Abstract

Objective

To assess the impact of recombinant human parathyroid hormone (1-84) [rhPTH(1-84)] compared with placebo, in combination with conventional therapy with vitamin D and/or calcium supplements, on health-related quality of life (HRQoL) in patients with symptomatic chronic hypoparathyroidism (cHypoPT).

Design

Randomized, double-blind, placebo-controlled, phase 3b-4 study (ClinicalTrials.gov ID: NCT03324880).

Methods

Eligible patients with symptomatic cHypoPT were randomized to receive subcutaneous rhPTH(1-84) 25-100 µg/day or placebo. The primary endpoint was the change from baseline to week 26 in Hypoparathyroidism Symptom Diary (HypoPT-SD) symptom subscale score. Key secondary endpoints were changes from baseline to week 26 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and in 36-item Short Form Health Survey physical component summary (SF-36v2 PCS).

Results

In total, 93 patients were randomized to receive treatment: 45 received rhPTH(1-84) and 48 received placebo. Change from baseline to week 26 in HypoPT-SD symptom subscale score was significantly greater (improved) in the rhPTH(1-84) group than in the placebo group (difference in least-squares mean changes, −0.53; 95% confidence interval, −0.90 to −0.15, P = .003). Key secondary endpoints, changes between baseline and week 26 in the FACIT-Fatigue and SF-36v2 PCS scores were also significantly greater (improved) in the rhPTH(1-84) group than in the placebo group. The safety profile of rhPTH(1-84) was consistent with previous findings, and no new safety signals were identified.

Conclusions

rhPTH(1-84) alongside conventional therapy improved symptom burden (as measured by the HypoPT-SD) and HRQoL to a greater extent than conventional therapy alone in patients with symptomatic cHypoPT.