Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Change From Baseline in Hypoparathyroidism Symptom Diary (HypoPT-SD) Symptom Subscale Score at Week 26 |
The HypoPT-SD is a 13-item patient-reported outcomes instrument that consists of the following items: symptom subscale (items 1-7), anxiety (item 8), sadness or depression (item 9) and impact subscale (items 10-13). For items 1-9, the individual score ranges from None=0 to Very severe=4; for items 10-13, it ranges from Not at all=0 to Very much=2. An item score was computed by taking the average of the daily item response over the 14-day period immediately before the visit. If data were not available for at least 4 out of 7 days during both 7-day periods within the 14-day period, the individual item score was set to missing. The symptom subscale score was computed as the average of symptom items 1-7 scores with more than 3 of the 7 symptom item scores were non-missing. Negative change in scores indicates improvement. A mixed model for repeated measures (MMRM) was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 26 |
The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) questionnaire contains 13 fatigue-related questions. The responses to the 13 items on the FACIT-Fatigue questionnaire are each measured on a 5-point Likert scale, where 0=Not at all, 1=A little bit, 2=Somewhat, 3=Quite a bit and 4=Very much. Thus, the total score ranges from 0 to 52. High scores represent less fatigue. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Physical Component Summary (PCS) Derived From 36-Item Short Form Health Survey Version 2 (SF-36v2) Scores at Week 26 |
The SF-36 is a validated instrument that questions participants about perceived physical and mental health and function. The SF-36 consists of 8 scaled scores (vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, and mental health), which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight; the lower the score the more disability. Change in PCS derived from SF-36v2 at Week 26 was reported. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Hypoparathyroidism Symptom Diary (HypoPT-SD) Impact Subscale Score at Week 26 |
The HypoPT-SD is a 13-item patient-reported outcomes instrument that consists of the following items: symptom subscale (items 1-7), anxiety (item 8), sadness or depression (item 9) and impact subscale (items 10-13). For items 1-9, the individual score ranges from None=0 to Very severe=4; for items 10-13, it ranges from Not at all=0 to Very much=2. An item score was computed by taking the average of the daily item response over the 14-day period immediately before the visit. If data were not available for at least 4 out of 7 days during both 7-day periods within the 14-day period, the individual item score was set to missing. The impact subscale score was computed as the average of impact items 10-13 scores with no impact item score was non-missing. Negative change in scores indicates improvement. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Individual Hypoparathyroidism Symptom Diary (HypoPT-SD) Impact Items Score at Week 26 |
The HypoPT-SD is a 13-item patient-reported outcomes instrument that consists of the following items: symptom subscale (items 1-7), anxiety (item 8), sadness or depression (item 9) and impact subscale (items 10-13). For items 1-9, the individual score ranges from None=0 to Very severe=4; for items 10-13, it ranges from Not at all=0 to Very much=2. An item score was computed by taking the average of the daily item response over the 14-day period immediately before the visit. If data were not available for at least 4 out of 7 days during both 7-day periods within the 14-day period, the individual item score was set to missing. The change in individual symptom item scores was reported. Negative change in scores indicates improvement. MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Hypoparathyroidism Symptom Diary (HypoPT-SD) Symptom Item Anxiety (Item 8) Score at Week 26 |
The HypoPT-SD is a 13-item patient-reported outcomes instrument that consists of the following items: symptom subscale (items 1-7), anxiety (item 8), sadness or depression (item 9) and impact subscale (items 10-13). For items 1-9, the individual score ranges from None=0 to Very severe=4; for items 10-13, it ranges from Not at all=0 to Very much=2. The anxiety item score was computed by taking the average of the daily item response over the 14-day period immediately before the visit. If data were not available for at least 4 out of 7 days during both 7-day periods within the 14-day period, the anxiety item score was set to missing. Negative change in scores indicates improvement. MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Hypoparathyroidism Symptom Diary (HypoPT-SD) Symptom Item Sadness or Depression (Item 9) Score at Week 26 |
The HypoPT-SD is a 13-item patient-reported outcomes instrument that consists of the following items: symptom subscale (items 1-7), anxiety (item 8), sadness or depression (item 9) and impact subscale (items 10-13). For items 1-9, the individual score ranges from None=0 to Very severe=4; for items 10-13, it ranges from Not at all=0 to Very much=2. The sadness or depression item score was computed by taking the average of the daily item response over the 14-day period immediately before the visit. If data were not available for at least 4 out of 7 days during both 7-day periods within the 14-day period, the item score was set to missing. Negative change in scores indicates improvement. MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Individual Hypoparathyroidism Symptom Diary (HypoPT-SD) Symptom Item Scores at Week 26 |
The HypoPT-SD is a 13-item patient-reported outcomes instrument that consists of the following items: symptom subscale (items 1-7), anxiety (item 8), sadness or depression (item 9) and impact subscale (items 10-13). For items 1-9, the individual score ranges from None=0 to Very severe=4; for items 10-13, it ranges from Not at all=0 to Very much=2. An item score was computed by taking the average of the daily item response over the 14-day period immediately before the visit. If data were not available for at least 4 out of 7 days during both 7-day periods within the 14-day period, the individual item score was set to missing. Negative change in scores indicates improvement. MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Number of Participants With Response at Week 26 [Early Termination (ET)] |
Response was defined as a 30% reduction in HypoPT-SD symptom subscale score from baseline. The HypoPT-SD is a 13-item patient-reported outcomes instrument that consists of the following items: symptom subscale (items 1-7), anxiety (item 8), sadness or depression (item 9) and impact subscale (items 10-13). For items 1-9, the individual score ranges from None=0 to Very severe=4; and for items 10-13, it ranges from Not at all=0 to Very much=2. An item score was computed by taking the average of the daily item response over the 14-day period immediately before the visit. If data were not available for at least 4 out of 7 days during both 7-day periods within the 14-day period, the individual item score was set to missing. The symptom subscale score was computed as the average of symptom items 1-7 scores with more than 3 of the 7 symptom item scores were non-missing. Data reported also includes results for early terminated participants. |
Baseline up to Week 26 |
|
| Secondary |
Change From Baseline in the Most Bothersome Symptom Score at Week 26 |
The HypoPT-SD is a 13-item patient-reported outcomes instrument that consists of the following items: symptom subscale (items 1-7), anxiety (item 8), sadness or depression (item 9) and impact subscale (items 10-13). For items 1-9, the individual score ranges from None=0 to Very severe=4 and for items 10-13, it ranges from Not at all=0 to Very much=2. An item score was computed by taking the average of the daily item response over the 14-day period immediately before the visit. If data were not available for at least 4 out of 7 days during both 7-day periods within the 14-day period, the individual item score was set to missing. The Most Bothersome Symptom Score was analyzed. Negative change in scores indicates improvement. MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Functional Assessment of Cancer Therapy-Cognitive Function (FACT-Cog) Perceived Cognitive Impairments (PCI) Score at Week 26 |
The Functional Assessment of Cancer Therapy-Cognitive Function (FACT-Cog) assessment is a 37-item instrument. The perceived cognitive impairment and the impact on quality of life domains were assessed in this study. These 2 domains include 22 items rated on a 5-point scale. The perceived cognitive impairments subscale contains 18 items and each item has a 5-point ordinal response scale (0=Never, 1= About once a week, 2 = Two to three times a week, 3= Nearly every day, 4 = Several times a day). Each item score is calculated as (4 minus item response), and the subscale score is [sum of (4 minus item response)]*18/(number of items answered)]. The perceived cognitive impairment subscale score ranges from 0 to 72, with higher scores indicate better cognitive function. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Functional Assessment of Cancer Therapy-Cognitive Function (FACT-Cog) Impact on Quality of Life (QoL) Score at Week 26 |
The Functional Assessment of Cancer Therapy-Cognitive Function (FACT-Cog) assessment is a 37-item instrument. The perceived cognitive impairment and the impact on quality of life domains were assessed in this study. These 2 domains include 22 items rated on a 5-point scale. The impact on quality of life domain contains 4 items and each item has a 5-point ordinal response scale (0=Never, 1= About once a week, 2 = Two to three times a week, 3= Nearly every day, 4 = Several times a day). Each item score is calculated as (4 minus item response), and the subscale score is [sum of (4 minus item response)]*4/(number of items answered)]. The impact on quality of life subscale score ranges from 0 to 16 with higher score indicates better cognitive function. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Individual Domains of 36-Item Short Form Health Survey Version 2 (SF-36v2) at Week 26 |
The SF-36 is a validated instruments that question participants about perceived physical and mental health and function. The SF-36 consists of 8 scaled scores (vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, and mental health), which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight; the lower the score the more disability. Change in the score of individual domains of SF-36v2 at Week 26 was reported. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Mental Component Summary (MCS) Score of 36-Item Short Form Health Survey Version 2 (SF-36v2) at Week 26 |
The SF-36 is a validated instruments that question participants about perceived physical and mental health and function. The SF-36 consists of 8 scaled scores (vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, and mental health), which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight; the lower the score the more disability. Change in the MCS of SF-36v2 at Week 26 was reported. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Work Productivity and Activity Impairment Questionnaire: Hypoparathyroidism (WPAI: Hypoparathyroidism) Score at Week 26 |
WPAI assessed impact of HypoPT on work productivity and daily activities. Concepts that WPAI: Hypoparathyroidism measures include time missed from work and impairment of work and other regular activities due to specific health problem (HypoPT). WPI was calculated based on 4 items including Q2: hours of work missed due to HPT; Q4: actual hours worked; Q5: HPT effect on productivity at work; Q6: HPT effect on daily activities. Scores for 4 subscales were calculated as Percent work time missed due to problem: Q2/(Q2+Q4)*100; Percent impairment while working due to problem: Q5/10*100; Percent overall work impairment due to problem: Q2/(Q2+Q4)+[(1(Q2/(Q2+Q4)))x(Q5/10)]*100; Percent activity impairment due to problem: Q6/10*100. WPAI outcomes are expressed as impairment percentages, with higher numbers indicating greater impairment and less productivity, i.e., worse outcomes. Change from baseline in questionnaire response was reported. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Scores of Patient's Assessment of Overall Health Status Using Patient Global Impression of Severity (PGI-S) at Week 26 |
The PGI-S is a global index that can be used to rate the severity of a specific condition. The PGI-S is a rating scale that asks the respondent to best describe how their symptoms severity. Response options are assessed as per 5-point scale: no symptoms (0), mild (1), moderate (2), severe (3), and very severe (4). Mean change in scores of PGI-S at Week 26 was reported. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Scores of Patient's Assessment of Overall Health Status Using Patient Global Impression of Change (PGI-C) at Week 26 |
The PGI-C is verbal rating scale asks the respondent to best describe change in symptoms compared to the beginning of study. Response options are assessed using a 7-point scale: very much improved (0), much improved (1), minimally improved (2), no change (3), minimally worse (4), much worse (5), and very much worse (6). Negative change indicates improvement. Mean change in scores of PGI-C at Week 26 was be reported. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in In-Clinic Neurocognitive Assessment Scores at Week 24 |
Neurocognitive test battery included tests evaluating frontal-executive domain, which encompasses functions attributable to prefrontal cortex and its connections to basal ganglia (mostly striatum). Tests included the CogState (CS) Brief Battery (including the Detection: speed [range from 2.001 to 6; lower scores (LS) indicate improvement (IMP)], Identification: speed [range from 2.001 to 6; LS indicate IMP], One Card Learning: accuracy [range from 0 to 1.5708; higher scores (HS) indicate IMP], One Back: speed [range from 2.001 to 6; LS indicate IMP]), CS Groton Maze Learning Test: total errors (range from 0 to infinity; LS indicate IMP), CS International Shopping List Task (ISLT): number of correct responses (range from 0 to infinity; HS indicate IMP), and CS ISLT -Delayed Recall: number of correct responses (range from 0 to infinity; HS indicate IMP). Change in in-clinic neurocognitive assessment scores at Week 24 was reported. Analysis of Covariance (ANCOVA) was used for analysis. |
Baseline, Week 24 |
|
| Secondary |
Change From Baseline in At-Home Neurocognitive Assessment Scores at Week 26 |
Neurocognitive test battery included tests evaluating frontal-executive domain, which encompasses functions attributable to prefrontal cortex and its connections to basal ganglia (mostly striatum). Tests included the CogState (CS) Brief Battery (including the Detection: speed [range from 2.001 to 6; lower scores (LS) indicate improvement (IMP)], Identification: speed [range from 2.001 to 6; LS indicate IMP], One Card Learning: accuracy [range from 0 to 1.5708; higher scores (HS) indicate IMP], One Back: speed [range from 2.001 to 6; LS indicate IMP]). Changes in at-home neurocognitive assessment scores (CS Brief Battery) at Week 26 was reported. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in 24-hour Urine Calcium Excretion at Week 26 |
Change in 24-hour urine calcium excretion at Week 26 was reported. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Serum Phosphate Level at Week 26 |
Change in serum phosphate level at Week 26 was reported. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Doses of Active Vitamin D at Week 26 |
Changes in doses of active vitamin D at Week 26 was reported. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Doses of Calcium Supplements at Week 26 |
Changes in doses of calcium supplements at Week 26 was reported. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Albumin-corrected Serum Calcium Control at Week 26 |
Change From Baseline in albumin-corrected serum calcium between 1.875 millimoles per liter (mmol/L) (7.5 milligram per deciliter [mg/dL]) and upper limit of normal (ULN) for the central laboratory normal range at Week 26 was reported. |
Week 26 |
|
| Secondary |
Number of Participants Who Achieve Composite Criteria for Albumin-corrected Serum Calcium Concentration, Active Vitamin D Dose and Oral Elemental Calcium Supplement Dose at Week 26 |
Number of participants achieving composite criteria of the following: albumin-corrected serum calcium between 1.875 mmol/L (7.5 mg/dL) and the ULN for the central laboratory normal range, dose of active vitamin D decreased by 50% and at least a 50% reduction from the baseline oral elemental calcium supplement dose at Week 26 was reported. |
Week 26 |
|
| Secondary |
Change From Baseline in Bone Turnover Marker Bone Specific Alkaline Phosphatase at Week 26 |
Bone turnover markers included serum bone-specific alkaline phosphatase, procollagen amino-terminal peptide, C-terminal telopeptide of type 1 collagen, and osteocalcin. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Bone Turnover Marker Type I Collagen C-Telopeptides at Week 26 |
Bone turnover markers included serum bone-specific alkaline phosphatase, procollagen amino-terminal peptide, C-terminal telopeptide of type 1 collagen, and osteocalcin. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Change From Baseline in Bone Turnover Marker Osteocalcin and Procollagen 1 N-Terminal Propeptide at Week 26 |
Bone turnover markers included serum bone-specific alkaline phosphatase, procollagen amino-terminal peptide, C-terminal telopeptide of type 1 collagen, and osteocalcin. A MMRM was used for analysis. |
Baseline, Week 26 |
|
| Secondary |
Number of Participants With Treatment Emergent Adverse Events (TEAEs) |
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs are defined as AEs that started or worsened on or after the date and time of the first dose of investigational product. |
From start of study drug administration to 4 weeks post follow-up (up to Week 36) |
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