Duchenne Muscular DystrophyNCT04740554
Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy, run by University of Sao Paulo.
Trial summary
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo (UNIFESP). Read more about this study
- Who is it for?
- Ages 11 to 18 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Duchenne Muscular Dystrophy group with Deflazacort, Duchenne Muscular Dystrophy group with Prednisone/Predisolone and Duchenne Muscular Dystrophy group without Corticosteroids therapy
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- 1 Mar 2013to 1 Feb 2015
- 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 11 to 18 years
- Men only
- Healthy volunteers accepted
- Child, Adult
Study population: 30 boys with Duchenne Muscular Dystrophy and 10 typically developing boys aged 11 to 18 years.
Who may be able to join
- Individuals diagnosed with DMD confirmed by molecular method and / or by protein expression of skeletal muscle.
- Individuals undergoing clinical follow-up at the outpatient clinic for neuromuscular diseases at the Federal University of São Paulo (UNIFESP)
- Individuals who had authorization from their parents or guardians to participate in the study
Who cannot join
- Patients with cardiac arrhythmias.
- Patients with atrioventricular block.
- Patients with congenital anomalies such as congenital heart defects, pulmonary deformity.
- Patients using drugs that interfere with ANS, such as antiarrhythmic agents and drugs for the treatment of diabetes mellitus, such as insulin.
About this study
Official title: Characterization of Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy - Influence of Corticosteroids and Betablockers
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo (UNIFESP). The recruited individuals were divided into 4 groups, namely: DMD that used deflazacort (DMD-D); DMD that used Prednisone/Prednisolone (DMD-P); DMD Control with no corticoid use (DMD-C) and Controls with typical development (CTD). The protocol was applied during the evaluation that was carried out at outpatient follow-up visits.
To assess the functionality of each patient, the Vignos scales were used to characterize the sample and the Motor Function Measure (MFM) for association with HRV indices.
All heart rate records were performed using a cardiofrequencymeter (V800, Polar). After placing the brace and monitor, the individuals were placed in the supine position and remained at rest spontaneously breathing for 25 minutes. For HRV analysis, indexes obtained by linear methods, in the domain of time and frequency, and non-linear methods were used.
Read the detailed description
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo (UNIFESP). The recruited individuals were divided into 4 groups, namely: DMD that used deflazacort (DMD-D) with n=11; DMD that used Prednisone/Prednisolone (DMD-P), with n=9; DMD Control with no corticoid use (DMD-C), and n=10 and Controls with typical development (CTD) with n=10. The protocol was applied during the evaluation that was carried out at outpatient follow-up visits. Anthropometry was analyzed in 4 groups and functionality was assessed in 3 groups with DMD. To assess the functionality of each patient, the Vignos scales were used to characterize the sample and the Motor Function Measure (MFM) for association with HRV indices.
Initially, the resting ECG was analyzed to verify the existence of sinus rhythm and to exclude individuals with arrhythmias and blocks. The measurements at rest were performed immediately before and after the HRV assessment, including systolic (SBP) and diastolic (DBP), heart rate (HR), Respiratory Rate (RF - blood pressure) to ensure that at the beginning and at the end of the collection FR remains between 9 - 24rpm, in the range of 0.15 - 0.40Hz) and partial oxygen saturation (SO2). Heart rate was recorded by the cardiofrequency meter (RS800CX, Polar). And the partial oxygen saturation by means of a digital oximeter (DX2010, Dixtal) connected to the participant's index finger or hallux, through a sensor of age-appropriate size, in room air. Hemoglobin saturation by oxygen was recorded after the first minute of stabilization, as the value that remains most constant during the second minute.
All heart rate records were performed using a cardiofrequencymeter (V800, Polar). After placing the brace and monitor, the individuals were placed in the supine position and remained at rest spontaneously breathing for 25 minutes. For HRV analysis, indexes obtained by linear methods, in the domain of time and frequency, and non-linear methods were used.
In the time domain, for linear analyses, each normal RR interval (sinus beats) was verified during a certain time interval and, using statistical and non-linear methods, the translating indexes of fluctuations in the duration of the cardiac cycles were calculated. With this, the indices expressed in ms were obtained: SDNN (Standard deviation of the normal RR intervals recorded in a time interval); rMSSD (square root of the mean of the square of the differences between adjacent normal RR intervals in a time interval).
In the frequency domain, the spectral power density is more used, mainly when treating individuals in resting conditions. This analysis divides HRV into fundamental oscillatory components, which were used the main: High Frequency normalized unite (HFnu) component and Low Frequency normalized unite (LFnu) component. O algoritmo utilizado para a análise espectral foi a transformada rápida de Fourier - FFT (janela de 256 s com 50% de overlap).
Among the nonlinear methods used for HRV analysis, we can mention the Detrended Fluctuations Analysis (DFA), Visual Recurrence Analysis (VRA) and symbolic analysis (SA), the three of which were verified in this study.
DFA is used to quantify the fractal property of RR interval time series, being used to detect possible abnormalities present in a subject, based on α coefficients. For this, short memory parameters α1, which corresponds to a period of 4 to 11 beats, and long memory α2, which corresponds to the period from 64 to 1024 beats, were used.
The VRA is used to study the time dependence of a series, that is, in the study of stationarity35. The recurrence graph makes it possible to visualize the behavior of trajectories in the phase space and, in addition, to show the times in which a state of a dynamic system is repeated, besides these factors can be confirmed regarding the quantitative analysis of this, wich presents such indices: Mean, SD, PerRec, PerDet, PerLam, TrapTim, Ratio.
The evaluation of the symbolic analysis is based on the quantification of the information carried in a time series, in the transformation of the previously selected iRRs into integers from zero to six, from which sequences of 3 symbols (symbolic patterns) are constructed. For this, all possible patterns will be grouped according to the number and type of variations between the symbols, shown subsequently. The patterns were (1) patterns, without variation (0V), three identical symbols; (2) patterns with a variation (1V), that is, two subsequent symbols that are the same and a different one; (3) patterns with two similar variations (2LV) that is, the three symbols form a ramp; (4) patterns with two different variations (2ULV), that is, the three symbols form a peak or a valley.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Duchenne Muscular Dystrophy group with Deflazacort, Duchenne Muscular Dystrophy group with Prednisone/Predisolone, Duchenne Muscular Dystrophy group without Corticosteroids therapy, Control Group Typically Developing
- Keywords
- Muscular Dystrophy, Duchenne, Prednisone, Deflazacort
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Duchenne Muscular Dystrophy group with DeflazacortBehavioural
Subjects with duchenne muscular dystrophy undergoing drug therapy with Deflazacort
Duchenne Muscular Dystrophy group with Prednisone/PredisoloneBehavioural
Subjects with duchenne muscular dystrophy undergoing drug therapy with Prednisona/Predinisolone
Duchenne Muscular Dystrophy group without Corticosteroids therapyBehavioural
Control group with duchenne muscular dystrophy without the use of corticosteroid drug therapy.
Control Group Typically DevelopingBehavioural
Control group with tipical development.
| Study group | Role | What they receive |
|---|---|---|
| Duchenne Muscular Dystrophy group with Deflazacort | Individuals Duchenne Muscular Dystrophy, with age 11 to 18 years which make use of deflazacort. Receives: Behavioral: Duchenne Muscular Dystrophy group with Deflazacort | |
| Duchenne Muscular Dystrophy group with Prednisone/Predisolone | Individuals Duchenne Muscular Dystrophy, with age 11 to 18 years which make use of Prednisone/Predinisolone. Receives: Behavioral: Duchenne Muscular Dystrophy group with Prednisone/Predisolone | |
| Duchenne Muscular Dystrophy group without Corticosteroids therapy | Individuals Duchenne Muscular Dystrophy, with age 11 to 18 years which don't use of corticosteroids. Receives: Behavioral: Duchenne Muscular Dystrophy group without Corticosteroids therapy | |
| Control Group Typically Developing | Individuals with typical development age 11 to 18 years which don't use of corticosteroids. Receives: Behavioral: Control Group Typically Developing |
What is being measured
The primary outcome is the main question the study is designed to answer.
Heart Rate Variability in adolescents with Duchenne Muscular Dystrophy undergoing therapy with corticosteroids
Heart rate variability indices at rest in adolescents with duchenne muscular dystrophy will be analyzed, which will be divided into the following groups: Under the use of Deflazacort, Predinisone / Predinisolone and without the use of corticosteroids, in addition to the analysis of a control group with typical development.
Measured: One day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2021
- Study start1 Mar 2013
- Primary completion1 Sep 2014
- Study completion1 Feb 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University of Sao PauloThe 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
- Cross-sectional
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Sao Paulo
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Talita Dias da Silva, University of Sao Paulo
- Investigators
- Talita D da Silva, Ph.D., Principal investigator, Universidade Federal de São Paulo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04740554
- Sponsor's protocol ID
- 09942913.4.0000.5505
- Status last verified
- Feb 2021
- First submitted
- 1 Feb 2021
- Last updated
- 5 Feb 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Characterization of Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy - Influence of Corticosteroids and Betablockers
- ClinicalTrials.gov ID
- NCT04740554
- Acronym / sponsor ID
- 09942913.4.0000.5505
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Muscular Dystrophy, Duchenne, Prednisone, Deflazacort
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- University of Sao PauloUniversity, hospital or other
- Study start
- 1 Mar 2013 (actual)
- Primary completion
- 1 Sep 2014 (actual)
- Study completion
- 1 Feb 2015 (actual)
- First posted
- 5 Feb 2021 (actual)
- 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.
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo (UNIFESP). The recruited individuals were divided into 4 groups, namely: DMD that used deflazacort (DMD-D); DMD that used Prednisone/Prednisolone (DMD-P); DMD Control with no corticoid use (DMD-C) and Controls with typical development (CTD). The protocol was applied during the evaluation that was carried out at outpatient follow-up visits.
To assess the functionality of each patient, the Vignos scales were used to characterize the sample and the Motor Function Measure (MFM) for association with HRV indices.
All heart rate records were performed using a cardiofrequencymeter (V800, Polar). After placing the brace and monitor, the individuals were placed in the supine position and remained at rest spontaneously breathing for 25 minutes. For HRV analysis, indexes obtained by linear methods, in the domain of time and frequency, and non-linear methods were used.
Detailed description
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo (UNIFESP). The recruited individuals were divided into 4 groups, namely: DMD that used deflazacort (DMD-D) with n=11; DMD that used Prednisone/Prednisolone (DMD-P), with n=9; DMD Control with no corticoid use (DMD-C), and n=10 and Controls with typical development (CTD) with n=10. The protocol was applied during the evaluation that was carried out at outpatient follow-up visits. Anthropometry was analyzed in 4 groups and functionality was assessed in 3 groups with DMD. To assess the functionality of each patient, the Vignos scales were used to characterize the sample and the Motor Function Measure (MFM) for association with HRV indices.
Initially, the resting ECG was analyzed to verify the existence of sinus rhythm and to exclude individuals with arrhythmias and blocks. The measurements at rest were performed immediately before and after the HRV assessment, including systolic (SBP) and diastolic (DBP), heart rate (HR), Respiratory Rate (RF - blood pressure) to ensure that at the beginning and at the end of the collection FR remains between 9 - 24rpm, in the range of 0.15 - 0.40Hz) and partial oxygen saturation (SO2). Heart rate was recorded by the cardiofrequency meter (RS800CX, Polar). And the partial oxygen saturation by means of a digital oximeter (DX2010, Dixtal) connected to the participant's index finger or hallux, through a sensor of age-appropriate size, in room air. Hemoglobin saturation by oxygen was recorded after the first minute of stabilization, as the value that remains most constant during the second minute.
All heart rate records were performed using a cardiofrequencymeter (V800, Polar). After placing the brace and monitor, the individuals were placed in the supine position and remained at rest spontaneously breathing for 25 minutes. For HRV analysis, indexes obtained by linear methods, in the domain of time and frequency, and non-linear methods were used.
In the time domain, for linear analyses, each normal RR interval (sinus beats) was verified during a certain time interval and, using statistical and non-linear methods, the translating indexes of fluctuations in the duration of the cardiac cycles were calculated. With this, the indices expressed in ms were obtained: SDNN (Standard deviation of the normal RR intervals recorded in a time interval); rMSSD (square root of the mean of the square of the differences between adjacent normal RR intervals in a time interval).
In the frequency domain, the spectral power density is more used, mainly when treating individuals in resting conditions. This analysis divides HRV into fundamental oscillatory components, which were used the main: High Frequency normalized unite (HFnu) component and Low Frequency normalized unite (LFnu) component. O algoritmo utilizado para a análise espectral foi a transformada rápida de Fourier - FFT (janela de 256 s com 50% de overlap).
Among the nonlinear methods used for HRV analysis, we can mention the Detrended Fluctuations Analysis (DFA), Visual Recurrence Analysis (VRA) and symbolic analysis (SA), the three of which were verified in this study.
DFA is used to quantify the fractal property of RR interval time series, being used to detect possible abnormalities present in a subject, based on α coefficients. For this, short memory parameters α1, which corresponds to a period of 4 to 11 beats, and long memory α2, which corresponds to the period from 64 to 1024 beats, were used.
The VRA is used to study the time dependence of a series, that is, in the study of stationarity35. The recurrence graph makes it possible to visualize the behavior of trajectories in the phase space and, in addition, to show the times in which a state of a dynamic system is repeated, besides these factors can be confirmed regarding the quantitative analysis of this, wich presents such indices: Mean, SD, PerRec, PerDet, PerLam, TrapTim, Ratio.
The evaluation of the symbolic analysis is based on the quantification of the information carried in a time series, in the transformation of the previously selected iRRs into integers from zero to six, from which sequences of 3 symbols (symbolic patterns) are constructed. For this, all possible patterns will be grouped according to the number and type of variations between the symbols, shown subsequently. The patterns were (1) patterns, without variation (0V), three identical symbols; (2) patterns with a variation (1V), that is, two subsequent symbols that are the same and a different one; (3) patterns with two similar variations (2LV) that is, the three symbols form a ramp; (4) patterns with two different variations (2ULV), that is, the three symbols form a peak or a valley.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Duchenne Muscular Dystrophy group with Deflazacort | – | Behavioral: Duchenne Muscular Dystrophy group with DeflazacortIndividuals Duchenne Muscular Dystrophy, with age 11 to 18 years which make use of deflazacort. |
| Duchenne Muscular Dystrophy group with Prednisone/Predisolone | – | Behavioral: Duchenne Muscular Dystrophy group with Prednisone/PredisoloneIndividuals Duchenne Muscular Dystrophy, with age 11 to 18 years which make use of Prednisone/Predinisolone. |
| Duchenne Muscular Dystrophy group without Corticosteroids therapy | – | Behavioral: Duchenne Muscular Dystrophy group without Corticosteroids therapyIndividuals Duchenne Muscular Dystrophy, with age 11 to 18 years which don't use of corticosteroids. |
| Control Group Typically Developing | – | Behavioral: Control Group Typically DevelopingIndividuals with typical development age 11 to 18 years which don't use of corticosteroids. |
Who may qualify
- Ages
- 11 to 18 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- 30 boys with Duchenne Muscular Dystrophy and 10 typically developing boys aged 11 to 18 years.
Key inclusion criteria (3 listed)
- Individuals diagnosed with DMD confirmed by molecular method and / or by protein expression of skeletal muscle.
- Individuals undergoing clinical follow-up at the outpatient clinic for neuromuscular diseases at the Federal University of São Paulo (UNIFESP)
- Individuals who had authorization from their parents or guardians to participate in the study
Key exclusion criteria (4 listed)
- Patients with cardiac arrhythmias.
- Patients with atrioventricular block.
- Patients with congenital anomalies such as congenital heart defects, pulmonary deformity.
- Patients using drugs that interfere with ANS, such as antiarrhythmic agents and drugs for the treatment of diabetes mellitus, such as insulin.
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 |
|---|---|---|
| Duchenne Muscular Dystrophy group with DeflazacortSubjects with duchenne muscular dystrophy undergoing drug therapy with Deflazacort | Behavioural | Duchenne Muscular Dystrophy group with Deflazacort |
| Duchenne Muscular Dystrophy group with Prednisone/PredisoloneSubjects with duchenne muscular dystrophy undergoing drug therapy with Prednisona/Predinisolone | Behavioural | Duchenne Muscular Dystrophy group with Prednisone/Predisolone |
| Duchenne Muscular Dystrophy group without Corticosteroids therapyControl group with duchenne muscular dystrophy without the use of corticosteroid drug therapy. | Behavioural | Duchenne Muscular Dystrophy group without Corticosteroids therapy |
| Control Group Typically DevelopingControl group with tipical development. | Behavioural | Control Group Typically Developing |
Primary outcome
Heart Rate Variability in adolescents with Duchenne Muscular Dystrophy undergoing therapy with corticosteroids
Time frame: One day
Heart rate variability indices at rest in adolescents with duchenne muscular dystrophy will be analyzed, which will be divided into the following groups: Under the use of Deflazacort, Predinisone / Predinisolone and without the use of corticosteroids, in addition to the analysis of a control group with typical development.
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 5 Feb 2021.
- 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.
Talita D da Silva, Ph.D. principal investigator
Universidade Federal de São Paulo
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by University of Sao Paulo.
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 3 inclusion and 4 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 5 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
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?
Heart Rate Variability in adolescents with Duchenne Muscular Dystrophy undergoing therapy with corticosteroids (time frame: One day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 Feb 2021.