A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

Overview

About this study

The primary objective of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Recessive Dystrophic Epidermolysis Bullosa (RDEB) and confirmed mutation of the type VII collagen gene (COL7A1) gene.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Subjects and/or their legal guardian must provide written informed consent, in accordance with federal and/or local laws, before any study procedures occur. In addition, if applicable, a minor child must provide written informed assent in accordance with federal and/or local laws as well as in compliance with the recommendations of the approving Institutional Review Board (IRB), before any study procedures occur.
  • Male or female, ≥ 2 years of age at the Screening visit (when consent/assent for study participation is given).
  • Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation.
  • At least two eligible persistent non-healing or recurrent wounds identified at Day 1 (Visit 2):
    • Each wound must have an approximate minimum area of 5 cm^2 (subjects 2 to 6 years of age at time of consent) or 10 cm^2 (subjects 7 years of age or older at time of consent) and a maximum of 50 cm^2;
    • Wound site was monitored for at least 12 weeks prior to Day 1 (Visit 2) and wound is open (unhealed);
    • The following wounds are excluded:
      • Wounds on a mucous membrane, the face, hands, feet, or fully across joints;
      • Wounds with active infection or evidence of active infection;
      • Wounds that have been previously treated with a gene therapy intervention.
  • Female subjects of childbearing potential and post-pubertal male subjects engaging in sexual activity that could lead to pregnancy who agree to using at least one of the following adequate birth control regimens while in the study and for 6 months after last dose of FCX-007 is administered:
    • Male partner with vasectomy; OR
    • Male condom AND partner use of one of the contraceptive options below:
      • Spermicide;
      • Intrauterine device or intrauterine system;
      • Oral contraceptive, either combined or progestogen alone;
      • Contraceptive subdermal implant (e.g., Norplant®);
      • Injectable progestogen (e.g., Depo-Provera®);
      • Contraceptive vaginal ring (e.g., NuvaRing®);
      • Transdermal contraceptive patches (e.g., Ortho Evra®).
  • Subjects of childbearing potential who are abstinent are eligible, but they must agree to use one of the birth control regimens listed above if they begin engaging in sexual activity that could lead to pregnancy during the study.
  • Periodic abstinence; e.g., calendar, ovulation, sympto-thermal, post-ovulation methods and withdrawal are not acceptable methods of contraception.
    • NOTE: Subjects using an acceptable hormonal based contraceptive must have been on a stable dose for at least 3 months before Day 1 (Visit 2) and be willing to continue stable birth control methods throughout the study.
  • Non-childbearing potential females are defined as females who are premenarchal, or postmenopausal (12 months with no menstrual period without an alternative medical cause), or who have undergone a hysterectomy, bilateral tubal ligation, bilateral oophorectomy (removal of the ovaries), or hysteroscopic sterilization. Documented verbal history from the subject is acceptable.
  • Subjects and/or their legal guardian, if applicable, who are, in the opinion of the Investigator, able to understand the study, cooperate with the study procedures and are willing to return to the clinic for all required follow-up visits.

Exclusion Criteria:

  • Female who is pregnant or breastfeeding.
  • Medical instability limiting ability to travel to the investigative site.
  • Active infection with human immunodeficiency virus (HIV), hepatitis B (Hep B) or hepatitis C (Hep C), as determined by Hep B surface antigen screening (HBsAg), detection of HIV or Hep C antibodies, or positive result of Hep C polymerase chain reaction (PCR) analysis.
  • The presence of COL7 antibodies in clinically significant titer by indirect immunofluorescence (IIF) using the subject’s serum or COL7 antibodies noted on direct immunofluorescence (DIF) of the subject’s skin biopsy.
  • Any clinically significant abnormal laboratory result identified from Day 1 (Visit 2) labs obtained prior to wound randomization.
  • Evidence of systemic infection.
  • Evidence or history of squamous cell carcinoma at the site to be injected.
  • Evidence of or history of metastatic squamous cell carcinoma.
  • Clinically significant abnormalities identified through medical history, AEs and physical examination prior to wound randomization on Day 1 (Visit 2).
  • Known allergy to any of the constituents of the product.
  • Hypersensitivity to anesthesia chosen (lidocaine/prilocaine cream, moderate sedation, or general anesthesia).
  • Active drug or alcohol addiction.
  • Receipt of a chemical or biological intervention for the specific treatment of RDEB in the past three (3) months prior to screening or anticipated/planned during the screening and treatment period for this study.
  • Receipt of chemotherapy in the past three (3) months prior to screening or anticipated/ planned during the screening and treatment period for this study.
  • Receipt of another investigational drug in the past 30 days prior to screening or anticipated/planned during the screening and treatment period for this study.
  • A history of or ongoing serious illness or medical, physical, laboratory abnormality, or psychiatric condition(s) that, in the Investigator’s opinion, may interfere with the subject’s participation in the study and/or ability to understand and give informed consent.

Eligibility last updated 8/23/21.  Questions regarding updates should be directed to the study team contact.

 

 

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Jennifer Hand, M.D.

Open for enrollment

Contact information:

Katrina Pierce CCRP

(507) 266-1078

Pierce.Katrina@mayo.edu

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available

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CLS-20516353

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