TY - JOUR
T1 - MPOWERED Trial Open-Label Extension
T2 - Long-term Efficacy and Safety Data for Oral Octreotide Capsules in Acromegaly
AU - Fleseriu, Maria
AU - Molitch, Mark
AU - Dreval, Alexander
AU - Pokramovich, Yulia
AU - Bondar, Irina
AU - Poteshkin, Yury
AU - Macut, Djuro
AU - Obermayer-Pietsch, Barbara
AU - Gilgun-Sherki, Yossi
AU - Haviv, Asi
AU - Biermasz, Nienke
AU - Strasburger, Christian J.
N1 - Publisher Copyright:
© The Author(s) 2023. Published by Oxford University Press on behalf of the Endocrine Society.
PY - 2023/11/17
Y1 - 2023/11/17
N2 - CONTEXT: The MPOWERED core trial (NCT02685709) and open-label extension (OLE) phase investigated long-term efficacy and safety of oral octreotide capsules (OOC) in patients with acromegaly. Core trial primary endpoint data demonstrated noninferiority to injectable somatostatin receptor ligands (iSRLs). Core trial completers were invited to participate in the OLE phase. OBJECTIVE: To assess long-term efficacy and safety of OOC in patients with acromegaly who previously responded to and tolerated both OOC and injectable octreotide/lanreotide and completed the core phase. METHODS: The unique study design of transitioning between OOC and iSRLs allowed within-patient evaluations. The proportion of biochemical responders (insulin-like growth factor I < 1.3 × upper limit of normal) at end of each extension year who entered that year as responders was the main outcome measure. RESULTS: At year 1 extension end, 52/58 patients from both the monotherapy and the combination therapy groups were responders (89.7%; 95% CI 78.8-96.1), 36/41 (87.8%; 95% CI 73.8-95.9) in year 2, and 29/31 (93.5%; 95% CI 78.6-99.2) in year 3. No new or unexpected safety signals were detected; 1 patient withdrew owing to treatment failure. Patients who transitioned from iSRLs in the core trial to OOC in the OLE phase reported improved treatment convenience/satisfaction and symptom control. CONCLUSION: Patient-reported outcome data support for the first time that transitioning patients randomized to iSRL (who previously responded to both OOC and iSRLs) back to OOC had a significant effect on patients' symptoms score in a prospective cohort. The MPOWERED OLE showed long-term maintenance of response and sustained safety with OOC.
AB - CONTEXT: The MPOWERED core trial (NCT02685709) and open-label extension (OLE) phase investigated long-term efficacy and safety of oral octreotide capsules (OOC) in patients with acromegaly. Core trial primary endpoint data demonstrated noninferiority to injectable somatostatin receptor ligands (iSRLs). Core trial completers were invited to participate in the OLE phase. OBJECTIVE: To assess long-term efficacy and safety of OOC in patients with acromegaly who previously responded to and tolerated both OOC and injectable octreotide/lanreotide and completed the core phase. METHODS: The unique study design of transitioning between OOC and iSRLs allowed within-patient evaluations. The proportion of biochemical responders (insulin-like growth factor I < 1.3 × upper limit of normal) at end of each extension year who entered that year as responders was the main outcome measure. RESULTS: At year 1 extension end, 52/58 patients from both the monotherapy and the combination therapy groups were responders (89.7%; 95% CI 78.8-96.1), 36/41 (87.8%; 95% CI 73.8-95.9) in year 2, and 29/31 (93.5%; 95% CI 78.6-99.2) in year 3. No new or unexpected safety signals were detected; 1 patient withdrew owing to treatment failure. Patients who transitioned from iSRLs in the core trial to OOC in the OLE phase reported improved treatment convenience/satisfaction and symptom control. CONCLUSION: Patient-reported outcome data support for the first time that transitioning patients randomized to iSRL (who previously responded to both OOC and iSRLs) back to OOC had a significant effect on patients' symptoms score in a prospective cohort. The MPOWERED OLE showed long-term maintenance of response and sustained safety with OOC.
KW - IGF-I
KW - acromegaly
KW - clinical trials
KW - growth hormone excess/acromegaly
KW - somatostatin
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U2 - 10.1210/clinem/dgad365
DO - 10.1210/clinem/dgad365
M3 - Article
C2 - 37319438
AN - SCOPUS:85175803023
SN - 0021-972X
VL - 108
SP - 3214
EP - 3222
JO - The Journal of clinical endocrinology and metabolism
JF - The Journal of clinical endocrinology and metabolism
IS - 12
ER -