Table 1 Baseline characteristics of enrolled patients.

From: A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism

Demographic variable

Demographic result

Mean age (years) (range)

19.5 (12–35)

Male (n)

12

Race (n)

  White

11

  Multiracial

1

Ethnicity (n)

  Not Hispanic or Latino

12

  Hispanic or Latino

0

Mean height (cm)

167.3 (150.4–187.7)

Mean weight (kg)

50.8 (31.4–85.9)

BMI (kg/m2)

17.6 (13.6–24.4)

Mean 6MWT (meters)

395.5

Mean BTHS-SA total fatigue

8

Mean 3D LV end-diastolic volume (Z-score [SD])

−2 (1.34)

Mean 3D LV stroke volume (Z-score [SD])

−1.84 (1.53)

Mean ejection fraction (% [SD])

60.6 (4.0)

  1. All 12 randomized subjects completed both treatment periods in part 1. Overall, the majority of subjects in part 1 were White (91.7%), Non-Hispanic or Latino (100.0%), with a mean (SD) age of 19.5 (7.65) years. Mean (SD) weight and BMI were 50.84 (18.934) and 17.56 (3.818), respectively. One subject (16.7%) included in the trial was Indian or an Alaskan Native. Sequence AB (elamipretide–placebo) were slightly older, ranging in age between 14 and 35 years (mean [SD]: 23.3 [9.07] years), while subjects in treatment sequence BA (placebo–elamipretide) were slightly younger, ranging in age between 12 and 21 years (mean [SD]: 15.7 [3.33] years), compared with the overall population, which ranged between 12 and 35 years. For subjects in treatment sequence AB, mean weight and BMI were 54.50 kg and 18.23 kg/m2, respectively. Mean weight and BMI were lower for subjects in treatment sequence BA (47.18 kg and 16.88 kg/m2, respectively). Demographics of each treatment sequence were generally similar to the overall population with some variability observed in overall age, age ranges, weight, and BMI.
  2. 6MWT 6-minute walk test, BTHS-SA Barth Syndrome Symptom Assessment scale, BMI body mass index, LV left ventricular.
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