CACIPLIQ20 in Wound Healing in Subjects With Epidermolysis Bullosa

NCT06007235 | Recruiting

• 1 other identifier: 2022-A01774-39
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief Summary

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 Preliminary results from several published and unpublished case reports (Al Malak and Barritault, 2012; Bodemer, unpublished observations) suggest that CACIPLIQ20 is safe and can improve wound healing and reduce pain in patients with epidermolysis bullosa.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.

Conditions

Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa, Junctional

Outcome Measures

Primary Outcomes (1)

• Change in total lesioned skin surface

  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".

  After 1 month of treatment

Secondary Outcomes (12)

• The rate of device related serious adverse events

  Up to 4 months

• The rate of device related adverse events

  Up to 4 months

• The rate of procedure related adverse events

  Up to 4 months

• The rate of procedure related serious adverse events

  Up to 4 months

• The rate of all adverse events (AEs).

  Up to 4 months

Study Arms (2)

A: CACIPLIQ20 before placebo

OTHER

Arm A will receive at first CACIPLIQ20 (experimental product) for 1 month, followed by 1-month washout, then 1 month of saline (placebo comparator).

Device: CACIPLIQ20; Drug: Placebo

B: placebo before CACIPLIQ20

OTHER

Arm B will receive at first saline (placebo comparator) for 1 month, followed by 1-month washout, then 1 month of CACIPLIQ20 (experimental product).

Device: CACIPLIQ20; Drug: Placebo

Interventions

CACIPLIQ20 DEVICE

CACIPLIQ20 contains RGTA heparan sulphate mimetics

A: CACIPLIQ20 before placebo; B: placebo before CACIPLIQ20

Placebo DRUG

Saline solution in identical spray bottles

A: CACIPLIQ20 before placebo; B: placebo before CACIPLIQ20

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of Dystrophic or Junctional EB.
• years ≤ age ≤ 18 years
• Informed consent form signed by the patient's legal representative; if the patient is minor but capable of providing consent, additional signed consent from the patient.
• Patient and caregiver must be willing to comply with all protocol requirements.

You may not qualify if:

• Use of any investigational drug within the last 30 days before enrolment.
• Current or former malignancy.
• Pregnancy or breastfeeding during the study.
• Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception.
• Use of CACIPLIQ20 within the last 30 days before enrolment.
• Patients intolerant to one of the study device components or to heparinoids.

Sponsors & Collaborators

• Organ, Tissue, Regeneration, Repair and Replacement: lead
• Euraxi Pharma: collaborator
• ARANZ Medical: collaborator

Study Sites (1)

Hopital Necker - enfants malades

Paris, 75000, France

RECRUITING

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa, Junctional

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Study Officials

• Christine Bodemer, MD, PhD

  Hopital Necker - enfants malades

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Frederic Sedel, MD, PhD

CONTACT

+33 (0)1 83627895; frederic.sedel@otr3.com

Martin Inizan

CONTACT

martin.inizan@otr3.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Blinding codes on treatments
• Purpose: SUPPORTIVE CARE
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2023
• First Posted: August 23, 2023
• Study Start: January 29, 2024
• Primary Completion: September 1, 2024
• Study Completion: September 1, 2024
• Last Updated: February 13, 2024

Record last verified: 2024-02

Locations

FR (1)