The effect of monoallelic variants in the ALPL gene on the natural course of hypophosphatasia (HPP) in children and adults in Russia (ATLANTIS)
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(1) Mean age (in full years) at the HPP diagnosis;
Timeframe: Day 0 (Visit 1)
(2) Mean age at the onset of initial HPP symptoms (including separately any, skeletal, and non-skeletal symptoms);
Timeframe: Day 0 (Visit 1)
(3) Proportions of male and female patients
Timeframe: Day 0 (Visit 1)
(4) Proportions of adults and children at baseline (childhood- and adult-onset HPP);
Timeframe: Day 0 (Visit 1)
(5) Proportion of patients with a family history of HPP in a first-degree relative
Timeframe: Day 0 (Visit 1)
(6) Proportions of patients with specific skeletal and non-skeletal manifestation locations/sites affected at baseline:
Timeframe: Day 0 (Visit 1)
(7) Proportions of patients with history and/or presence of specific skeletal manifestations at baseline:
Timeframe: Day 0 (Visit 1)
(8) Proportions of patients with history and/or presence of specific dental manifestations at baseline:
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Timeframe: Day 0 (Visit 1)
(9) Proportions of patients with history and/or presence of specific muscular manifestations at baseline:
Timeframe: Day 0 (Visit 1)
(10) Proportions of patients with history and/or presence of specific rheumatologic manifestations at baseline:
Timeframe: Day 0 (Visit 1)
(11) Proportions of patients with history and/or presence of specific renal manifestations at baseline:
Timeframe: Day 0 (Visit 1)
(12) Proportions of patients with history and/or presence of specific respiratory manifestations at baseline:
Timeframe: Day 0 (Visit 1)
(13) Proportions of patients with history and/or presence of specific neurologic / psychiatric manifestations at baseline:
Timeframe: Day 0 (Visit 1)
(14) Proportions of patients with history and/or presence of other specific manifestations at baseline:
Timeframe: Day 0 (Visit 1)
(15) Median Standard Deviation Score (SDS) for height at baseline (for patients aged <18 years at the respective timepoints only);
Timeframe: Day 0 (Visit 1)
(16) Proportion of patients with short stature at baseline (SDS for height <-2.0);
Timeframe: Day 0 (Visit 1)
(17) Proportions of patients with other non-specific comorbidities at baseline;
Timeframe: Day 0 (Visit 1)
(18) Proportions of patients with a history and/or presence of specific radiologic signs (X-ray of both hands, posteroanterior view on a single film including the distal forearms) at baseline:
Timeframe: Day 0 (Visit 1)
(19) Proportions of patients with history and/or presence of specific radiologic signs (radiography of both lower limbs, posteroanterior view on a single full-length film, including the distal lower legs) at baseline:
Timeframe: Day 0 (Visit 1)
(20) Proportions of patients with history and/or presence of specific radiologic signs on standing spine radiographs in anteroposterior and lateral views (preferably full-length) at baseline
Timeframe: Day 0 (Visit 1)
(21) Proportions of patients with history and/or presence of specific radiologic signs on skull radiographs in anteroposterior and lateral views at baseline:
Timeframe: Day 0 (Visit 1)
(22) Proportions of patients with history and/or presence of specific radiologic signs on foot radiographs in anteroposterior and lateral views (in the presence of clinical signs of a pathological "march" fracture) at baseline:
Timeframe: Day 0 (Visit 1)
(23) Median Rickets Severity Scale (RSS) at baseline (only for children β€14 years at the respective timepoints);
Timeframe: Day 0 (Visit 1)
(24) Proportions of patients with history and/or presence of specific laboratory findings at baseline:
Timeframe: Day 0 (Visit 1)
(25) a Mean and median values of specific laboratory findings at baseline:
Timeframe: Day 0 (Visit 1)
(26) Proportions of patients with specific ALPL gene mutations (for each gene variant);
Timeframe: Day 0 (Visit 1)
(27) Mean and median number of fractures at baseline;
Timeframe: Day 0 (Visit 1)
(28) Mean and median number of dental losses at baseline;
Timeframe: Day 0 (Visit 1)
(29) Mean and median 6-Minute Walk Test (6MWT) distance (m) at baseline (only for patients β₯5 years);
Timeframe: Day 0 (Visit 1)
(30) Proportion of patients with decreased 6MWT distance (m) at baseline (<80% of the predicted norm, only for patients β₯5 years);
Timeframe: Day 0 (Visit 1)
(31) Mean and median Chair-Rise Test time (s) at baseline (only for patients β₯18 years);
Timeframe: Day 0 (Visit 1)
(32) Mean and median Timed Up and Go (TUG) Test time (s) at baseline (only for adults β₯18 years);
Timeframe: Day 0 (Visit 1)
(33) a Mean and median values of specific bioimpedance parameters at baseline
Timeframe: Day 0 (Visit 1)
(34) a Mean and median Rating of Perceived Exertion (RPE) score, based on Borg Scale, during the following specific tests at baseline (when the respective tests have been performed):
Timeframe: Day 0 (Visit 1)
(35) Mean and median weekly physical activity duration, measured using the International Physical Activity Questionnaire (IPAQ) Diary, at baseline
Timeframe: Day 0 (Visit 1)
(36) Mean and median Pediatric Quality of Life Inventory (PedsQL) score at baseline (only for children <18 years);
Timeframe: Day 0 (Visit 1)
(37) Proportion of patients with different levels of European Quality of Life 5 Dimensions 3-Level (EQ-5D-3L) questionnaire for each of the following dimensions at baseline
Timeframe: Day 0 (Visit 1)
(38) Mean and median EQ-5D-3L Visual Analogue Scale (VAS) score at baseline (only for adults aged β₯ 18 years);
Timeframe: Day 0 (Visit 1)
(39) Proportion of patients with different Modified Ashworth Scale (MAS) scores (0, 1, 1+, 2, 3, or 4) for each specific muscle / muscle group at baseline:
Timeframe: Day 0 (Visit 1)
(40) Proportion of patients with clinically significant spasticity (MAS score β₯2 in at least one muscle / muscle group) at baseline;
Timeframe: Day 0 (Visit 1)
(41) Mean and median dynamic component (D) measure based on the modified Tardieu Scale (MTS) on the for each specific muscle / muscle group at baseline (degrees):
Timeframe: Day 0 (Visit 1)
(42) Proportion of patients with different MTS Resistance scores (0, 1, 2, 3, 4, or 5) for each specific muscle / muscle group at baseline:
Timeframe: Day 0 (Visit 1)
(43) Proportion of patients with clonus (MTS Resistance score β₯3 in at least one muscle / muscle group) at baseline;
Timeframe: Day 0 (Visit 1)
(44) Proportion of patients with previous hospitalizations due to HPP manifestations at baseline;
Timeframe: Day 0 (Visit 1)
(45) Median number of previous hospitalizations due to HPP manifestations at baseline
Timeframe: Day 0 (Visit 1)
(46) Annualized rate of hospitalizations due to HPP manifestations prior to baseline
Timeframe: Day 0 (Visit 1)
(47) Mean and median duration of previous hospitalizations due to HPP manifestations at baseline
Timeframe: Day 0 (Visit 1)
(48) Proportions of patients with different degrees of disability according to national criteria at baseline
Timeframe: Day 0 (Visit 1)
(49) Proportions of patients who had specific previous medical interventions & surgeries at baseline:
Timeframe: Day 0 (Visit 1)
(50) Proportions of patients who previously received specific treatments at baseline:
Timeframe: Day 0 (Visit 1)
(34) b Mean and median Rating of Perceived Exertion (RPE) score, based on Borg Scale, during the following specific tests at baseline (when the respective tests have been performed):
Timeframe: Day 0 (Visit 1)
(34) c Mean and median Rating of Perceived Exertion (RPE) score, based on Borg Scale, during the following specific tests at baseline (when the respective tests have been performed):
Timeframe: Day 0 (Visit 1)
(33) b Mean and median values of specific bioimpedance parameters at baseline
Timeframe: Day 0 (Visit 1)
(33) c Mean and median values of specific bioimpedance parameters at baseline
Timeframe: Day 0 (Visit 1)
(25) b Mean and median values of specific laboratory findings at baseline:
Timeframe: Day 0 (Visit 1)
(25) c Mean and median values of specific laboratory findings at baseline:
Timeframe: Day 0 (Visit 1)
(25) d Mean and median values of specific laboratory findings at baseline:
Timeframe: Day 0 (Visit 1)
(25) e Mean and median values of specific laboratory findings at baseline:
Timeframe: Day 0 (Visit 1)
(25) f Mean and median values of specific laboratory findings at baseline:
Timeframe: Day 0 (Visit 1)
(25) g Mean and median values of specific laboratory findings at baseline:
Timeframe: Day 0 (Visit 1)
(25) h Mean and median values of specific laboratory findings at baseline:
Timeframe: Day 0 (Visit 1)