk of participation for that
    subject
    5. Past medical history and/or evidence of renal impairment and/or condition including
    moderate/severe renal insufficiency (glomerular filtration rate [GFR] <60 mL/min) as estimated most recently during qualifying participation in a feeder study) and/or under care of a nephrologist
    6. Any other condition that in the opinion of the investigator or sponsor, would interfere
    with the subject’s ability to participate in the study or increase the risk of participation for
    that subject
    7. Requirement for concomitant treatment with any drug known to inhibit folate synthesis
    (eg, methotrexate)
    8. Concomitant treatment with BH4 supplementation (eg, sapropterin dihydrochloride,
    KUVAN) or pegvaliase-pqpz (PALYNZIQ)
    Additional criteria for subjects who did not participate in a feeder study:
    9. 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
    10. History of gastric surgery, including Roux-en-Y gastric bypass
    surgery or an antrectomy with vagotomy, or gastrectomy
    11. History of allergies or adverse reactions to synthetic BH4 or
    sepiapterin
    12. Current participation in any other investigational drug study or use
    of any investigational agent within 30 days prior to Screening
    13. Any clinically significant laboratory abnormality as determined by the
    investigator. In general, each laboratory value from Screening and
    baseline chemistry and hematology panels should fall within the limits of
    the normal laboratory reference range, unless deemed not clinically
    significant by the investigator
    14. Any abnormal physical examination and/or laboratory findings
    indicative of signs or symptoms of renal disease, including calculated
    GFR <60 mL/min/1.73 m2.
    In subjects ≥18 years of age, the Modification of Diet in Renal Disease
    Equation should be used to determine GFR.
    In subjects <18 years of age, the Bedside Schwartz Equation should be
    used to determine GFR.
    15. Confirmed diagnosis of a primary BH4 deficiency as evidenced by
    biallelic pathogenic mutations in 6-pyruvoyltetrahydropterin synthase,
    recessive GTP cyclohydrolase I, sepiapterin reductase, quinoid
    dihydropteridine reductase, or pterin 4-alpha-carbinolamine dehydratase
    genes
    16. Major surgery within the prior 90 days of screening
    17. Unwillingness to washout from BH4 supplementation (eg,
    sapropterin dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ)
    E.5 End points
    E.5.1Primary end point(s)
    Safety is a primary endpoint: Safety of long-term treatment with PTC923 will be measured by
    number of treatment-emergent adverse events (TEAEs), including assessment of severity of
    TEAEs, clinical laboratory tests, vital signs, and physical examinations.

    Primary efficacy endpoint is the change from baseline in dietary Phe/protein consumption
    measured during Dietary Phe Tolerance Assessment period.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety of long-term treatment with PTC923 will be measured by
    number of treatment-emergent adverse events (TEAEs), including assessment of severity of
    TEAEs, clinical laboratory tests, vital signs, and physical examinations.

    Dietary Phe tolerance evaluated at Baseline and every 2 weeks over the course of 26 weeks assessment period
    E.5.2Secondary end point(s)
    • Changes from baseline in QOL using PKU-QOL questionnaire in the subset of
    subjects who are able to complete the PKU-QOL (ie, subjects whose primary
    language is English [British or American], Turkish, Dutch, German, Spanish,
    Italian, Portuguese, or French) (ages 6 to 8 years Parent PKU-QOL; ages 9 to
    11 years Child PKU-QOL; ages 12 to 17 years Adolescent PKU-QOL;
    ages ≥18 years Adult PKU-QOL)
    • Changes from baseline in QOL using the EQ-5D (EQ-5D-Y Proxy Version 1
    [3 to 7 years]; EQ-5D-Y [8 to 15 years]; EQ-5D-5L ([≥16 years])
    •PK assessment of sepiapterin and BH4 concentrations in plasma following dosing of sepiapterin
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary measures will be evaluated at 6-month intervals starting at M8D1.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Brazil
    Canada
    Mexico
    United States
    Georgia
    Turkey
    Denmark
    France
    Germany
    Italy
    Netherlands
    Portugal
    Spain
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 200
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 10
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 20
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Informed consent and assent (if necessary, at the investigator’s discretion [ie, for children and/or subjects who are mentally impaired secondary to disease]) with parental/legal guardian consent
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    0-18 years
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 98
    F.4.2.2In the whole clinical trial 200
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    none
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-04-28
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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