Table 1 Summary of wound closure analyses

From: In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial

Complete wound closurea at weeks 8, 10, or 12

Week

Wounds closeda n (%)

% difference

B-VEC

Placebob

8 (n = 21)

13/14 (93)

0/7 (0)

93

10 (N = 20)

11/14 (79)

2/6 (33)

46

12 (n = 19)

10/12 (83)

1/7 (14)

69

Complete wound closurea at weeks 8 and 10 or weeks 10 and 12

Weeks

Wounds closeda n (%)

% Difference

B-VEC

Placebo

8 & 10 (N = 21)

11/14 (79)

0/7 (0)

79

10 and 12 (N = 19)

9/12 (75)

0/7 (0)

75

Responder analysis: complete wound closurea at weeks 8 and 10 or weeks 10 and 12

8 and 10 or 10 and 12 (n = 21)

11/14 (79)

0/7 (0)

% difference

P valuec

79

0.0026*

Time to complete closurea,d (days)

Median (95% CI)e,f

B-VEC

Placebo

13.5 (8, 21)

22.5 (8, 64)

Duration of closureg (days)

103.0 (94, 118)

16.5 (0, 66)

  1. aComplete closure defined as ≥95% reduction in wound surface area from baseline.
  2. bThe placebo wound on patient 11 was not imaged, therefore the wound could not be confirmed as closed or open and patient 11’s wounds were not included in the responder analysis.
  3. cP value based on McNemar’s test (one-sided).
  4. dTime to wound closure was defined as the time from the first treatment to complete wound closure (≥95% reduction in wound surface area from baseline) observed for 2 consecutive weeks.
  5. eThe median estimate and the 95% confidence interval (CI) were derived using the Kaplan–Meier method.
  6. fNumerically the trend favors B-VEC.
  7. gDuration of wound closure was defined as the time from complete closure (≥95% reduction in wound surface area from baseline) to the first re-opening.
  8. *Significant at P < 0.025.
  9. The phase 1 protocol collected wound closure data at weeks 6 and 12 (no week 8 or week 10 datapoints), therefore the phase 1 patients (patients 1 and 2) were excluded from the responder analysis.
  10. Patient 4 withdrew after initial dosing due to inability to travel.
  11. Included in the analysis are data from week 8 for patients 3–10, week 10 for patients 3–10 (missing datapoint for patient 10, placebo), and week 12 for patients 3–10 (missing datapoints for patient 7, B-VEC). Results and analysis reported are based upon observed data without imputed values for missing data.
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