xml: <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/NCT06007235</url>
    </required_header>
    <id_info>
      <org_study_id>2022-A01774-39</org_study_id>
      <nct_id>NCT06007235</nct_id>
    </id_info>
    <brief_title>CACIPLIQ20 in Wound Healing in Subjects With Epidermolysis Bullosa</brief_title>
    <acronym>MATHBULL</acronym>
    <official_title>A Randomized, Double-Blind, Placebo-Controlled, Crossover, Pilot Stage Clinical Trial Evaluating CACIPLIQ20 in Wound Healing in Subjects With Epidermolysis Bullosa</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Organ, Tissue, Regeneration, Repair and Replacement</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
      <collaborator>
        <agency>Euraxi Pharma</agency>
        <agency_class>Industry</agency_class>
      </collaborator>
      <collaborator>
        <agency>ARANZ Medical</agency>
        <agency_class>Other</agency_class>
      </collaborator>
    </sponsors>
    <source>Organ, Tissue, Regeneration, Repair and Replacement</source>
    <oversight_info>
      <has_dmc>No</has_dmc>
      <is_fda_regulated_drug>No</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        Epidermolysis bullosa (EB) is a group of inherited disorders characterized by fragility of&#xD;
        the skin and mucous membranes within the basement membrane zone. It is characterized by&#xD;
        moderate to excessive fragility of epithelial tissues with prototypic blistering or erosions&#xD;
        following minimal trauma (mechanobullous dermatoses). The chronic pain associated with EB,&#xD;
        the hardship placed on caregivers, and the high risk for complications places a considerable&#xD;
        psychosocial burden on both patients and their families. Despite considerable research to&#xD;
        advance the understanding of EB pathophysiology, no treatments have been approved by&#xD;
        regulatory authorities to date.&#xD;
  &#xD;
        Heparan sulfates are key elements of the Extra Cellular Matrix scaffold which act both as&#xD;
        linkers, bridging structural matrix proteins such as collagens, laminin and as storage and&#xD;
        protector sites to communication peptides, playing a pivotal role in the regulation of cell&#xD;
        proliferation, migration and differentiation that are all required for tissue regeneration&#xD;
        and repair. CACIPLIQ20 is a bioengineered structural analogue of heparan sulfate&#xD;
        glycosaminoglycans. Numerous experimental studies have provided strong evidence that&#xD;
        CACIPLIQ20 promotes tissue regeneration by reconstructing the cellular microenvironment&#xD;
        following tissue injury. CACIPLIQ20 is currently a class III CE marked medical device&#xD;
        (NSAI-0050 CE MARK ECDECNL-A4 (6) and EC Annex II of the directive. NL-A4 (7)) with the&#xD;
        following indications: Chronic ulcers showing no tendency to heal after 6 months of standard&#xD;
        care, or still unhealed after 12 months:&#xD;
  &#xD;
          -  Pressure ulcers.&#xD;
  &#xD;
          -  Peripheral arterial disease (such as Stage IV Leriche &amp; Fontaine) ulcers.&#xD;
  &#xD;
          -  Diabetic ulcers (including amputation).&#xD;
  &#xD;
        Preliminary results from several published and unpublished case reports (Al Malak and&#xD;
        Barritault, 2012; Bodemer, unpublished observations) suggest that CACIPLIQ20 is safe and can&#xD;
        improve wound healing and reduce pain in patients with epidermolysis bullosa.&#xD;
  &#xD;
        The goal of the MATHBULL study is to confirm preliminary observations in a placebo-controlled&#xD;
        double-blind pilot study. The results of this pilot study will help to design a pivotal&#xD;
        study.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        The patients are outpatients at inclusion and may be already followed by the investigators,&#xD;
        and they have not yet been treated with CACIPLIQ20 for at least one month.&#xD;
  &#xD;
        These patients will be randomly allocated (1:1 ratio) to Arm A and B. Every patient will have&#xD;
        two treatment periods. Arm A will receive at first CACIPLIQ20 for 1 month, followed by&#xD;
        1-month washout then 1-month Placebo, and the reverse sequence (Placebo followed by&#xD;
        CACIPLIQ20) in Arm B. The sponsor, investigator and patient will remain blinded to the&#xD;
        treatment Arm in which a patient will be randomized until the end of the study and database&#xD;
        lock.&#xD;
  &#xD;
        Two baselines will be defined before the two treatment periods, Baseline 1 at&#xD;
        inclusion/before treatment period 1, and Baseline 2 at the end of washout/before treatment&#xD;
        period 2.&#xD;
  &#xD;
        For each patient, all active wounds will be treated by CACIPLIQ20 or Placebo, which will be&#xD;
        added to the best standard of care decided by the participating physicians. Treatments will&#xD;
        be provided in indistinguishable spray bottles, labelled with treatment numbers using a&#xD;
        randomization list provided by an independent statistician. CACIPLIQ20 or Placebo will be&#xD;
        applied every 3 to 4 days for 30 days. Patients will be followed every month according to&#xD;
        investigators' standard practices. At least five visits will be programmed during the study,&#xD;
        including Inclusion Visit, End of Treatment Period 1 Visit at Month 1, End of Washout Visit&#xD;
        at Month 2, End of Treatment Period 2 Visit at Month 3, End of Study Visit at Month 4. In&#xD;
        addition to these visits, two phone calls will be organized 15 days after the start of each&#xD;
        treatment period to identify potential adverse events.&#xD;
  &#xD;
        At each visit, the investigator will take pictures of treated wounds with an imaging device&#xD;
        (Silhouette®), in order to assess the total lesioned skin surface.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">October 2023</start_date>
    <completion_date type="Anticipated">September 2024</completion_date>
    <primary_completion_date type="Anticipated">September 2024</primary_completion_date>
    <phase>N/A</phase>
    <study_type>Interventional</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <allocation>Randomized</allocation>
      <intervention_model>Crossover Assignment</intervention_model>
      <primary_purpose>Supportive Care</primary_purpose>
      <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
      <masking_description>Blinding codes on treatments</masking_description>
    </study_design_info>
    <primary_outcome>
      <measure>Change in total lesioned skin surface</measure>
      <time_frame>After 1 month of treatment</time_frame>
      <description>Change in total lesioned skin surface from baseline* to the end of the CACIPLIQ20 vs Placebo treatment periods&#xD;
  * Baseline 1 at inclusion/before treatment period 1, and Baseline 2 at the end of washout/before treatment period 2 Change in total lesioned skin surface at the end of each treatment period will be assessed blindly regarding treatment allocation, using the SilhouetteConnect™ software in reference to &quot;Baseline 1&quot; and &quot;Baseline 2&quot;.</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>The rate of device related serious adverse events</measure>
      <time_frame>Up to 4 months</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>The rate of device related adverse events</measure>
      <time_frame>Up to 4 months</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>The rate of procedure related adverse events</measure>
      <time_frame>Up to 4 months</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>The rate of procedure related serious adverse events</measure>
      <time_frame>Up to 4 months</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>The rate of all adverse events (AEs).</measure>
      <time_frame>Up to 4 months</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>The rate of all serious adverse events (SAE)</measure>
      <time_frame>Up to 4 months</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Proportion of wounds achieving complete closure at the end of the CACIPLIQ20 versus Placebo treatment periods</measure>
      <time_frame>After 1 month of treatment</time_frame>
      <description>This parameter will be assessed with the ARANZ Silhouette® system by comparing wounds areas before and after each treatment period.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Mean CGI at the end of the CACIPLIQ20 versus Placebo treatment periods</measure>
      <time_frame>After 1 month of treatment</time_frame>
      <description>The CGI (clinical assessment) will be rated on the following sevenpoint scale:&#xD;
  = Very much improved-nearly all better; good level of functioning; minimal symptoms; represents a very substantial change&#xD;
  = Much improved-notably better with significant reduction of symptoms; increase in the level of functioning but some symptoms remain&#xD;
  = Minimally improved-slightly better with little or no clinically meaningful reduction of symptoms. Represents very little change in basic clinical status, level of care, or functional capacity&#xD;
  = No change-symptoms remain essentially unchanged&#xD;
  = Minimally worse-slightly worse but may not be clinically meaningful; may represent very little change in basic clinical status or functional capacity&#xD;
  = Much worse-clinically significant increase in symptoms and diminished functioning&#xD;
  = Very much worse-severe exacerbation of symptoms and loss of functioning</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Mean SGI at the end of the CACIPLIQ20 versus Placebo treatment periods</measure>
      <time_frame>After 1 month of treatment</time_frame>
      <description>The SGI (subject/family assessment) will be rated on the following sevenpoint scale:&#xD;
  = Very much improved-nearly all better; good level of functioning; minimal symptoms; represents a very substantial change&#xD;
  = Much improved-notably better with significant reduction of symptoms; increase in the level of functioning but some symptoms remain&#xD;
  = Minimally improved-slightly better with little or no clinically meaningful reduction of symptoms. Represents very little change in basic clinical status, level of care, or functional capacity&#xD;
  = No change-symptoms remain essentially unchanged&#xD;
  = Minimally worse-slightly worse but may not be clinically meaningful; may represent very little change in basic clinical status or functional capacity&#xD;
  = Much worse-clinically significant increase in symptoms and diminished functioning&#xD;
  = Very much worse-severe exacerbation of symptoms and loss of functioning</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in pain assessed from baseline to the end of the CACIPLIQ20 versus Placebo treatment periods (for patients from 3 to 4 years of age)</measure>
      <time_frame>After 1 month of treatment</time_frame>
      <description>Change in pain will be assessed at the end of the one-month treatment period 1 and 2 compared to baseline 1 and baseline 2, respectively, using the &quot;FLACC scale&quot; for patients from 3 to 4 years of age.&#xD;
  FLACC: Face Legs Activity Cry Consolability. Score range for the FLACC scale is between 0-10 where higher score represents more pain.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in pain assessed from baseline to the end of the CACIPLIQ20 versus Placebo treatment periods (for patients 4 years of age and older)</measure>
      <time_frame>After 1 month of treatment</time_frame>
      <description>Change in pain will be assessed at the end of the one-month treatment period 1 and 2 compared to baseline 1 and baseline 2, respectively, using the &quot;Wong Faces Pain Scale&quot; for patients 4 years of age and older.&#xD;
  The Wong Faces Pain Scale rates pain on a 0-10 scale, where higher score represents more pain.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in itching assessed from baseline to the end of the CACIPLIQ20 versus Placebo treatment periods</measure>
      <time_frame>After 1 month of treatment</time_frame>
      <description>Change in itching will be assessed at the end of one-month treatment period 1 and 2 with eference to baseline 1 and baseline 2, respectively. Itching will be measured using the &quot;Itch Man Pruritus Assessment Tool&quot;. In patients from 3 to 6 years of age, assessments will be performed by caretakers whereas patients who are 6 years of age and older will self-report their itching using the &quot;Itch Man Pruritus Assessment Tool&quot;.&#xD;
  Itching assessments based on the following scores: 0=Comfortable, no itch; 1=itches a little, does not interfere with activity; 2=itches more, sometimes interferes with activity; 3=itches a lot, difficult to be still, concentrate; 4=itches most terribly, impossible to sit still or concentrate.</description>
    </secondary_outcome>
    <number_of_arms>2</number_of_arms>
    <enrollment type="Anticipated">16</enrollment>
    <condition>Epidermolysis Bullosa Dystrophica</condition>
    <condition>Epidermolysis Bullosa, Junctional</condition>
    <arm_group>
      <arm_group_label>A: CACIPLIQ20 before placebo</arm_group_label>
      <arm_group_type>Other</arm_group_type>
      <description>Arm A will receive at first CACIPLIQ20 (experimental product) for 1 month, followed by 1-month washout, then 1 month of saline (placebo comparator).</description>
    </arm_group>
    <arm_group>
      <arm_group_label>B: placebo before CACIPLIQ20</arm_group_label>
      <arm_group_type>Other</arm_group_type>
      <description>Arm B will receive at first saline (placebo comparator) for 1 month, followed by 1-month washout, then 1 month of CACIPLIQ20 (experimental product).</description>
    </arm_group>
    <intervention>
      <intervention_type>Device</intervention_type>
      <intervention_name>CACIPLIQ20</intervention_name>
      <description>CACIPLIQ20 contains RGTA heparan sulphate mimetics</description>
      <arm_group_label>A: CACIPLIQ20 before placebo</arm_group_label>
      <arm_group_label>B: placebo before CACIPLIQ20</arm_group_label>
    </intervention>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Placebo</intervention_name>
      <description>Saline solution in identical spray bottles</description>
      <arm_group_label>A: CACIPLIQ20 before placebo</arm_group_label>
      <arm_group_label>B: placebo before CACIPLIQ20</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Diagnosis of Dystrophic or Junctional EB.&#xD;
  &#xD;
            -  3 years ≤ age ≤ 18 years&#xD;
  &#xD;
            -  At least one active wound at inclusion&#xD;
  &#xD;
            -  Informed consent form signed by the patient's legal representative; if the patient is&#xD;
               minor but capable of providing consent, additional signed consent from the patient.&#xD;
  &#xD;
            -  Patient and caregiver must be willing to comply with all protocol requirements.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Use of any investigational drug within the last 30 days before enrolment.&#xD;
  &#xD;
            -  Current or former malignancy.&#xD;
  &#xD;
            -  Pregnancy or breastfeeding during the study.&#xD;
  &#xD;
            -  Females of childbearing potential who are not abstinent and not practicing a medically&#xD;
               acceptable method of contraception.&#xD;
  &#xD;
            -  Use of CACIPLIQ20 within the last 30 days before enrolment.&#xD;
  &#xD;
            -  Patients intolerant to one of the study device components or to heparinoids.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>3 Years</minimum_age>
      <maximum_age>18 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Christine Bodemer, MD, PhD</last_name>
      <role>Principal Investigator</role>
      <affiliation>Hopital Necker - enfants malades</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Frederic Sedel, MD, PhD</last_name>
      <phone>+33 (0)1 83627895</phone>
      <email>frederic.sedel@otr3.com</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Martin Inizan</last_name>
      <email>martin.inizan@otr3.com</email>
    </overall_contact_backup>
    <location>
      <facility>
        <name>Hopital Necker - enfants malades</name>
        <address>
          <city>Paris</city>
          <zip>75000</zip>
          <country>France</country>
        </address>
      </facility>
      <contact>
        <last_name>Christine Bodemer, MD, PhD</last_name>
      </contact>
    </location>
    <location_countries>
      <country>France</country>
    </location_countries>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 7, 2023</study_first_submitted>
    <study_first_submitted_qc>August 17, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 23, 2023</study_first_posted>
    <last_update_submitted>August 17, 2023</last_update_submitted>
    <last_update_submitted_qc>August 17, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 23, 2023</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>Epidermolysis Bullosa</mesh_term>
      <mesh_term>Epidermolysis Bullosa Dystrophica</mesh_term>
      <mesh_term>Epidermolysis Bullosa, Junctional</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>Undecided</sharing_ipd>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT06007235
  org study id: 2022-A01774-39
  nct id: NCT06007235
  lead sponsor: 
  collaborator: 
  collaborator: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  Epidermolysis bullosa (EB) is a group of inherited disorders characterized by fragility of
        the skin and mucous membranes within the basement membrane zone. It is characterized by
        moderate to excessive fragility of epithelial tissues with prototypic blistering or erosions
        following minimal trauma (mechanobullous dermatoses). The chronic pain associated with EB,
        the hardship placed on caregivers, and the high risk for complications places a considerable
        psychosocial burden on both patients and their families. Despite considerable research to
        advance the understanding of EB pathophysiology, no treatments have been approved by
        regulatory authorities to date.
  
        Heparan sulfates are key elements of the Extra Cellular Matrix scaffold which act both as
        linkers, bridging structural matrix proteins such as collagens, laminin and as storage and
        protector sites to communication peptides, playing a pivotal role in the regulation of cell
        proliferation, migration and differentiation that are all required for tissue regeneration
        and repair. CACIPLIQ20 is a bioengineered structural analogue of heparan sulfate
        glycosaminoglycans. Numerous experimental studies have provided strong evidence that
        CACIPLIQ20 promotes tissue regeneration by reconstructing the cellular microenvironment
        following tissue injury. CACIPLIQ20 is currently a class III CE marked medical device
        (NSAI-0050 CE MARK ECDECNL-A4 (6) and EC Annex II of the directive. NL-A4 (7)) with the
        following indications: Chronic ulcers showing no tendency to heal after 6 months of standard
        care, or still unhealed after 12 months:
  
          -  Pressure ulcers.
  
          -  Peripheral arterial disease (such as Stage IV Leriche & Fontaine) ulcers.
  
          -  Diabetic ulcers (including amputation).
  
        Preliminary results from several published and unpublished case reports (Al Malak and
        Barritault, 2012; Bodemer, unpublished observations) suggest that CACIPLIQ20 is safe and can
        improve wound healing and reduce pain in patients with epidermolysis bullosa.
  
        The goal of the MATHBULL study is to confirm preliminary observations in a placebo-controlled
        double-blind pilot study. The results of this pilot study will help to design a pivotal
        study.
  The patients are outpatients at inclusion and may be already followed by the investigators,
        and they have not yet been treated with CACIPLIQ20 for at least one month.
  
        These patients will be randomly allocated (1:1 ratio) to Arm A and B. Every patient will have
        two treatment periods. Arm A will receive at first CACIPLIQ20 for 1 month, followed by
        1-month washout then 1-month Placebo, and the reverse sequence (Placebo followed by
        CACIPLIQ20) in Arm B. The sponsor, investigator and patient will remain blinded to the
        treatment Arm in which a patient will be randomized until the end of the study and database
        lock.
  
        Two baselines will be defined before the two treatment periods, Baseline 1 at
        inclusion/before treatment period 1, and Baseline 2 at the end of washout/before treatment
        period 2.
  
        For each patient, all active wounds will be treated by CACIPLIQ20 or Placebo, which will be
        added to the best standard of care decided by the participating physicians. Treatments will
        be provided in indistinguishable spray bottles, labelled with treatment numbers using a
        randomization list provided by an independent statistician. CACIPLIQ20 or Placebo will be
        applied every 3 to 4 days for 30 days. Patients will be followed every month according to
        investigators' standard practices. At least five visits will be programmed during the study,
        including Inclusion Visit, End of Treatment Period 1 Visit at Month 1, End of Washout Visit
        at Month 2, End of Treatment Period 2 Visit at Month 3, End of Study Visit at Month 4. In
        addition to these visits, two phone calls will be organized 15 days after the start of each
        treatment period to identify potential adverse events.
  
        At each visit, the investigator will take pictures of treated wounds with an imaging device
        (Silhouette®), in order to assess the total lesioned skin surface.
  allocation: Randomized
  intervention model: Crossover Assignment
  primary purpose: Supportive Care
  masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  masking description: Blinding codes on treatments
  measure: Change in total lesioned skin surface
  time frame: After 1 month of treatment
  description: Change in total lesioned skin surface from baseline* to the end of the CACIPLIQ20 vs Placebo treatment periods
  * Baseline 1 at inclusion/before treatment period 1, and Baseline 2 at the end of washout/before treatment period 2 Change in total lesioned skin surface at the end of each treatment period will be assessed blindly regarding treatment allocation, using the SilhouetteConnect™ software in reference to "Baseline 1" and "Baseline 2".
  measure: The rate of device related serious adverse events
  time frame: Up to 4 months
  measure: The rate of device related adverse events
  time frame: Up to 4 months
  measure: The rate of procedure related adverse events
  time frame: Up to 4 months
  measure: The rate of procedure related serious adverse events
  time frame: Up to 4 months
  measure: The rate of all adverse events (AEs).
  time frame: Up to 4 months
  measure: The rate of all serious adverse events (SAE)
  time frame: Up to 4 months
  measure: Proportion of wounds achieving complete closure at the end of the CACIPLIQ20 versus Placebo treatment periods
  time frame: After 1 month of treatment
  description: This parameter will be assessed with the ARANZ Silhouette® system by comparing wounds areas before and after each treatment period.
  measure: Mean CGI at the end of the CACIPLIQ20 versus Placebo treatment periods
  time frame: After 1 month of treatment
  description: The CGI (clinical assessment) will be rated on the following sevenpoint scale:
  = Very much improved-nearly all better; good level of functioning; minimal symptoms; represents a very substantial change
  = Much improved-notably better with significant reduction of symptoms; increase in the level of functioning but some symptoms remain
  = Minimally improved-slightly better with little or no clinically meaningful reduction of symptoms. Represents very little change in basic clinical status, level of care, or functional capacity
  = No change-symptoms remain essentially unchanged
  = Minimally worse-slightly worse but may not be clinically meaningful; may represent very little change in basic clinical status or functional capacity
  = Much worse-clinically significant increase in symptoms and diminished functioning
  = Very much worse-severe exacerbation of symptoms and loss of functioning
  measure: Mean SGI at the end of the CACIPLIQ20 versus Placebo treatment periods
  time frame: After 1 month of treatment
  description: The SGI (subject/family assessment) will be rated on the following sevenpoint scale:
  = Very much improved-nearly all better; good level of functioning; minimal symptoms; represents a very substantial change
  = Much improved-notably better with significant reduction of symptoms; increase in the level of functioning but some symptoms remain
  = Minimally improved-slightly better with little or no clinically meaningful reduction of symptoms. Represents very little change in basic clinical status, level of care, or functional capacity
  = No change-symptoms remain essentially unchanged
  = Minimally worse-slightly worse but may not be clinically meaningful; may represent very little change in basic clinical status or functional capacity
  = Much worse-clinically significant increase in symptoms and diminished functioning
  = Very much worse-severe exacerbation of symptoms and loss of functioning
  measure: Change in pain assessed from baseline to the end of the CACIPLIQ20 versus Placebo treatment periods (for patients from 3 to 4 years of age)
  time frame: After 1 month of treatment
  description: Change in pain will be assessed at the end of the one-month treatment period 1 and 2 compared to baseline 1 and baseline 2, respectively, using the "FLACC scale" for patients from 3 to 4 years of age.
  FLACC: Face Legs Activity Cry Consolability. Score range for the FLACC scale is between 0-10 where higher score represents more pain.
  measure: Change in pain assessed from baseline to the end of the CACIPLIQ20 versus Placebo treatment periods (for patients 4 years of age and older)
  time frame: After 1 month of treatment
  description: Change in pain will be assessed at the end of the one-month treatment period 1 and 2 compared to baseline 1 and baseline 2, respectively, using the "Wong Faces Pain Scale" for patients 4 years of age and older.
  The Wong Faces Pain Scale rates pain on a 0-10 scale, where higher score represents more pain.
  measure: Change in itching assessed from baseline to the end of the CACIPLIQ20 versus Placebo treatment periods
  time frame: After 1 month of treatment
  description: Change in itching will be assessed at the end of one-month treatment period 1 and 2 with eference to baseline 1 and baseline 2, respectively. Itching will be measured using the "Itch Man Pruritus Assessment Tool". In patients from 3 to 6 years of age, assessments will be performed by caretakers whereas patients who are 6 years of age and older will self-report their itching using the "Itch Man Pruritus Assessment Tool".
  Itching assessments based on the following scores: 0=Comfortable, no itch; 1=itches a little, does not interfere with activity; 2=itches more, sometimes interferes with activity; 3=itches a lot, difficult to be still, concentrate; 4=itches most terribly, impossible to sit still or concentrate.
  arm group label: A: CACIPLIQ20 before placebo
  arm group type: Other
  description: Arm A will receive at first CACIPLIQ20 (experimental product) for 1 month, followed by 1-month washout, then 1 month of saline (placebo comparator).
  arm group label: B: placebo before CACIPLIQ20
  arm group type: Other
  description: Arm B will receive at first saline (placebo comparator) for 1 month, followed by 1-month washout, then 1 month of CACIPLIQ20 (experimental product).
  intervention type: Device
  intervention name: CACIPLIQ20
  description: CACIPLIQ20 contains RGTA heparan sulphate mimetics
  arm group label: A: CACIPLIQ20 before placebo
  arm group label: B: placebo before CACIPLIQ20
  intervention type: Drug
  intervention name: Placebo
  description: Saline solution in identical spray bottles
  arm group label: A: CACIPLIQ20 before placebo
  arm group label: B: placebo before CACIPLIQ20
  criteria: 
  gender: All
  minimum age: 3 Years
  maximum age: 18 Years
  healthy volunteers: No
  last name: Christine Bodemer, MD, PhD
  role: Principal Investigator
  affiliation: Hopital Necker - enfants malades
  last name: Frederic Sedel, MD, PhD
  phone: +33 (0)1 83627895
  email: frederic.sedel@otr3.com
  last name: Martin Inizan
  email: martin.inizan@otr3.com
  facility: 
  contact: 
  country: France
  responsible party type: Sponsor
  mesh term: Epidermolysis Bullosa
  mesh term: Epidermolysis Bullosa Dystrophica
  mesh term: Epidermolysis Bullosa, Junctional
  sharing ipd: Undecided