A Pilot Multicentric Open Study Assessing the Efficacy and Safety of NEMOlizumab in Patients ≥ 12yo With Dystrophic Epidermolysis Bullosa (NEMODE)

November 3, 2026 updated by: Centre Hospitalier Universitaire de Nice

Dystrophic epidermolysis bullosa (DEB) is an inherited mucocutaneous fragility disorder caused by pathogenic variants in COL7A1, leading to blistering, scarring, and significant morbidity. Pruritus is a major complication that substantially impairs patients' quality of life, yet effective treatment options remain limited. Nemolizumab, an interleukin-31 receptor alpha antagonist, inhibits IL-31 signaling, a pathway potentially involved in DEB-associated pruritus. Elevated IL-31 levels have been reported in patients with recessive DEB (RDEB) and correlated with pruritus severity, supporting its potential role as a therapeutic target. We hypothesize that nemolizumab may represent an effective and safe treatment option for pruritus in patients with DEB.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Not yet recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Epidermolysis bullosa (EB) is a heterogeneous group of rare inherited disorders characterized by skin and mucosal fragility leading to the formation of blisters, erosions, and scars. It is traditionally subdivided into four main subtypes: simplex, junctional, dystrophic (EBD), and Kindler. The dystrophic form (DEB) is associated with dominant or recessive pathogenic variants in the COL7A1 gene, which encodes type VII collagen, an essential component of the anchoring fibrils that ensure dermo-epidermal adhesion.

Available epidemiological data estimate the prevalence of hereditary EB at approximately 11 cases per million people in the United States, with an incidence of about 2.72 new cases per 1 million live births. DEB accounts for approximately 25% of EB cases and can present in autosomal dominant or recessive forms.

EBD is associated with significant morbidity, particularly due to chronic complications such as pruritus and fibrosis, which significantly impair patients' quality of life and independence. To date, there is no curative treatment. Management relies on a multidisciplinary approach that is primarily symptomatic. Recently, B-VEC, a topical gene therapy designed to restore COL7A1 expression in skin lesions to promote healing, has received regulatory approval. However, its accessibility is limited

Translated with DeepL.com (free version)

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
15

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • France
    • Fr
      • Nice, Fr, France, 06000
        • CHU de Nice
        • Sub-Investigator:
          • Thomas HUBICHE, Doctor
        • Contact:
    • France
      • Paris, France, France, 75015
      • Paris, France, France, 75018
      • Toulouse, France, France, 31059

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years to 99 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Subject male or female
  2. Age ≥ 12 years old
  3. Subject has recessive (Severe, Intermediate, Inversa or Pruriginosa) or dominant (Intermediate or Pruriginosa) dystrophic epidermolysis bullosa, confirmed by skin immunomapping and/or molecular analysis (one or 2 COL7A1 mutation(s)), according to the last EB classification 4) Subject has at least a baseline pruritus score ≥ 4/10.

5) Subject agrees not to use any topical therapies other than the investigator approved (VYJUVEK gel is accepted if ongoing at the time of the study, excepted on the left leg) 6) A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least 1 of the following conditions applies: 7) (a) Is not a Women of Childbearing Potential (WOCBP) OR 8) Is a WOCBP and using a contraceptive method that is highly effective (with a failure rate of <1% per year), preferably with low user dependency, during the study period until the end of her participation to the study. Effective method of birth control recommended by the Clinical Trials Facilitation and Coordination group are combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral or intravaginal or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral or injectable or implantable); intrauterine device; intrauterine hormone-releasing system; bilateral tubal occlusion 9) Subject is in good general health and free of any known disease state or physical condition which, in the investigator's opinion, might impair his evaluation or which exposes the subject to an unacceptable risk by study participation.

10) Subject is willing and able to follow all study instructions and to attend all study visits.

11) Subject is able to comprehend and willing to sign an Informed Consent and/or Assent Form.

12) Affiliation with a social security scheme.

Exclusion Criteria:

  1. Subjects who are treated with DUPILUMAB or JAK inhibitor. A wash out of 2 months is mandatory to be included.

  2. Subjects with uncontrolled asthma.

  3. Medical History and Concurrent Diseases:

    1. Any major surgery within 8 weeks prior to Day 1, or any planned surgery for the first 28 weeks of the study

    2. Has donated blood >500mLwithin 4 weeks prior to Day1, or plans to donate blood during the course of the study

    3. Drug or alcohol abuse, as determined by the investigator, within 6 months prior to Day 1

    4. Medical marijuana or prescription marijuana taken for medicinal reasons

    5. Any major illness/condition or evidence of an unstable clinical condition (eg, renal, hepatic, hematologic, gastrointestinal, endocrine, pulmonary, psychiatric, neurologic, immunologic, or local active infection/infectious illness) that, in the investigator's judgment or after consultation with the Medical Monitor or designee, will substantially increase the risk to the participant if he or she participates in the study

    6. Unstable cardiovascular disease, defined as a recent clinical cardiovascular event (eg, unstable angina, myocardial infarction, stroke, rapid atrial fibrillation) in the last 3 months prior to screening, or a cardiac hospitalization (eg revascularization procedure, pacemaker implantation) within 3 months prior to screening, or patients at risk of major adverse cardiovascular events, deep venous thrombosis or pulmonary embolism.

    7. Has uncontrolled arterial hypertension characterized by a systolic blood pressure (BP) >160 mm Hg or diastolic BP >100 mm Hg.

      Note: Determined by 2 consecutive elevated readings. If an initial BP reading exceeds this limit, the BP may be repeated once after the participant has rested sitting for ≥10 minutes. If the repeat value is less than the criterion limits, the second value may be accepted.

    8. Class III or IV congestive heart failure by New York Heart Association Criteria

    9. Has cancer or history of cancer (solid organ or hematologic including myelodysplastic syndrome) or lymphoproliferative disease within the previous 5 years (other than resected cutaneous basal cell or squamous cell carcinoma, or carcinoma of cervix in situ that has been treated with no evidence of recurrence)

    10. Any significant/uncontrolled neuropsychiatric illness as clinically judged by the investigator during

  4. Females of child-bearing potential who meet the following criteria for pregnancy testing:

  5. Subjects with a positive serum pregnancy test at the Screening Visit or a positive urine pregnancy test at Baseline prior to the first dose of study treatment (local practices may require serum pregnancy testing at Baseline).

  6. Subjects with a borderline serum pregnancy test at Screening must have absence of clinical suspicion of pregnancy or other pathological causes of borderline results and a serum pregnancy test ≥ 3 days later to document continued lack of a positive result (unless inclusion of subjects with a borderline pregnancy test may be prohibited by local requirements).

  7. Subjects with a urine pregnancy test at Baseline that is borderline or ambiguous must have a serum pregnancy test performed. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.

  8. Female subjects of childbearing potential who are not able and/or willing to practice at least 1 protocol-specified method of birth control that is highly effective from Study Day 1 through at least 30 days after the last dose of study drug (local practices may require an additional method of contraception). Female subjects of non-childbearing potential do not need to use birth control.

  9. Female subjects who are pregnant, breastfeeding, or considering becoming pregnant or donating eggs during the study and for 30 days after the last dose of study drug.

  10. Subject has another member of her/his immediate family (i.e., living in the same household) enrolled in this study.

  11. Subject has used any systemic product that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study assessments within 30 days prior to Visit 1.

  12. Vulnerable persons as mentioned in articles L1121-5, L1121-6, L1121-7, L1121-8 and L1122-1-2 of the public health code.

  13. Subject has a history of sensitivity to any of the ingredients of Nemolizumab.

  14. Withdrawal of the informed consent.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Nemolizumab will be administered as a 60 mg loading dose (two 30 mg injections) at Day 0, followed b

Neolizumab
Drug: Neolizumab

Nemolizumab will be administered as a 60 mg loading dose (two 30 mg injections) at Day 0, followed by 30 mg subcutaneously every 4 weeks for 24 weeks,

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
To study the efficacy of Nemolizumab on pruritus in patients > 12yo with DEB after 4 weeks of treatment, in a multicentric, prospective, and open pilot study.
Time Frame: 4 weeks

The primary endpoint will be the proportion of patients achieving a clinically meaningful improvement in pruritus severity at Week 4 compared with baseline, defined as a reduction of at least 4 points on the Peak Pruritus Numerical Rating Scale (PP-NRS), which ranges from 0 (no itch) to 10 (worst itch imaginable).
4 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
To describe the evolution of the following efficacy and health outcomes measures after 24 weeks of treatment
Time Frame: at 24 weeks

pain (scale 0-10)
at 24 weeks
To describe the evolution of the following efficacy and health outcomes measures after 24 weeks of treatment
Time Frame: at baseline

pain (scale 0-10)
at baseline
To describe the evolution of the following efficacy and health outcomes measures after 24 weeks of treatment
Time Frame: at 24 weeks

quality of life (scale 0-30)
at 24 weeks
To describe the evolution of the following efficacy and health outcomes measures after 24 weeks of treatment
Time Frame: at baseline

quality of life (scale 0-30)
at baseline

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Centre Hospitalier Universitaire de Nice

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 31, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 3, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 3, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 9, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 9, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 3, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 26-PP-01
  • 2026-526417-28-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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