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    Summary
    EudraCT Number:2015-003131-35
    Sponsor's Protocol Code Number:AA-HPP-208
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-12-03
    Trial results View 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 number2015-003131-35
    A.3Full title of the trial
    A Phase 2a Randomized, Multi-center, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients with Pediatric-Onset Hypophosphatasia
    Eine offene, randomisierte, multizentrische, pharmakokinetische Dosis-Wirkungs-Studie der Phase IIA von Asfotase bei erwachsenen Patienten mit seit dem Kindesalter bestehender Hypophosphatasie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2a Randomized, Multi-center, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients with Pediatric-Onset Hypophosphatasia
    A.4.1Sponsor's protocol code numberAA-HPP-208
    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 SponsorAlexion Pharma GmbH
    B.1.3.4CountrySwitzerland
    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 supportAlexion Pharma GmbH
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlexion Europe SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address1-15 avenue Edouard Belin
    B.5.3.2Town/ cityRueil-Malmaison
    B.5.3.3Post code92500
    B.5.3.4CountryFrance
    B.5.4Telephone number+33147100606
    B.5.5Fax number+33147100611
    B.5.6E-mailclinicaltrials.eu@alexion.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 Yes
    D.2.1.1.1Trade name Strensiq
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Europe SAS
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/08/594
    D.3 Description of the IMP
    D.3.1Product nameasfotase alfa
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNASFOTASE ALFA
    D.3.9.3Other descriptive nameHuman Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein
    D.3.9.4EV Substance CodeSUB168381
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    Hypophosphatasia is a rare inborn error of bone metabolism caused by inactivating mutations in the gene encoding the Tissue-nonspecific alkaline phosphatase isoenzyme.
    With deficiency of Tissue-nonspecific alkaline phosphatase, there is a buildup of extracellular inorganic pyrophosphate, which inhibits mineralization of bone matrix.
    E.1.1.1Medical condition in easily understood language
    Hypophosphatasia a bone disorder caused by genetic changes called mutations. These gene mutations result in low levels of an enzyme needed to harden children's bones.
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10049933
    E.1.2Term Hypophosphatasia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 pharmacokinetics (PK) and pharmacodynamics (PD) of asfotase alfa following administration of a range of dose regimens that encompasses the dose proven to be effective in children ( in adult patients with pediatric-onset HPP.
    E.2.2Secondary objectives of the trial
    to evaluate the safety and tolerability of asfotase alfa in adult patients with pediatric-onset HPP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all of the following criteria for enrollment in this study:
    1. Patients or their legal representative(s) must provide written informed consent prior to undergoing any study-related procedures
    2. Patient has pediatric-onset HPP, defined as onset of first sign(s)/symptom(s) of HPP prior to 18 years of age
    3. Documented diagnosis of HPP as indicated by a documented history of HPP-related skeletal abnormalities and 1 or more of the following:
    − Documented TNSALP gene mutation(s) from a certified laboratory
    − Serum ALP level below the age-adjusted normal range AND plasma PLP above the upper limit of normal at Screening.
    4. Patients must have a plasma PPi level of ≥3.9 μM at Screening
    5. Patients must be ≥18 years of age at Screening
    6. Sexually active male and female patients of childbearing potential must agree to use a highly effective method of birth control during the study and for 3 months following the last dose of study drug. Male patients must also not donate sperm during the Screening and treatment periods and for at least 3 months after the last dose of asfotase alfa. Highly effective contraceptive methods are as follows:
    a. Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation− Oral
    b. Progestogen-only hormonal contraception associated with inhibition of ovulation− Oral
    c. Intrauterine device
    d. Intrauterine hormone-releasing system
    e. Bilateral tubal occlusion
    f. Vasectomy with documented medical assessment of surgical success
    g. Condom and spermicide
    7. Female patients of childbearing potential must have a negative pregnancy test at the time of enrollment
    8. Female patients not of child-bearing potential due to surgical sterilization (at least 6 weeks after surgical bilateral oophorectomy with or without hysterectomy or at least 6 weeks after tubal ligation) confirmed by medical history, or menopause
    9. Patients must be willing to comply with study procedures and the visit schedule
    E.4Principal exclusion criteria
    Patients will be excluded from participation in this study if they meet any of the following exclusion criteria:
    1. Investigational site personnel directly affiliated with this study and/or their immediate families. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.
    2. Employees of Alexion Pharmaceuticals
    3. Currently enrolled in a clinical study involving another study drug or nonapproved use of a drug or device, or concurrently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study (excluding the HPP registry, in which concurrent enrollment is allowed).
    4. Participated, within the last 30 days, in a clinical study involving a study drug (other than the study drug used in this study). If the previous study drug has a long half-life, 3 months or 5 half-lives (whichever is longer) should have passed.
    5. Have completed or withdrawn from this study or any other study investigating asfotase alfa in the previous 3 years. This exclusion criterion does not apply to patients who are re-screened prior to randomization or patients enrolled in the HPP registry.
    6. Women who are pregnant, planning to become pregnant, or breastfeeding
    7. Serum 25-OH Vitamin D below 20 ng/mL at Screening
    8. Screening serum creatinine or parathyroid hormone (PTH) levels 1.5 times the upper limit of normal
    9. Medical condition, serious concurrent illness and/or injury, recent orthopedic surgery, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance or study endpoints, including all prescribed evaluations and follow-up activities
    10. Prior treatment with bisphosphonates within 2 years of study entry for any length of time or for more than 2 consecutive years at any prior timepoint
    11. Treatment with PTH, strontium, or sclerostin inhibitors within 6 months prior to the first dose of study drug
    12. Unwilling or unable to comply with the use of a data collection device on which study patients will directly record data
    E.5 End points
    E.5.1Primary end point(s)
    Change in PPi
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week -12 to -2, -1, 1, 2, 3-5, 7-8, 9, 10-12, 13
    E.5.2Secondary end point(s)
    Change of PLP, assessment of PK parameters and assessment of safety
    E.5.2.1Timepoint(s) of evaluation of this end point
    Change of PLP: Week -12 to -2, -1, 1, 2, 3-5, 7-8, 9, 10-12, 13
    PK parameters: Week 1, 2, 3-5, 7-8, 9, 10-12, 13
    Safety: study duration
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Yes
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    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
    Germany
    United States
    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 years0
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days10
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days10
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 36
    F.1.3Elderly (>=65 years) No
    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 No
    F.3.3.7Others No
    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 15
    F.4.2.2In the whole clinical trial 36
    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)
    return to the standard of care
    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 Decision2016-04-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-04-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-06-21
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