Authorised Phase I and Phase II (Integrated)- First administration to humans Recessive dystrophic epidermolysis bullosa (RDEB)

Phase I/II ex vivo gene therapy clinical trial for RDEB using autologous skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector

EU CTIS ID: 2024-518368-10-00

What this study is testing

The primary objective of this phase I/II clinical trial is to evaluate the safety of grafting SIN RV-mediated COL7A1 gene-modified autologous SE in adults with RDEB at M1, M2, M3, M6, M12 after grafting. This evaluation will be continued during a reglementary 5-year follow up at M18, M24, M30, M36, M42, M48, M54, and M60.

Phase I and Phase II (Integrated)- First administration to humans

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

1.≥ 18 year-old
2.Clinical and molecular diagnosis of RDEB with confirmed bi-allelic COL7A1 mutations
3.Significantly reduced staining of C7 on skin biopsy, measured by immunofluorescence microscopy (IF)
4.A reduced number of or morphologically abnormal anchoring fibrils confirmed by TEM
5.Presence of non-collagenous-1 domain (NC-1) of C7 on skin biopsy, measured by immunofluorescence microscopy (IF) and/or Western blot analysis
6.Presence of ≥100cm2 of blistered and/or erosive skin areas including chronic wounds suitable for skin grafting

You likely can't join if

1.Recipients of other investigational medicinal products within 6 months prior to enrolment into this study
2.Past medical history of biopsy proven skin malignancy
3.Immunotherapy including oral corticosteroids (Prednisolone >1mg/kg) for more than one week (intranasal and topical preparations are permitted) or chemotherapy within 60 days of enrolment into this study
4.Known allergy to any of the constituents of the investigational medicinal product (IMP) including Penicillin
5.Subjects with BOTH: •positive serum antibodies to C7 confirmed by ELISA and •positive IIF with binding to the base of salt split skin and/or •positive Western blot
6.Positive results for HIV, Hepatitis BsAg, Hepatitis BcAb, Hepatitis C IgG, HTLV1&2 or Syphilis serology

See the full eligibility criteria

Who can join

1.≥ 18 year-old
2.Clinical and molecular diagnosis of RDEB with confirmed bi-allelic COL7A1 mutations
3.Significantly reduced staining of C7 on skin biopsy, measured by immunofluorescence microscopy (IF)
4.A reduced number of or morphologically abnormal anchoring fibrils confirmed by TEM
5.Presence of non-collagenous-1 domain (NC-1) of C7 on skin biopsy, measured by immunofluorescence microscopy (IF) and/or Western blot analysis
6.Presence of ≥100cm2 of blistered and/or erosive skin areas including chronic wounds suitable for skin grafting
7.Ability to undergo anaesthesia for grafting procedures
8.Subjects aged ≥ 18years, willing and able to give informed consent

What rules you out

1.Recipients of other investigational medicinal products within 6 months prior to enrolment into this study
2.Past medical history of biopsy proven skin malignancy
3.Immunotherapy including oral corticosteroids (Prednisolone >1mg/kg) for more than one week (intranasal and topical preparations are permitted) or chemotherapy within 60 days of enrolment into this study
4.Known allergy to any of the constituents of the investigational medicinal product (IMP) including Penicillin
5.Subjects with BOTH: •positive serum antibodies to C7 confirmed by ELISA and •positive IIF with binding to the base of salt split skin and/or •positive Western blot
6.Positive results for HIV, Hepatitis BsAg, Hepatitis BcAb, Hepatitis C IgG, HTLV1&2 or Syphilis serology
7.Clinically significant medical, psychological or laboratory abnormalities limiting the ability of the subject to travel to the trial site(s) and to undergo grafting and follow-up procedures, as determined by the Investigator
8.Absence of adequate social support
9.Subjects who are pregnant, breast-feeding or of child-bearing potential who are neither abstinent nor practicing an acceptable means of contraception when this is in line with the usual and preferred lifestyle of the subject, as determined by the Investigator, for the duration of the trial

The study team makes the final eligibility decision.

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.