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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

Reid Thompson, W., Hornby, Brittany, Manuel, Ryan, Bradley, Elena, Laux, Janice, Carr, Jim, Vernon, Hilary J.·2021·Genetics in Medicine
DOI 10.1038/s41436-020-01006-8PMC PMC7935714
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Fig. 1 Study participant flowsheet for SPIBA 201 to test the efficacy and safety of single daily subcutaneous (SC) doses of 40 mg elamipretide (vs. placebo) in subjects with Barth syndrome. Subjects were randomized (1:1) to one of two sequence groups: 12 weeks of single daily SC doses of 40 mg elamipretide in treatment period 1 followed by 12 weeks of treatment with placebo in treatment period 2 (separated by 4-week washout period), or vice versa. The crossover design was selected because of the additional anticipated power of subjects serving as their own control, given the anticipated scarcity of subjects available to participate in this study due to the ultrarare nature of Barth syndrome.
Fig. 1 Study participant flowsheet for SPIBA 201 to test the efficacy and safety of single daily subcutaneous (SC) doses of 40 mg elamipretide (vs. placebo) in subjects with Barth syndrome. Subjects were randomized (1:1) to one of two sequence groups: 12 weeks of single daily SC doses of 40 mg elamipretide in treatment period 1 followed by 12 weeks of treatment with placebo in treatment period 2 (separated by 4-week washout period), or vice versa. The crossover design was selected because of the additional anticipated power of subjects serving as their own control, given the anticipated scarcity of subjects available to participate in this study due to the ultrarare nature of Barth syndrome.CC BY-NC-ND · PubMed Central
Fig. 2 Outcome measures at week 36, part 2 in 8 patients. Statistical significance was achieved in improved measures for 6-minute walk test (m), the Barth Syndrome Symptom Assessment (BTHS-SA) total fatigue scale (mean score), Patient Global Impression (PGI) of symptoms (mean score), muscle strength measured by handheld dynamometry (HHD) (newtons), and the EQ-5D questionnaire Fig. 2. Favorable treatment effects were also seen in other measures (Promis Fatigue [mean score], Clinician Global Impression [CGI] of Barth syndrome symptoms [mean score], and SWAY balance), though not achieving statistical significance. Treatment effect was rescaled to a T-score and standard error unit of 1. *p values represent a t-test for matched pairs, comparing mean at baseline to mean at week 36 in part 2.
Fig. 2 Outcome measures at week 36, part 2 in 8 patients. Statistical significance was achieved in improved measures for 6-minute walk test (m), the Barth Syndrome Symptom Assessment (BTHS-SA) total fatigue scale (mean score), Patient Global Impression (PGI) of symptoms (mean score), muscle strength measured by handheld dynamometry (HHD) (newtons), and the EQ-5D questionnaire Fig. 2. Favorable treatment effects were also seen in other measures (Promis Fatigue [mean score], Clinician Global Impression [CGI] of Barth syndrome symptoms [mean score], and SWAY balance), though not achieving statistical significance. Treatment effect was rescaled to a T-score and standard error unit of 1. *p values represent a t-test for matched pairs, comparing mean at baseline to mean at week 36 in part 2.CC BY-NC-ND · PubMed Central
Fig. 3 6-minute walk test and hand held dynamometry results per participant from baseline to open label extension, week 36. (a) Change in 6-minute walk test (6MWT) results per participant from baseline to open label extension (OLE) week 36. The mean distance walked on the 6MWT increased from baseline across ten subjects at week 12, part 2, with a mean improvement of 60.5 m (16% increase, paired t-test: p = 0.02). At week 36, part 2 across eight subjects the mean improvement from baseline was 95.9 m (25% improvement, paired t-test: p = 0.02). (b) Change in handheld dynamometry (HHD) results per participant from baseline to open label extension (OLE) week 36. At week 12, part 2 there was a mean improvement from baseline HHD of 37.9 newtons (a 30% improvement, paired t-test: p = 0.003) across the ten subjects. At week 36, part 2 there was a mean improvement of +56.0 newtons (a 42% improvement, paired t-test: p = 0.001) across the eight participants who reached the 36 week study visit.
Fig. 3 6-minute walk test and hand held dynamometry results per participant from baseline to open label extension, week 36. (a) Change in 6-minute walk test (6MWT) results per participant from baseline to open label extension (OLE) week 36. The mean distance walked on the 6MWT increased from baseline across ten subjects at week 12, part 2, with a mean improvement of 60.5 m (16% increase, paired t-test: p = 0.02). At week 36, part 2 across eight subjects the mean improvement from baseline was 95.9 m (25% improvement, paired t-test: p = 0.02). (b) Change in handheld dynamometry (HHD) results per participant from baseline to open label extension (OLE) week 36. At week 12, part 2 there was a mean improvement from baseline HHD of 37.9 newtons (a 30% improvement, paired t-test: p = 0.003) across the ten subjects. At week 36, part 2 there was a mean improvement of +56.0 newtons (a 42% improvement, paired t-test: p = 0.001) across the eight participants who reached the 36 week study visit.CC BY-NC-ND · PubMed Central

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Authors
Reid Thompson, W., Hornby, B., Manuel, R., Bradley, E., Laux, J., Carr, J., & Vernon, H. J.
ORCID
Vernon, Hilary J.
Venue
Genetics in Medicine
Publisher
Elsevier BV
Published
2021-03
Volume
23
Issue
3
Pages
471-478
ISSN
1098-3600
DOI
10.1038/s41436-020-01006-8
PMC
PMC7935714
Cited by
127
References
0
Language
en
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