Investigation of cell therapy for recessive dystrophic epidermolysis bullosa (RDEB) in Australia through intradermal injection of allogeneic fibroblasts.
In adult patients with recessive dystrophic epidermolysis bullosa (RDEB), are intradermal allogeneic cultured fibroblast wound injections in transport solution (Plasmalyte and 2% albumex) more effective than a placebo in promoting wound healing and collagen production?
Research Infrastructure Support Services (RISS)
5 participants
Oct 14, 2008
Interventional
Conditions
Summary
This is a randomized, single-institution double-blind, placebo-controlled clinical trial. Enrolled patients with a diagnosis of RDEB-GS will be randomized to receive either cell therapy with intradermal injection of cultured allogeneic fibroblasts in transport media or placebo (i.e. transport media alone) on two to eight symmetrical wounds on each side of their body (arms, trunk, upper and lower legs). Up to 5 patients will be enrolled. All patients will undergo clinical examination, photography and skin punch biopsies for immunofluorescence and electron microscopy studies (if not previously done). The efficacy endpoint for the primary objective is median healing time of lesions for each treatment group (fibroblast-treated versus placebo). Secondary efficacy end points are percentage change in size, change in appearance score (VAS), pain score (VAS) and pruritus score (VAS) of wounds for each treatment group. The efficacy endpoint for the co-primary objective are quantitative change in expression of collagen VII on immunofluorescence mapping and change in electron microscopy ultrastructure (i.e. numbers and appearance of anchoring fibrils) after cell therapy.
Eligibility
Inclusion Criteria4
- Diagnosis of Recessive dystrophic epidermolysis bullosa- generalized severe (RDEB-GS) (patients should have detectable low levels of collagen VII expression and reduced / abnormal anchoring fibrils on electron microscopy).
- At least 2 symmetric wounds on each side of the body (arms, chest/abdomen, legs) of approximately the same size (at least 4 cm2 as measured accurately by the Visitrak wound measurement system [Smith & Nephew Healthcare Ltd., Hull]).
- Ability to sign informed consent, which indicates the investigational nature of this study.
- Age: 18 years or older
Exclusion Criteria6
- Clinical evidence of local infection of the targeted wound(s).
- Histopathologic evidence of malignancy (i.e. squamous cell carcinoma - SCC) of the targeted wound(s).
- Use of systemic or topical steroid therapy within 30 days before enrolment.
- Use of any investigational drug within the 30 days before enrolment.
- Patients considered by the investigators to be unreliable or have poor compliance
- Patients who are pregnant, or who suspect they may be pregnant at the time of the study and lactating women. Women of childbearing age should use effective contraception.
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Interventions
Patients will have paired ulcers randomly treated with intradermal injection of cultured allogeneic fibroblasts (5 x10^6 per cm2) in a plasmalyte and 2% albumex solution. This will be performed during the baseline visit. Each paired ulcer site (placebo and treatment) will be treated with plasmalyte and 2% albumex solution injections or fibroblasts in a plasmalyte and 2% albumex solution. These paired sites will be injected at the same time or shortly after one another during the baseline visit. At 6 months, if the patient chooses to have the treatment sites re-treated with fibroblast injections, then the opportunity will be provided for patients to have this performed.
Locations(1)
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ACTRN12610000760077