Table 1.
Baseline characteristics of the participants in MMPOWER‐2.
| Treatment sequence (elamipretide: placebo) n = 14 | Treatment sequence (placebo: elamipretide) n = 16 | All patients N = 30 | |
|---|---|---|---|
| Age, mean (range), years | 41.5 (17–63) | 48.6 (25–65) | 45.3 (17–65) |
| Sex, n (%) | |||
| Female | 10 (71) | 15 (94) | 25 (83) |
| Male | 4 (29) | 1 (6) | 5 (17) |
| Race/ethnicity, n (%) | |||
| White | 13 (93) | 16 (100) | 29 (97) |
| Multiple (White/Asian/other) | 1 (7) | 0 | 1 (3) |
| Non‐Hispanic or Latino | 14 (100) | 15 (94) | 29 (97) |
| Hispanic or Latino | 0 | 1 (6) | 1 (3) |
| Weight, mean (SD), kg | 60.5 (±10.0) | 69.2 (±16.3) | 65.1 (±14.2) |
| BMI, mean (range), kg/m2 | 22.8 (15.8–33.2) | 25.3 (19.0–36.0) | 24.1 (15.8–36.0) |
| Baseline 6MWT, mean (SD), m | 381.2 (±30.4) | 396.5 (±36.3) | 389.4 (±23.6) |
| Baseline 6MWT, n (%), m | |||
| <450 | 13 (93) | 9 (56) | 22 (73) |
| ≥450 | 1 (7) | 7 (44) | 8 (27) |
| Genotype characteristics: Mitochondrial DNA (mtDNA) | |||
| Disorders involving mtDNA mutations that impair mitochondrial protein synthesis in toto | 19 | ||
| • Mitochondrial deletion syndrome | 11 | ||
| • m.3243A > G | 4 | ||
| • m.8344A > G | 3 | ||
| • Multisystem mitochondrial disorder (MT‐TH and tRNA) | 1 | ||
| Disorders involving mtDNA mutations that affect the subunits of the respiratory chain | 5 | ||
| • Multisystem mitochondrial disorder (MT‐COX1) | 1 | ||
| • Mitochondrial Myopathy (MTCYB) | 1 | ||
| • LHON Plus | 1 | ||
| • Multisystem Mitochondrial Disorder (MT‐ND3) | 1 | ||
| • Leigh syndrome (NDUFV1) | 1 | ||
| Nuclear DNA (nDNA) | |||
| Disorders involving nDNA mutations causing defects of intergenomic signalling | 3 | ||
| • POLG‐related disorder | 3 | ||
| Disorders involving nDNA mutations causing alterations of the lipid milieu of the inner mitochondrial membrane | 1 | ||
| • MEGDEL | 1 | ||
| Disorders involving nDNA mutations causing alterations of mitochondrial motility or fission | 2 | ||
| • Multisystem mitochondrial disorder (OPA1) | 2 | ||
6MWT, 6 min walk test; BMI, body mass index; SD, standard deviation.
The bold text describes the overall number of patients within the headlined category. i.e there are 19 patients overall in the disorders involving mtDNA mutations that impair mitochondrial protein synthesis in toto.