Abstract
PurposeResults are presented from 2 to 3 trials investigating oral octreotide capsules (OOC) as an alternative to injectable somatostatin receptor ligands (iSRLs) in the treatment of acromegaly.MethodsCH-ACM-01 was an open-label trial (N = 155) and CHIASMA OPTIMAL was a double-blind placebo-controlled (DPC) trial (N = 56), both investigating OOC as maintenance therapy for patients with acromegaly who were biochemical responders receiving iSRLs.ResultsBaseline characteristics in both trials reflected those expected of patients with acromegaly responding to treatment and were similar between trials, despite differences in inclusion criteria. OOC demonstrated a consistent degree of biochemical response across trials, with 65% of patients in CH-ACM-01 maintaining response during the core period and 64% of patients in CHIASMA OPTIMAL at the end of the DPC. Mean insulin-like growth factor I (IGF-I) levels remained within inclusion criteria at the end of treatment in both trials. Of 110 patients entering the fixed-dose phase in CH-ACM-01, 80% maintained or improved acromegaly symptoms from baseline to the end of treatment. Over 85% of patients in both trials elected to continue into the extension phases. OOC were found to be well tolerated across both trials, and no dose-related adverse events were observed.ConclusionsOOC demonstrated remarkably consistent results for biochemical response, durability of response, and preference to continue with oral treatment across these 2 complementary landmark phase 3 trials, despite differences in the design of each.Trial registration NCT03252353 (August 2017), NCT01412424 (August 2011).
Highlights
Is characterized by excessive circulating levels of growth hormone (GH) and insulin-like growth factor I (IGF-I), usually resulting from a GH-secreting pituitary adenoma [1,2,3,4,5]
For CHIASMA OPTIMAL, 119 individuals were screened; 63 (53%) failed screening and 56 of were enrolled into the trial, 28 of whom were randomized to octreotide capsules (OOC)
The baseline disease characteristics of patients enrolled in both trials were very similar despite the difference in biochemical values due to varying inclusion criteria (IGF-I < 1.3 × ULN for the CH-ACM-01 trial and IGF-I ≤ 1.0 × ULN for CHIASMA OPTIMAL)
Summary
Is characterized by excessive circulating levels of growth hormone (GH) and insulin-like growth factor I (IGF-I), usually resulting from a GH-secreting pituitary adenoma [1,2,3,4,5]. Treatment for acromegaly is aimed at normalizing GH and IGF-I levels, shrinking tumors, amelioration of symptoms, improving quality of life, and reducing mortality with as few side effects as possible [1, 6, 7]. Long-acting injectable SRLs (iSRLs) are a cornerstone of medical treatment in acromegaly and have demonstrated efficacy in attenuating serum GH and IGF-I levels, reducing tumor size, and improving symptoms [12]. The Acromegaly Treatment Satisfaction Questionnaire (Acro-TSQ) is a scale that was developed to provide a complete assessment of the disease and treatment burdens associated with acromegaly and for tracking the overall efficacy and unmet needs of new treatments for patients with acromegaly [9, 14]. Potentially by substituting an oral formulation, was mentioned as a top preference for new acromegaly treatments among 85% of respondents [13]
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