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The European Union Clinical Trials Register allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   41198   clinical trials with a EudraCT protocol, of which   6743   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
     
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    Summary
    EudraCT Number:2013-000321-31
    Sponsor's Protocol Code Number:rhLAMAN-09
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2018-02-21
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2013-000321-31
    A.3Full title of the trial
    A multi-center, un-controlled, open-labeled trial of the long-term safety of Velmanase Alfa aftercare
    treatment of subjects with alpha-Mannosidosis whom previously participated in Velmanase Alfa - trials
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A long-term safety trial of repeated Velmanase Alfa treatment of subjects with Alpha-Mannosidosis
    A.3.2Name or abbreviated title of the trial where available
    Phase IIIb
    A.4.1Sponsor's protocol code numberrhLAMAN-09
    A.5.4Other Identifiers
    Name:EudraCTNumber:2013-000321-31
    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 SponsorChiesi Farmaceutici S.p.A.
    B.1.3.4CountryItaly
    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 supportChiesi Farmaceutici S.p.A
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationChiesi Farmaceutici S.p.A
    B.5.2Functional name of contact pointClinical Project Manager
    B.5.3 Address:
    B.5.3.1Street AddressVia Palermo
    B.5.3.2Town/ cityParma
    B.5.3.3Post code43122
    B.5.3.4CountryItaly
    B.5.4Telephone number003905211689176
    B.5.6E-mailclinicaltrials_info@chiesi.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/3/04/260
    D.3 Description of the IMP
    D.3.1Product nameVelmanase Alfa
    D.3.2Product code rhLAMAN
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNvelmanase alfa
    D.3.9.1CAS number 1492823-75-2
    D.3.9.2Current sponsor coderhLAMAN
    D.3.9.3Other descriptive namerecombinant human alpha-mannosidase
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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
    Treatement of Alpha-Mannosidosis
    E.1.1.1Medical condition in easily understood language
    Treatement of Alpha-Mannosidosis
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10032658
    E.1.2Term Other specified disorders of carbohydrate transport and metabolism
    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
    The overall objective of this trial is to provide aftercare treatment with Velmanase- Alfa and to evaluate the safety and efficacy of repeated Velmanase- Alfa i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Velmanase- Alfa -trial. This protocol only concern subjects, where compassionate use program was not accepted (OR NO PHYSICIAN COULD BE IDENTIFIED)
    Efficacy will be evaluated once yearly to follow the subject’s progress in clinical parameters and at one additional comprehensive evaluation visit (2500) to evaluate additional efficacy parameters.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. The subject must have participated in previous Velmanase- Alfa -trials: phase 2b: 2011-004355-40 or phase 3: 2012-000979-17
    2. Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
    3. The subject and his/her guardian(s) must have the ability to comply with the protocol
    E.4Principal exclusion criteria
    1. Presence of known clinically significant cardiovascular, hepatic, pulmonary, or renal disease or other medical conditions that, in the opinion of the Investigator, would preclude participation in the trial
    2. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
    3. Pregnancy: Pregnant woman is excluded. Before start of the treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
    4. Psychosis; any psychotic disease, also in remission, is an exclusion criteria
    5. Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
    E.5 End points
    E.5.1Primary end point(s)
    All endpoints are evaluated as change from baseline (pre-treatment in the subject’s original Velmanase- Alfa trial).
    Safety Endpoints:
    • Adverse events (AEs)
    • Vital signs and change in physical examination
    • Development of Velmanase- Alfa antibodies and neutralizing/inhibitory antibodies
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety will be assessed at every visit.
    Development of antibodies are measured every 12th week
    E.5.2Secondary end point(s)
    All endpoints are evaluated as change from baseline (pre-treatment in the subject’s original Velmanase- Alfa trial).
    The Efficacy endpoints of the trial are evaluation of:
    o Oligosaccharides in serum (visit 2500 only)
    o Motor proficiency (BOT-2) (visit 2500 only)
    o Three minute stair climb test (3MSCT)
    o Leiter R
    o Forced Vital Capacity (FVC)
    o Six minute walk test (6MWT)
    o Pure tone Audiometry
    o Questionnaires
    CNS parameters (visit 2500 only):
    o Assessment of mannose-rich oligosaccharides in brain tissue as measured by MRS visual score (for patients who previously participated in rhLAMAN-02), MRI diffusion coefficient in white matter, gray matter and centrum semi oval
    o Cerebrospinal fluid neuro-degeneration biomarker (Tau, NFL, GFAp)
    o Oligosaccharides in CSF
    Other laboratory parameters (visit 2500 only):
    o Plasma PK profile
    o Oligosaccharides in 24-h urine collection
    o Measurement of in vivo biological activity
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy will be assessed yearly, counted from the latest evaluation in the subject's previous Velmanase- Alfa trial, and at one additional comprehensive evaluation visit (2500) to evaluate additional efficacy parameters.
    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 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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years4
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years9
    E.8.9.2In all countries concerned by the trial months4
    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: 5
    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.1.1.5.1Number of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Due to the disease related mental retardation the subjects are under gardianship of the parents
    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 8
    F.4.2.2In the whole clinical trial 8
    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)
    When the trial is completed, given no safety concerns have arisen, all subjects will be offered to receive Velmanase- Alfa as post study therapy in the home country. This will last until the product is available on the market or until the sponsor decides to terminate the project. If the project is terminated, Chiesi has no further obligation to provide the study drug, even for post study therapy
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Wieslab AB (former BioMonitor)
    G.4.3.4Network Country Sweden
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-06-27
    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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