A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 13 February 2023
• Main ID: NCT03536143
• Date of registration: 20/04/2018
• Prospective Registration: Yes
• Primary sponsor: Krystal Biotech, Inc.
• Public title: A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients GEM-1
• Scientific title: A Phase I/II Study of KB103, a Non-Integrating, Replication-Incompetent HSV Vector Expressing the Human Collagen VII Protein, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)
• Date of first enrolment: May 6, 2018
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03536143
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Clinical diagnosis of the recessive form of dystrophic epidermolysis bullosa (RDEB).

- Age

1. Phase 1: 18 years old or older,

2. Phase 2a: 5 years old or older,

3. Phase 2b: 2 years old or older,

4. Phase 2c: 2 years old or older.

- Willing and able to give consent/assent

- Confirmation of RDEB diagnosis by genetic testing, IF, and IEM

- LH24 antibody negative (non-collagenous [NC] 2domain [NC2-]) and NC1 domain [NC1+]).
(This criterion is applicable to the first 2 adults on the study (Phase 1). Subsequent
subjects can be NC1+ or NC1-)

- Confirmed RDEB COL7A1 mutations in subject

- Wound that meets the wound size/surface area entry criteria:

1. Phase 1: Two wounds up to 10 cm2; 1 randomized to B-VEC and 1 randomized to
placebo

2. Phase 2a and 2b: At least 3 wounds up to 20 cm2; 2 wounds randomized to B-VEC and
1 randomized to placebo

3. Phase 2c: At least 2 wounds up to 50 cm2; at least 1 randomized to B-VEC and 1
randomized to placebo

- Subjects, 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
the required follow-up visits.

Exclusion Criteria:

- Medical instability limiting ability to travel to the investigative center

- The presence of medical illness expected to complicate participation and/or compromise
the safety of this technique, such as active infection with human immunodeficiency
virus (HIV), hepatitis B (as determined by hepatitis B surface antigen screening), or
hepatitis C (as determined by detection of hepatitis C antibodies, or positive result
of hepatitis C polymerase chain reaction [PCR] analysis)

- Serum antibodies to COL7 demonstrated on enzyme-linked immunosorbent assay (ELISA),
indirect immunofluorescence microscopy, Western blot, or cell-mediated immunity to
enzyme-lined ImmunoSpot® (subjects with negative results within 12 months of screening
are eligible)

- Active infection in the area that will undergo administration

- Evidence of systemic infection

- Known allergy to any of the constituents of the product

- Current evidence or a history of squamous cell carcinoma in the area that will undergo
treatment

- Active drug or alcohol addiction

- Hypersensitivity to local anesthesia (lidocaine/prilocaine cream)

- Receipt of chemical or biological study product for the specific treatment of RDEB in
the past 3 months

- Specific wounds that have previously been administered investigational gene or cell
therapy

- Subjects who have taken systemic antibiotics within 7 days

- Positive pregnancy test or breast-feeding

- Clinically significant abnormalities as determined by the investigator



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Dystrophic Epidermolysis Bullosa

Intervention(s)

Biological: Topical beremagene geperpavec

Biological: Placebo gel

Primary Outcome(s)

Complete Wound Closure Responder, ITT Population [Time Frame: from baseline at Weeks 8, 10, and 12]

Number of Adverse Events Reported, Safety Population [Time Frame: baseline to 12 weeks]

Number of Subjects Reported at Least One Adverse Event, Safety Population [Time Frame: baseline to 12 weeks]

Duration of Wound Closure, ITT Population [Time Frame: Time from the complete closure to the first reopening of the same wound]

Time to Wound Closure Analysis, ITT Population [Time Frame: baseline to complete wound closure]

Secondary ID(s)

KB103-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 31/01/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03536143