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    Summary
    EudraCT Number:2005-004893-26
    Sponsor's Protocol Code Number:D6992C00044
    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:2006-06-21
    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 number2005-004893-26
    A.3Full title of the trial
    An open-label, non-comparative trial to evaluate the safety, efficacy and pharmacokinetics of FASLODEX (fulvestrant) in girls with progressive precocious puberty associated with McCune-Albright Syndrome.
    A.4.1Sponsor's protocol code numberD6992C00044
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAstraZeneca AB
    B.1.3.4CountrySweden
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Faslodex 250 mg/5 ml solution for injection
    D.2.1.1.2Name of the Marketing Authorisation holderAstraZeneca UK Limited
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameFaslodex
    D.3.2Product code ZD9238
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNfulvestrant
    D.3.9.1CAS number 129453-61-8
    D.3.9.2Current sponsor codeZD9238
    D.3.9.3Other descriptive nameICI 182,780
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    Progressive precocious puberty associated with McCune-Albright Syndrome
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the trial is composed of two components: A safety and efficacy component and a pharmacokinetics (PK) component. All patients fulfilling the eligibility criteria will participate in both components of the trial.
    The safety of study treatment will be evaluated by assessments of adverse events, withdrawals, laboratory data, ovarian volume as assessed by ultrasound, including the number of ovarian cysts and size of the largest cyst, and uterine volume. The efficacy of study treatment will be based on change in frequency of vaginal bleeding days, rate of increase in bone age, and growth rate.
    The second component will assess the pharmacokinetics of fulvestrant in girls with progressive precocious puberty (PPP) associated with McCune-Albright syndrome (MAS).
    E.2.2Secondary objectives of the trial
    Secondary objectives include assessments of pubertal progression through Tanner Staging and predicted adult height (PAH) for children over age six. The presence of a MAS associated Gsa mutation will be assessed by molecular analysis provided separate specific consent is obtained. No other genetic analysis will be performed with these specimens. The participation of patients and investigative sites in this analysis will be voluntary and any individual patient or site decision not to participate will not exclude them from this clinical study.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Provision of written informed consent of parent/legal guardian and subject assent (as required by local law)
    2. For patients consenting to participate in the genetic portion of the study, provision of an additional written informed consent specific for DNA sampling and genetic analysis.
    3. Females less than or equal to 10 years of age (prior to 11th birthday) at the start of trial therapy (Visit 0).
    4. Diagnosis of McCune-Albright Syndrome based on having the following clinical criteria:
    · Precocious puberty evident before the age of 8 years
    And at least one of the following clinical criteria:
    · Café au lait spots
    · Fibrous dysplasia
    · Presence of Gsa mutation
    5. Progressive precocious puberty associated with MAS. Progressive precocious puberty will be defined as:
    o An increase of at least one in the breast Tanner Stage over the observation period

    and/or
    o The development or persistence of vaginal bleeding during the observation period.
    6. At least one of the following two criteria must also be fulfilled:
    o Advanced bone age: defined as, bone age of at least 12 months beyond chronological age at the time of screening.
    o Rapid growth rate: a growth rate over the observation period that is more than 2 standard deviations above the mean for age, where the growth rate is defined as the change in height (or length) (in cm) divided by the change in time (annualized in years).
    7. Patients are eligible provided they are in one of the following categories:
    o Received no previous treatment and have documented retrospective data of at least 6 months for bone age, Tanner Stage, height, weight, and vaginal bleeding (number of bleeding days).
    o Received no previous treatment and do not have documented retrospective data of at least 6 months for bone age, Tanner Stage, height, weight and vaginal bleeding (number of bleeding days) and can be observed for 6 months without treatment (i.e., 6-month observation period).
    o Documented progression on treatment with anastrozole, tamoxifen, testolactone, or other aromatase inhibitors, an anti-androgen, or a progestin requiring immediate treatment provided there exists retrospective data of at least 6 months for bone age, Tanner Stage, height, weight, and vaginal bleeding (number of bleeding days) and are off these agents for 1 month prior to first dose of study drug.
    o Previously treated with any drug for PPP in which therapy was stopped for at least 6 months with subsequent clinical evidence of progression of disease meeting entry criteria No. 5 and 6 and retrospective data of at least 6 months for bone age, Tanner Stage, height, weight, and vaginal bleeding (number of bleeding days).
    8. If central precocious puberty (CPP) exists, the patient must have been on a GnRH analog (e.g., Lupron) for at least 6 months prior to study enrollment (date of written consent of parent/legal guardian and patient assent).
    E.4Principal exclusion criteria
    1. Male gender
    2. Any prior treatment of PPP associated with MAS with fulvestrant
    3. Concomitant treatment of PPP associated with MAS, with the exception of bisphosphonates for fibrous dysplasia and GnRH analogs in the case of CPP
    4. Liver function tests (AST, ALT) at screening ³ 3´ the upper limit of the reference range for age
    5. Platelet count at screening less than 100 ´ 109/L
    6. International normalized ratio (INR) greater than 1.6.
    7. History of bleeding diathesis or long-term anticoagulant therapy (other than antiplatelet therapy)
    8. Any severe concomitant condition that makes it undesirable for the patient to participate in this study
    9. Known hypersensitivity to any component of the study drug product
    E.5 End points
    E.5.1Primary end point(s)
    (a) Tolerability and safety, including adverse events, withdrawals and laboratory data
    (b) Change in frequency of annualized days of vaginal bleeding on treatment compared to baseline
    (c) Proportion of patients with baseline vaginal bleeding who experienced ³50% reduction in the number of vaginal bleeding days on treatment compared to baseline
    (d) Proportion of patients with baseline vaginal bleeding who experienced cessation of vaginal bleeding over a 6-month trial period and over the entire 12-month trial.
    (e) Change in bone age advancement (rate of increase in bone age) over a 6-month trial period and over the entire 12-month trial on treatment compared to baseline
    (f) Change in growth rate over a 6-month trial period and over the entire 12-month trial on treatment compared to baseline
    PK variables for the population PK analysis will include area under the concentration-time curve (AUC), maximum and minimum plasma concentration (Cmax and Cmin, respectively), half-life (T1/2), clearance (CL/F) and apparent volume of distribution (Vss). The effects of demographic covariates (e.g., age, body weight, race) on fulvestrant PK will also be explored.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Information not present in EudraCT
    E.6.2Prophylaxis Information not present in EudraCT
    E.6.3Therapy Information not present in EudraCT
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Information not present in EudraCT
    E.6.8Bioequivalence Information not present in EudraCT
    E.6.9Dose response Information not present in EudraCT
    E.6.10Pharmacogenetic Information not present in EudraCT
    E.6.11Pharmacogenomic Information not present in EudraCT
    E.6.12Pharmacoeconomic Information not present in EudraCT
    E.6.13Others Information not present in EudraCT
    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 No
    E.8.1.2Open No
    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.8.3 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.8.5The trial involves multiple Member States Yes
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    Last visit of last patient. Patients have the option to continue treatment, as long as the investigator considers it is in their best interest, until the onset of normal puberty, or if the patient experiences a drug-related toxicity requiring treatment discontinuation.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male No
    F.3 Group of trial subjects
    F.3.1Healthy volunteers Information not present in EudraCT
    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 No
    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
    Adolescent children (less than 11 years of age) will be included in the study.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 30
    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)
    Patients have the option to continue treatment, as long as the investigator considers it is in their best interest, until the onset of normal puberty, or if the patient experiences a drug-related toxicity requiring treatment discontinuation.
    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 Decision2006-11-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2007-02-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-07-20
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