Registered outcomes55

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

1 to 20 of 55

List	Outcome	Time frame	Description
Primary	Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs)	Up to Week 56 (Safety Follow-Up +/-7d)	A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration. A severity grade was defined by the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.03. As per CTCAE, Grade 1 scales as Mild; Grade 2 scales as Moderate; Grade 3 scales as severe or medically significant but not immediately life threatening; Grade 4 scales as life-threatening consequences; and Grade 5 scales as death related to AE. Safety Population includes all sentinel and randomized participants in the FAS who received at least one dose of vosoritide or placebo in this study. Serious adverse event (SAE)
Primary	Change From Baseline in Height Z-score at Week 52.	Baseline to Week 52	Z-Scores were derived using age-sex specific reference data (means and SDS) for average stature children per the Centers for Disease Control and Prevention. A height Z score of 0 would indicate that the subject's height is equal to the mean height for the average stature population of the same sex and age. A positive height Z score indicates that the subjects height is above the mean height for the average stature population of the same sex and age, whilst a negative height Z score indicates that the subjects height is below the mean height for the average stature population of the same sex and age. To conclude if the height Z score increases then this means the height deficit has decreased. standard deviation score (SDS). The primary efficacy analysis population was the subset of randomized participants in the FAS.
Secondary	Change From Baseline in Height at Week 52	Baseline to Week 52	As a general rule, standing height/sitting height was used if participants were aged \>= 24 months at baseline. Body length/crown to rump was used if participants were aged \< 24 months at baseline. If body length was not measured and standing height was available, standing height was used.
Secondary	Change From Baseline in Annualized Growth Velocity (AGV) at Week 52	Baseline to Week 52	AGV was derived over 12-month intervals starting from the baseline visit. Annualized growth velocity (AGV) = Standing Height at Date 2 - Standing Height at Date 1/Interval Length (Days) x 365.25. Difference in LS means were obtained from an analysis of covariance model.
Secondary	Change From Baseline in Upper to Lower Body Segment Ratio at Week 52	Baseline to Week 52	Upper to Lower Body ratio=Sitting Height / (Standing Height - Sitting Height).
Secondary	Change From Baseline in Other Growth Measures (Upper Body Length, Head Circumference, Arm Span, Upper Arm Length, Lower Arm Length, Lower Body Length, Upper Leg Length, Knee to Heel Length, and Tibial Length) at Week 52	Baseline to Week 52	Change from baseline in other growth measures (upper body length, head circumference, arm span, upper arm length, lower arm (Forearm) length, Lower Body Length, Upper Leg Length (Thigh), knee to heel length, and tibial length) at Week 52. Participants aged \<24 months, body length and crown to rump length take precedence over standing height and sitting height. Participants aged \<24 months at baseline and \>=24 months at Week 52, body length and crown to rump length take precedence.
Secondary	Change From Baseline in Other Body Proportion Ratios (Arm Span to Height Ratio, Upper Arm Length to Lower Arm [Forearm] Length Ratio, Upper Leg Length [Thigh] to Knee to Heel Length Ratio, and Upper Leg Length (Thigh) to Tibial Length Ratio) at Week 52	Baseline to Week 52	For Height: subjects aged \< 24 months, body length takes precedence over standing height. Subjects aged \< 24 months at baseline and \>= 24 months at Week 52, body length takes precedence.
Secondary	Change From Baseline in Body Mass Index (BMI) at Week 52	Baseline to Week 52	Body Mass Index (BMI): For height used for BMI calculation, participants aged \< 24 months, body length takes precedence over standing height. Participants aged \< 24 months at baseline and \>= 24 months at Week 52, body length takes precedence.
Secondary	Change From Baseline in BMI Z-score at Week 52	Baseline to Week 52	The body mass index (BMI) Z-score represents the participant's BMI converted to an age and sex appropriate standard deviation score (SDS) by comparison with an average stature reference population. The weight Z-score of 0 represents the mean BMI for age and sex of the reference population. A positive BMI Z-score means BMI is above the mean BMI for the average stature population of the same sex and age and a negative BMI Z-Score means BMI is below the mean BMI for the average stature population of the same sex and age. BMI Z-scores are derived only for participants aged 24 months or older, the change from baseline to Week 52 in BMI Z-score is summarized for Cohort 1 only as no participants in Cohort 2 or Cohort 3 were 24 months at baseline.
Secondary	Change From Baseline in Weight Z-score at Week 52	Baseline to Week 52	The weight Z-score represents the participant's weight converted to an age and sex appropriate standard deviation score (SDS) by comparison with an average stature reference population. The weight Z-score of 0 represents the mean weight for age and sex of the reference population. A positive weight Z-score means weight is above the mean weight for the average stature population of the same sex and age and a negative weight Z-Score means weight is below the mean weight for the average stature population of the same sex and age.
Secondary	Change From Baseline in Infant Toddler Quality of Life (ITQoL) Scores at Week 52	Baseline to Week 52	97(ITQOL) item full-length version was used. For each concept, item responses are scored, summed, \& transformed on a scale from 0(worst health) to 100(best health).
Secondary	Change From Baseline in ITQoL-Behavior Scores at Week 52	Baseline to Week 52	97(ITQOL) item full-length version was used. For each concept, item responses are scored, summed, \& transformed on a scale from 0(worst health) to 100(best health). The number of subjects assessed in Cohort 3 Placebo is 0 as assessment was not done or overall score could not be derived because no more than half assessments were done at Screening/ baseline. Hence, Mean and Standard Deviation (SD) are not applicable. Note: For each variable, there are different numbers of individual questions. The variable can only be derived if at least half of the questions were completed.
Secondary	Change From Baseline in ITQoL-Global Behavior Scores at Week 52	Baseline to Week 52	97(ITQOL) item full-length version was used. For each concept, item responses are scored, summed, \& transformed on a scale from 0(worst health) to 100(best health). A higher score indicates better global behaviour. The number of subjects assessed in Cohort 3 Placebo is 0 as assessment was not done or overall score could not be derived because no more than half assessments were done at Screening/ baseline. Hence, Mean and Standard Deviation (SD) are not applicable. Note: For each variable, there are different numbers of individual questions. The variable can only be derived if at least half of the questions were completed.
Secondary	Change From Baseline in ITQoL-Getting On With Other Score at Week 52	Baseline to Week 52	97(ITQOL) item full-length version was used. For each concept, item responses are scored, summed, \& transformed on a scale from 0(worst health) to 100(best health). A higher score indicates better global health perceptions. The number of subjects assessed in Cohort 3 Placebo and Cohort 3 vosoritide are 0 as assessment was not done or overall score could not be derived because no more than half assessments were done at Screening/baseline. Hence, Mean and Standard Deviation (SD) are not applicable. Note: For each variable, there are different numbers of individual questions. The variable can only be derived if at least half of the questions were completed.
Secondary	Change From Baseline in ITQoL-Health Score at Week 52	Baseline to Week 52	97(ITQOL) item full-length version was used. For each concept, item responses are scored, summed, \& transformed on a scale from 0(worst health) to 100(best health). A higher score indicates better global change in health. The number of subjects assessed in Cohort 3 Placebo is 0 as assessment was not done or overall score could not be derived because no more than half assessments were done at Screening/ baseline. Hence, Mean and Standard Deviation (SD) are not applicable. Note: For each variable, there are different numbers of individual questions. The variable can only be derived if at least half of the questions were completed.
Secondary	Change From Baseline in Functional Independence Measure for Children (WeeFIM-II) Score at Week 52	Baseline to Week 52	The Pediatric Functional Independence Measure-II (WeeFIM®-II) is designed to measure functional independence of children between the ages of 6 months \& 18 yrs who have physical or general developmental limitations. The WeeFIM-II is comprised of 3 domains that are rated by clinicians based on information obtained from parents/caregivers (self-care \[score range 8 to 56\], mobility \[score range 8 to 35\], \& cognition \[score range 8 to 35\]) \& provides a total score between 18 (worst) \& 126 (Best). Higher score reflects higher level of independence. Performance of a child on each of individual items within WeeFIM is assigned to one of seven levels on an ordinal scale that represent function from complete \& modified independence (levels 7 \& 6) without a helping person to modified \& complete dependence (levels 5 to 1) with a helping person. Functional Independence Measure (Wee-FIM-II) is only validated in children ages 6 months to 18 yrs. Therefore results for Cohort 3 is not summarized.
Secondary	Change From Baseline in Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Scores at Week 52	Baseline to Week 52	BSID-III is performance-based clinician-reported outcome assessment for use in children from 1 to 42 months (1 month to 3.5 years). individually administered by the trained clinician to the participant/child Scales include Cognitive subscale, Receptive and Expressive subscales, and Gross and Fine Motor subscales. The two language scales make up a composite Language Scale score and the Gross and Fine Motor subscales yield a composite Motor Scale score. In addition, there is a Social-Emotional Scale and Adaptive Behavior Scale, which is a questionnaire read and completed by parent or caregiver. Scores range from 0 to 2, and indicate mastery (2), emerging (1) or zero (not present) Each (sub)scale yields a total raw score, standardized according to the participant's chronological age (scaled scores)
Secondary	Change From Baseline in Child Behaviour Checklist (CBCL) at Week 52	Baseline to Week 52	The CBCL is used as a screening tool for emotional or behavioral problems, measuring initial behavior severity, tracking changes in behavioral problems over the course of treatment, and treatment planning. The CBCL 1.5-5 years old consists of 100 questions, scored on a three-point Likert scale (0=Not True (as far as you know), 1= Somewhat or Sometimes True, 2=Very True or Often True). The scores for each individual question within a domain are summed to give a domain score. The CBCL uses a normative sample to create standard scores, which are scaled so that 50 is average with a standard deviation of 10, where higher scores indicate more emotional or behavioral issues. CBCL was waived for children \< 18 months old. Therefore results for Cohort 3 is not summarized.
Secondary	Change From Baseline in Bilateral X-rays of Lower Extremities at Week 52	Baseline to Week 52
Secondary	Change From Baseline in Bilateral X-rays of Left/Right Tibia Bowing Angle at Week 52	Baseline to Week 52

Source ClinicalTrials.govtrials-2026-11-12-0.5.0

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

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ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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Registered outcomes55

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