Pediatric Pulmonary HypertensionNCT02909608
Actigraphy in Pediatric Pulmonary Hypertension
An observational study of Pediatric Pulmonary Hypertension, run by University of Colorado, Denver at 2 sites in United States.
Trial summary
Physical activity is an important factor in understanding how diseases can affect a child. Decreases in physical activity are sometimes the first thing that happens before a child is diagnosed with a disease. When a child sees their doctor, that visit reflects a single point in time. Read more about this study
- Who is it for?
- Ages 8 to 14 yearsHealthy volunteers can take part
- Conditions the study is for
- Pediatric Pulmonary Hypertension
- How many people
- 116 participants
- Where
- 2 sitesUnited States
- Study dates
- 7 Oct 2016to 2 Apr 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 14 years
- All sexes
- Healthy volunteers accepted
- Child
Study population: We will include children with pulmonary hypertension and children without pulmonary hypertension into this study. All enrolled children must be between the ages of 8-14 years at the time of enrollment.
Who may be able to join
- Ages 8-14 years at the time of consent
- Current diagnosis of pulmonary hypertension in WHO Diagnostic Group 1 as per established clinical criteria
- Ages 8-14 years at the time of consent
- Absence of significant cardiopulmonary disease as per medical history
Who cannot join
- Current disease severity of Panama functional class IIIb or IV
- Any bone, neuromuscular, or other pathology that may limit activity
- Use of any medications known to limit activity
- Active infection, or has any of the following:
- cardiovascular,
- liver,
Show all 19Show fewer
- renal,
- hematologic,
- gastrointestinal,
- immunologic,
- endocrine,
- metabolic, or
- central nervous system
- disease or condition that, in the opinion of the Investigator, may adversely affect the safety of the subject or interfere with the interpretation of study assessments.
- Actively listed for transplantation
- Subject and/or legal guardian has/have
- an unstable psychiatric condition or
- is/are mentally incapable of understanding the objectives, nature, or consequences of the trial, or
- has any condition in which the Investigator's opinion would constitute an unacceptable risk to the subject's safety.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
University of Colorado
Aurora, Colorado, 80045
About this study
Official title: Investigation of Actigraphy, an Exercise Measurement Device, as a Novel, Well-defined, Reliable, Feasible, Easy to Use, and Non-Invasive Study Endpoint to Facilitate Pediatric Pulmonary Arterial Hypertension Trials and Drug Development
Physical activity is an important factor in understanding how diseases can affect a child. Decreases in physical activity are sometimes the first thing that happens before a child is diagnosed with a disease. When a child sees their doctor, that visit reflects a single point in time. It does not capture how a child feels during the days in between visits to their doctor. This study plans to use special monitors called actigraphs to collect information about physical activity in children with a type of disease called pulmonary hypertension. This study will measure how active these children are and compare their activity to clinical information and to actigraphy measurements in children without pulmonary hypertension.
Read the detailed description
Pediatric pulmonary arterial hypertension (PAH) (roughly 1-5 cases per 1 million children) is a severe disorder with high mortality and morbidity, but limited treatment options. Pediatric PAH is defined the same as that in adults, which is the presence of abnormally high pulmonary artery pressure. In comparison with adults, pediatric PAH is likely a more severe disorder with higher mortality and morbidity without treatment. Except for INOmax for persistent pulmonary hypertension of the newborn, no drug is currently approved in the US to treat pediatric PAH patients 1-17 years of age. Drug development to treat pediatric PAH is an unmet public health need for children and a priority for the Food and Drug Administration (FDA). However, studies that address the safety and efficacy of PAH therapies are rare, in part due to the lack of suitable clinical endpoints and quantitative and qualitative measures of disease severity.
A major limitation towards enhancing outcomes of children with PAH is the lack of pediatric efficacy endpoints or surrogate measures that are capable of reproducibly and reliably reflecting changes in pulmonary arterial pressure in response to a therapeutic intervention that is being assessed in a pediatric clinical trial. Children are often too young and developmentally unable to perform standard cardiopulmonary exercise testing and the use of several metrics are not as accurate for reflecting clinical status in children as in adults, such as 6 minute walking distance. Additionally, the use of cardiac catheterization to directly measure pulmonary vascular resistance is invasive, requires anesthesia, and has additional risks for complications.
Therefore, to address this critical and unmet medical need in children with PAH, we propose to begin develop a novel, developmentally-appropriate non-invasive endpoint in children through the use of actigraphy. Actigraphy is a mobile device that directly, reproducibly and non-invasively measures physical activity, which can be readily assessed in the ambulatory setting, and may provide a novel, simple and inexpensive approach. Impaired exercise tolerance is a prominent feature of PAH and contributes significantly to reduced quality of life. Assessing exercise capacity is an integral part of the clinical evaluation of PAH in adults and the use of the 6 minute walk distance (6MWD) is the most common primary endpoint in adult PAH clinical trials. Importantly, the 6MWD test and other existing exercise performance tests that are readily applied in adult studies are not reliable and applicable for young children and infants. We propose that actigraphy may provide a novel endpoint for assessing drug efficacy in children if proven to be strongly predictive and reflect clinical course and outcomes of children with PAH. If successful, this early study has great potential for having a significant regulatory impact that will advance the public health mission, as actigraphy could possibly become an endpoint that would be accepted in pediatrics as a regulatory standard for PAH clinical trials.
The purpose of the current study is to describe the use of actigraphy in children with PAH and to determine if correlations exist between actigraphy data and clinical data in children with PAH.
- Conditions
- Pediatric Pulmonary Hypertension
- Being tested
- No interventions / observations only
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventions / observations onlyOther
Also called There are no interventions as part of this study
This is an observational study only
| Study group | Role | What they receive |
|---|---|---|
| Controls | Not described Receives: Other: No interventions / observations only | |
| Children With Pulmonary Hypertension | Not described Receives: Other: No interventions / observations only |
What is being measured
The primary outcome is the main question the study is designed to answer.
Investigate and define the utility of the measurement properties of actigraphy in children with PAH: Baseline
Children will wear both actigraphy devices for 2 weeks at 2 different time points. The first time point is at Baseline when the child enters the study. The second time point is at Week 26, which is 26 weeks after the Baseline timepoint.
Measured: Baseline
Investigate and define the utility of the measurement properties of actigraphy in children with PAH: Week 26
Children will wear both actigraphy devices for 2 weeks at 2 different time points. The first time point is at Baseline when the child enters the study. The second time point is at Week 26, which is 26 weeks after the Baseline timepoint.
Measured: Week 26
Secondary outcomes (1)
Use statistical analysis to determine correlations of actigraphy with disease severity, progression, clinical worsening, and survival, based on data from this prospective clinical study.
Children with PH will have information collected from their medical charts that will be compared to their actigraphy data
Measured: For 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2016 (estimated)
- Study start7 Oct 2016
- Primary completion31 Mar 2020
- Study completion2 Apr 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Pediatric Pulmonary HypertensionStudies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 116 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dunbar Ivy, MD, Principal investigator, University of Colorado, Denver
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02909608
- Sponsor's protocol ID
- 16-1422
- Status last verified
- Jun 2021
- First submitted
- 16 Sep 2016
- Last updated
- 7 Jun 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Kleppinger C, Ivy D, Stockbridge N, Bates A, Handler S, Krishnan US, Mullen MP, Yung D, Hopper RK, Varghese NP, Fineman J, Austin ED, Avitabile CM, Freire G, Clark J, Sun H. Procedural Variation May Contribute to 6-Minute Walk Distance Variability in Real-World Pediatric Pulmonary Arterial Hypertension Study. AAPS J. 2025 Jun 27;27(5):114. doi: 10.1208/s12248-025-01098-7.
PubMed 40579609Sun H, Stockbridge N, Ivy DD, Clark J, Bates A, Handler SS, Krishnan US, Mullen MP, Yung D, Hopper RK, Varghese NP, Avitabile CM, Fineman J, Austin ED, Freire G. Actigraphy Study Endpoints to Reduce Sample Size and Facilitate Drug Development for Pediatric Pulmonary Arterial Hypertension. J Pediatr. 2025 Feb;277:114383. doi: 10.1016/j.jpeds.2024.114383. Epub 2024 Nov 1.
PubMed 39489285
Study at a glance
From the registry record, last updated by the sponsor on 7 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigation of Actigraphy, an Exercise Measurement Device, as a Novel, Well-defined, Reliable, Feasible, Easy to Use, and Non-Invasive Study Endpoint to Facilitate Pediatric Pulmonary Arterial Hypertension Trials and Drug Development
- ClinicalTrials.gov ID
- NCT02909608
- Acronym / sponsor ID
- 16-1422
- Conditions
- Pediatric Pulmonary Hypertension
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 116 participants (actual)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Study start
- 7 Oct 2016 (actual)
- Primary completion
- 31 Mar 2020 (actual)
- Study completion
- 2 Apr 2020 (actual)
- First posted
- 21 Sep 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Physical activity is an important factor in understanding how diseases can affect a child. Decreases in physical activity are sometimes the first thing that happens before a child is diagnosed with a disease. When a child sees their doctor, that visit reflects a single point in time. It does not capture how a child feels during the days in between visits to their doctor. This study plans to use special monitors called actigraphs to collect information about physical activity in children with a type of disease called pulmonary hypertension. This study will measure how active these children are and compare their activity to clinical information and to actigraphy measurements in children without pulmonary hypertension.
Detailed description
Pediatric pulmonary arterial hypertension (PAH) (roughly 1-5 cases per 1 million children) is a severe disorder with high mortality and morbidity, but limited treatment options. Pediatric PAH is defined the same as that in adults, which is the presence of abnormally high pulmonary artery pressure. In comparison with adults, pediatric PAH is likely a more severe disorder with higher mortality and morbidity without treatment. Except for INOmax for persistent pulmonary hypertension of the newborn, no drug is currently approved in the US to treat pediatric PAH patients 1-17 years of age. Drug development to treat pediatric PAH is an unmet public health need for children and a priority for the Food and Drug Administration (FDA). However, studies that address the safety and efficacy of PAH therapies are rare, in part due to the lack of suitable clinical endpoints and quantitative and qualitative measures of disease severity.
A major limitation towards enhancing outcomes of children with PAH is the lack of pediatric efficacy endpoints or surrogate measures that are capable of reproducibly and reliably reflecting changes in pulmonary arterial pressure in response to a therapeutic intervention that is being assessed in a pediatric clinical trial. Children are often too young and developmentally unable to perform standard cardiopulmonary exercise testing and the use of several metrics are not as accurate for reflecting clinical status in children as in adults, such as 6 minute walking distance. Additionally, the use of cardiac catheterization to directly measure pulmonary vascular resistance is invasive, requires anesthesia, and has additional risks for complications.
Therefore, to address this critical and unmet medical need in children with PAH, we propose to begin develop a novel, developmentally-appropriate non-invasive endpoint in children through the use of actigraphy. Actigraphy is a mobile device that directly, reproducibly and non-invasively measures physical activity, which can be readily assessed in the ambulatory setting, and may provide a novel, simple and inexpensive approach. Impaired exercise tolerance is a prominent feature of PAH and contributes significantly to reduced quality of life. Assessing exercise capacity is an integral part of the clinical evaluation of PAH in adults and the use of the 6 minute walk distance (6MWD) is the most common primary endpoint in adult PAH clinical trials. Importantly, the 6MWD test and other existing exercise performance tests that are readily applied in adult studies are not reliable and applicable for young children and infants. We propose that actigraphy may provide a novel endpoint for assessing drug efficacy in children if proven to be strongly predictive and reflect clinical course and outcomes of children with PAH. If successful, this early study has great potential for having a significant regulatory impact that will advance the public health mission, as actigraphy could possibly become an endpoint that would be accepted in pediatrics as a regulatory standard for PAH clinical trials.
The purpose of the current study is to describe the use of actigraphy in children with PAH and to determine if correlations exist between actigraphy data and clinical data in children with PAH.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Controls | – | Other: No interventions / observations only |
| Children With Pulmonary Hypertension | – | Other: No interventions / observations only |
Who may qualify
- Ages
- 8 to 14 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- Study population
- We will include children with pulmonary hypertension and children without pulmonary hypertension into this study. All enrolled children must be between the ages of 8-14 years at the time of enrollment.
Key inclusion criteria (4 listed)
- Ages 8-14 years at the time of consent
- Current diagnosis of pulmonary hypertension in WHO Diagnostic Group 1 as per established clinical criteria
- Ages 8-14 years at the time of consent
- Absence of significant cardiopulmonary disease as per medical history
Key exclusion criteria (19 listed)
- Current disease severity of Panama functional class IIIb or IV
- Any bone, neuromuscular, or other pathology that may limit activity
- Use of any medications known to limit activity
- Active infection, or has any of the following:
- disease or condition that, in the opinion of the Investigator, may adversely affect the safety of the subject or interfere with the interpretation of study assessments.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventions / observations onlyAlso: There are no interventions as part of this studyThis is an observational study only | Other | Children With Pulmonary Hypertension, Controls |
Primary outcome
Investigate and define the utility of the measurement properties of actigraphy in children with PAH: Baseline
Time frame: Baseline
Children will wear both actigraphy devices for 2 weeks at 2 different time points. The first time point is at Baseline when the child enters the study. The second time point is at Week 26, which is 26 weeks after the Baseline timepoint.
Investigate and define the utility of the measurement properties of actigraphy in children with PAH: Week 26
Time frame: Week 26
Children will wear both actigraphy devices for 2 weeks at 2 different time points. The first time point is at Baseline when the child enters the study. The second time point is at Week 26, which is 26 weeks after the Baseline timepoint.
1 secondary outcome
Use statistical analysis to determine correlations of actigraphy with disease severity, progression, clinical worsening, and survival, based on data from this prospective clinical study.
Time frame: For 18 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jun 2021.
- Derived from this study: PubMed 40579609Kleppinger C, Ivy D, Stockbridge N, Bates A, Handler S, Krishnan US, Mullen MP, Yung D, Hopper RK, Varghese NP, Fineman J, Austin ED, Avitabile CM, Freire G, Clark J, Sun H. Procedural Variation May Contribute to 6-Minute Walk Distance Variability in Real-World Pediatric Pulmonary Arterial Hypertension Study. AAPS J. 2025 Jun 27;27(5):114. doi: 10.1208/s12248-025-01098-7.
- Derived from this study: PubMed 39489285Sun H, Stockbridge N, Ivy DD, Clark J, Bates A, Handler SS, Krishnan US, Mullen MP, Yung D, Hopper RK, Varghese NP, Avitabile CM, Fineman J, Austin ED, Freire G. Actigraphy Study Endpoints to Reduce Sample Size and Facilitate Drug Development for Pediatric Pulmonary Arterial Hypertension. J Pediatr. 2025 Feb;277:114383. doi: 10.1016/j.jpeds.2024.114383. Epub 2024 Nov 1.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dunbar Ivy, MD principal investigator
University of Colorado, Denver
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pediatric Pulmonary Hypertension, run by University of Colorado, Denver at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Investigate and define the utility of the measurement properties of actigraphy in children with PAH: Baseline (time frame: Baseline). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02909608, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jun 2021.