Mesenchymal stromal cell therapy for children with recessive dystrophic epidermolysis bullosa

Not Applicable

• Conditions: Congenital disorders of the skin, recessive dystrophic epidermolysis bullosa; Skin and Connective Tissue Diseases; Epidermolysis bullosa dystrophica

• Registration Number: ISRCTN14409785
• Lead Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-03-25
• Last Update Posted: 3 June 2024
• Study Completion: 2025-10-31

Recruitment & Eligibility

• Status: Ongoing
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

1. Diagnosis of RDEB characterised by partial or complete C7 deficiency including generalised severe and generalised intermediate subtypes
2. Aged >6 months and <16 years at time of enrolment
3. Responsible parent/guardian has voluntarily signed and dated an Informed Consent Form (ICF) prior to the first study intervention. Whenever the minor child is able to give consent, the minor’s assent will be obtained in addition to the signed consent of the minor’s legal guardian.

Exclusion Criteria

Current participant exclusion criteria as of 07/03/2023:
1. Other subtypes of EB such as EB simplex, EB junctional, dominant dystrophic EB, and Kindler EB
2. Received oral or topical corticosteroids for >7 consecutive days within 30 days or enrolment into this study, except for treatment with oral budesonide
3. Known allergy to any of the constituents of the investigational product
4. Signs of active infection that require treatment with oral or intravenous antibiotics within 7 days of screening
5. History or evidence of active malignancy, including cutaneous squamous cell carcinoma
6. Positive C7 ELISA and positive indirect immunofluorescence (IIF) with binding to the base of salt split skin at screening
7. Pregnant or of child-bearing potential who are not abstinent or practicing an acceptable means of contraception, as determined by the Investigator, for the duration of the treatment phase
8. Received MSC infusions in the last 9 months
9. Simultaneous or previous participation in any interventional trial within 3 months before entering this trial. Participation in simultaneous registry and diagnostic trials during the trial is allowed.

____________

Previous participant exclusion criteria as of 22/04/2022 to 07/03/2023:
1. Other subtypes of EB such as EB simplex, EB junctional, dominant dystrophic EB, and Kindler EB
2. Received oral or topical corticosteroids for >7 consecutive days within 30 days or enrolment into this study, except for treatment with oral budesonide
3. Known allergy to any of the constituents of the investigational product
4. Signs of active infection that require treatment with oral or intravenous antibiotics within 7 days of screening
5. History or evidence of active malignancy, including cutaneous squamous cell carcinoma
6. Positive C7 ELISA and positive indirect immunofluorescence (IIF) with binding to the base of salt split skin
7. Pregnant or of child-bearing potential who are not abstinent or practicing an acceptable means of contraception, as determined by the Investigator, for the duration of the treatment phase
8. Received MSC infusions in the last 9 months
9. Simultaneous or previous participation in any interventional trial within 3 months before entering this trial. Participation in simultaneous registry and diagnostic trials during the trial is allowed.

____________

Previous participant exclusion criteria:
1. Other subtypes of EB such as EB simplex, EB junctional, dominant dystrophic EB, and Kindler EB
2. Received oral or topical corticosteroids for >1 week within 30 days of enrolment into this study
3. Known allergy to any of the constituents of the investigational product
4. Signs of active infection that require treatment with oral or intravenous antibiotics within 7 days of screening
5. History or evidence of active malignancy, including cutaneous squamous cell carcinoma
6. Positive C7 ELISA and positive indirect immunofluorescence (IIF) with binding to the base of salt split skin
7. Pregnant or of child-bearing potential who are not abstinent or practicing an acceptable means of contraception, as determined by the Investigator, for the duration of the treatment phase
8. Received MSC infusions in the last 9 months
9. Simultaneous or previous participation in any interventional trial within 3 months before entering this trial. Participation in simultaneous registry and diagnostic trials during the trial is allowed.

Study & Design

• Study Type: Interventional
• Study Design: Not specified