Patient-reported outcomes in palopegteriparatide-treated adults with hypoparathyroidism: PaTH Forward trial extension.

Palopegteriparatide treatment demonstrated significant improvements in disease-specific symptoms from baseline at week 12 that were sustained through week 110.

• PRO measures were collected at baseline, weeks 4, 12, 26, 58, and annually thereafter through the end of the trial (week 266).
• The Hypoparathyroidism Patient Experience Scales (HPES) were used to assess disease-specific symptoms and impacts on functioning and well-being.
• Mean changes in PROs met thresholds for clinically meaningful within-patient improvement.
• Data were analyzed using descriptive statistics and Mixed Models for Repeated Measures (MMRM).

Palopegteriparatide treatment was associated with significant improvements in general health-related quality of life as measured by the SF-36v2.

• The Short Form Health Survey (SF-36v2) was used to measure general health-related QoL.
• Significant improvements in SF-36v2 scores were observed during palopegteriparatide treatment.
• The PaTH Forward trial included a 4-week randomized, double-blind, placebo-controlled period followed by an open-label extension period lasting through trial week 266.

Improvements in patient-reported outcomes with palopegteriparatide were generally similar across demographics and patient characteristics.

• Associations between patient characteristics and changes in patient-reported outcomes were analyzed as a secondary objective.
• Results were reported as generally consistent across subgroups defined by demographics and patient characteristics.
• This analysis covered adults with chronic hypoparathyroidism enrolled in the PaTH Forward phase 2 clinical trial.

Adults with hypoparathyroidism experience a range of physical and cognitive symptoms and reduced quality of life despite conventional therapy.

• Conventional therapy consists of active vitamin D and calcium supplementation.
• The burden of hypoparathyroidism including symptoms and reduced QoL persists despite standard management, providing the rationale for PTH replacement therapy.
• The disease-specific HPES instrument was used because it captures the particular symptom burden and functional impacts relevant to hypoparathyroidism.

The PaTH Forward trial was a phase 2 clinical trial evaluating palopegteriparatide with both a blinded and an extended open-label period.

• The trial included a 4-week randomized, double-blind, placebo-controlled period.
• This was followed by an open-label extension period lasting through trial week 266.
• PRO measures included the HPES for disease-specific outcomes and the SF-36v2 for general health-related QoL.
• PRO data collection time points were baseline, weeks 4, 12, 26, 58, and annually thereafter.