<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT01681940</url>
  </required_header>
  <id_info>
    <org_study_id>rhLAMAN-04</org_study_id>
    <secondary_id>2011-004355-40</secondary_id>
    <nct_id>NCT01681940</nct_id>
  </id_info>
  <brief_title>Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis</brief_title>
  <official_title>A Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Zymenex A/S</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>European Commission</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Chiesi Farmaceutici S.p.A.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The overall objective is to evaluate the long-term efficacy, safety and tolerability of&#xD;
      repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>January 2012</start_date>
  <completion_date type="Actual">September 2013</completion_date>
  <primary_completion_date type="Actual">August 2012</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Reduction of Oligosaccharides in blood serum</measure>
    <time_frame>6 months</time_frame>
    <description>Efficacy endpoint evaluation as change from baseline</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>The number of steps climbed in 3 minutes (3-minute stair climb)</measure>
    <time_frame>6 months</time_frame>
    <description>Efficacy endpoint evaluation as change from baseline</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Reduction of Oligosaccharides in CSF</measure>
    <time_frame>6 months</time_frame>
    <description>Efficacy endpoint evaluation as change from baseline</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The distance walked in 6 minutes (6-minute walk test)</measure>
    <time_frame>6 months</time_frame>
    <description>Efficacy endpoint evaluation as change from baseline</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pulmonary function</measure>
    <time_frame>6 months</time_frame>
    <description>Efficacy endpoint evaluation as change from baseline</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Adverse events</measure>
    <time_frame>1 week</time_frame>
    <description>Safety endpoint assesed weekly throughout the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Development of clinically significant changes in vital signs and change in physical examination</measure>
    <time_frame>1 week</time_frame>
    <description>Safety endpoint assesed weekly throughout the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)</measure>
    <time_frame>1 week</time_frame>
    <description>Safety endpoint assesed weekly throughout the trial</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies</measure>
    <time_frame>1 week</time_frame>
    <description>Safety endpoint assesed weekly throughout the trial</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">10</enrollment>
  <condition>Alpha-Mannosidosis</condition>
  <arm_group>
    <arm_group_label>Lamazym</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>1 mg/kg body weight</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Lamazym</intervention_name>
    <description>ERT, i.v. infusions weekly</description>
    <arm_group_label>Lamazym</arm_group_label>
    <other_name>rhLAMAN</other_name>
    <other_name>recombinant human alpha-mannosidase</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  The subject must have participated in the phase 1 trial (EudraCT number:&#xD;
             2010-022084-36) and phase 2a trial (EudraCT number: 2010-022085-26)&#xD;
&#xD;
          -  Subject or subjects legally authorized guardian(s) must provide signed, informed&#xD;
             consent prior to performing any trial-related activities (trial-related activities are&#xD;
             any procedures that would not have been performed during normal management of the&#xD;
             subject)&#xD;
&#xD;
          -  The subject and his/her guardian(s) must have the ability to comply with the protocol&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  The subject cannot walk without support&#xD;
&#xD;
          -  Presence of known chromosomal abnormality and syndromes affecting psychomotor&#xD;
             development, other than alpha-Mannosidosis&#xD;
&#xD;
          -  History of bone marrow transplantation&#xD;
&#xD;
          -  Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal&#xD;
             disease or other medical conditions that, in the opinion of the Investigator, would&#xD;
             preclude participation in the trial&#xD;
&#xD;
          -  Any other medical condition or serious intercurrent illness, or extenuating&#xD;
             circumstance that, in the opinion of the investigator, would preclude participation in&#xD;
             the trial.&#xD;
&#xD;
          -  Pregnancy: Before the start of the treatment the investigators will decide whether or&#xD;
             not there is a need for contraception. This assessment will be done through interviews&#xD;
             with the patient and parents. The evaluation will be done continuously during the&#xD;
             study&#xD;
&#xD;
          -  Psychosis within the last 3 months&#xD;
&#xD;
          -  Planned major surgery that, in the opinion of the investigator, would preclude&#xD;
             participation in the trial&#xD;
&#xD;
          -  Participation in other interventional trials testing IMP except for studies with&#xD;
             Lamazym&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>5 Years</minimum_age>
    <maximum_age>21 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Allan M Lund, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Jens Fogh</last_name>
    <role>Study Chair</role>
    <affiliation>Zymenex A/S</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101</name>
      <address>
        <city>Brussel</city>
        <zip>1090</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9</name>
      <address>
        <city>Copenhagen</city>
        <zip>DK-2100</zip>
        <country>Denmark</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Servicio de Pediatría, Hospital Materno Infantil, Reina Sofía, Avda Menéndez Pidal sn</name>
      <address>
        <city>Córdoba</city>
        <zip>14004</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,</name>
      <address>
        <city>Manchester</city>
        <zip>M13 9WL</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Belgium</country>
    <country>Denmark</country>
    <country>Spain</country>
    <country>United Kingdom</country>
  </location_countries>
  <link>
    <url>https://www.clinicaltrialsregister.eu/ctr-search/search?query=2011-004355-40</url>
    <description>Study Record on EU Clinical Trials Register including results</description>
  </link>
  <verification_date>March 2017</verification_date>
  <study_first_submitted>August 22, 2012</study_first_submitted>
  <study_first_submitted_qc>September 5, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 10, 2012</study_first_posted>
  <last_update_submitted>March 28, 2017</last_update_submitted>
  <last_update_submitted_qc>March 28, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">March 29, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Mannosidase Deficiency Diseases</mesh_term>
    <mesh_term>alpha-Mannosidosis</mesh_term>
  </condition_browse>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT01681940
org study id: rhLAMAN-04
secondary id: 2011-004355-40
nct id: NCT01681940
lead sponsor: 
collaborator: 
has dmc: No
The overall objective is to evaluate the long-term efficacy, safety and tolerability of
      repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Reduction of Oligosaccharides in blood serum
time frame: 6 months
description: Efficacy endpoint evaluation as change from baseline
measure: The number of steps climbed in 3 minutes (3-minute stair climb)
time frame: 6 months
description: Efficacy endpoint evaluation as change from baseline
measure: Reduction of Oligosaccharides in CSF
time frame: 6 months
description: Efficacy endpoint evaluation as change from baseline
measure: The distance walked in 6 minutes (6-minute walk test)
time frame: 6 months
description: Efficacy endpoint evaluation as change from baseline
measure: Pulmonary function
time frame: 6 months
description: Efficacy endpoint evaluation as change from baseline
measure: Adverse events
time frame: 1 week
description: Safety endpoint assesed weekly throughout the trial
measure: Development of clinically significant changes in vital signs and change in physical examination
time frame: 1 week
description: Safety endpoint assesed weekly throughout the trial
measure: Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)
time frame: 1 week
description: Safety endpoint assesed weekly throughout the trial
measure: Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies
time frame: 1 week
description: Safety endpoint assesed weekly throughout the trial
arm group label: Lamazym
arm group type: Experimental
description: 1 mg/kg body weight
intervention type: Drug
intervention name: Lamazym
description: ERT, i.v. infusions weekly
arm group label: Lamazym
other name: rhLAMAN
other name: recombinant human alpha-mannosidase
criteria: 
gender: All
minimum age: 5 Years
maximum age: 21 Years
healthy volunteers: No
last name: Allan M Lund, MD
role: Principal Investigator
affiliation: Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics
last name: Jens Fogh
role: Study Chair
affiliation: Zymenex A/S
facility: 
facility: 
facility: 
facility: 
country: Belgium
country: Denmark
country: Spain
country: United Kingdom
url: https://www.clinicaltrialsregister.eu/ctr-search/search?query=2011-004355-40
description: Study Record on EU Clinical Trials Register including results
responsible party type: Sponsor
mesh term: Mannosidase Deficiency Diseases
mesh term: alpha-Mannosidosis