CACIPLIQ20 in Wound Healing in Subjects With Epidermolysis Bullosa
Status:
Recruiting
Trial end date:
2024-09-01
Target enrollment:
Participant gender:
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Preliminary results from several published and unpublished case reports (Al Malak and
Barritault, 2012; Bodemer, unpublished observations) suggest that CACIPLIQ20 is safe and can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.
Phase:
N/A
Details
Lead Sponsor:
Organ, Tissue, Regeneration, Repair and Replacement