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Evaluation of Caregiver Burden, Needs, and Expectations of Spinal Muscular Atrophy Parents

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04228718
Recruitment Status : Recruiting
First Posted : January 14, 2020
Last Update Posted : January 31, 2020
Sponsor:
Information provided by (Responsible Party):
Marmara University

Brief Summary:
The purpose of this study is to assess carer burden, needs, and expectations of Spinal Muscular Atrophy Parents

Condition or disease Intervention/treatment
Spinal Muscular Atrophy Caregiver Burden Behavioral: Zarit Burden Interview Scale Behavioral: Family Needs Survey (FNS) Other: Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Other: Hammersmith Functional Motor Scale Expanded (HMFSE)

Detailed Description:

Spinal muscular atrophy (SMA) is a group of neuromuscular diseases involving the spinal cord anterior horn cells and brain stem motor nuclei. It is followed by programmed cell death. It progresses with symmetrical weakness and atrophy of voluntary muscles throughout the body. It leads to a decrease in mobility with progressive loss of power, and pulmonary function impairment with the involvement of the respiratory muscles.

Families who are confronted with these problems that arise after the birth of the baby or shortly after birth have a serious adaptation problem. When the course of the disease and its incurability are explained, patients and their families are under severe stress. Physical and emotional health is worse than normal healthy growing children's parents.

They need information, psychological and social support. The role of the family is very important in the life of children with disabilities. Family-oriented care; has been developed to facilitate the care process of children with special needs and to help their families. The main characteristics of the family-oriented approach are that families know their children in the best way, that each family is unique and different, and that family and community support is provided for the child's functionality. The assessment of family function helps the planning and management of treatment according to the concerns of the family. The education of the family, the socio-cultural structure and the psychological approach of the parents play an important role in the development of the child with SMA.

The aim of this study was to evaluate the care burden, needs and expectations of SMA parents and to reveal the problems clearly; so that this information can be used in rehabilitation planning and interpretation of results.

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Study Type : Observational
Estimated Enrollment : 34 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Evaluation of Caregiver Burden, Needs, and Expectations of Spinal Muscular Atrophy Parents
Actual Study Start Date : July 1, 2019
Estimated Primary Completion Date : January 30, 2020
Estimated Study Completion Date : February 28, 2020



Intervention Details:
  • Behavioral: Zarit Burden Interview Scale
    It is a questionnaire consisting of 22 items. A minimum score of 0 and a maximum score of 88 can be obtained. The higher the scale score, the higher the difficulty experienced.
  • Behavioral: Family Needs Survey (FNS)
    It is a questionnaire consisting of 35 items prepared to determine the requirements. In the FNS survey; The points given to the items can be directly collected and the amount of family needs can be determined over the total score. The lowest score that can be obtained from FNS is 29 and the highest score is 87. As the scores obtained from the scale and subscales increase, it can be said that the families' level of necessity increases.
  • Other: Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)
    CHOP-INTEND uses a scale of 0-64 points. Higher scores indicate better motor function. It is used to reliably measure natural motor function decline in infants with SMA Type 1.
  • Other: Hammersmith Functional Motor Scale Expanded (HMFSE)
    It is an extended version of the 20-item HFMS. Based on another scale known as GMFM, 13 items were added and expanded HFMS to include these elements. Therefore, HFMSE is a scale used to evaluate motor functions in types 2 and 3 SMA. It contains 33 items with a total score of 66 on a scale of 0, 1, 2.


Primary Outcome Measures :
  1. Zarit Burden Interview Scale [ Time Frame: Day 0 ]
    It is a questionnaire consisting of 22 items. A minimum score of 0 and a maximum score of 88 can be obtained. The higher the scale score, the higher the difficulty experienced.

  2. Family Needs Survey (FNS) [ Time Frame: Day 0 ]
    It is a questionnaire consisting of 35 items prepared to determine the requirements. In the FNS survey; The points given to the items can be directly collected and the amount of family needs can be determined over the total score. The lowest score that can be obtained from FNS is 29 and the highest score is 87. As the scores obtained from the scale and subscales increase, it can be said that the families' level of necessity increases.


Secondary Outcome Measures :
  1. Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) [ Time Frame: Day 0 ]
    CHOP-INTEND uses a scale of 0-64 points. Higher scores indicate better motor function. It is used to reliably measure natural motor function decline in infants with SMA Type 1.

  2. Hammersmith Functional Motor Scale Expanded (HMFSE) [ Time Frame: Day 0 ]
    It is an extended version of the 20-item HFMS. Based on another scale known as Gross Motor Function Measure, 13 items were added and expanded HFMS to include these elements. Therefore, HFMSE is a scale used to evaluate motor functions in types 2 and 3 SMA. It contains 33 items with a total score of 66 on a scale of 0, 1, 2.



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients with SMA type 1, type 2 or type 3 between the ages of 0-18 and their caregivers.
Criteria

Inclusion Criteria:

  1. Patients with spinal muscular atrophy at between the ages of 0-18
  2. Agree to participate in the study

Exclusion Criteria:

1-Refuse to participate in the study


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04228718


Contacts
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Contact: Ayca Evkaya, PT (MSc) 00902166570606 ayca.evkaya@gmail.com
Contact: Naime Evrim Karadag Saygi, Prof 00902166570606 evrimkaradag4@hotmail.com

Locations
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Turkey
Marmara University School of Medicine Department of Physical Medicine and Rehabilitation Recruiting
Istanbul, Turkey, 34890
Contact: Naime Evrim Karadag Saygi, Prof    00902166570606 ext 1628    evrimkaradag4@hotmail.com   
Contact: Ayca Evkaya, PT (MSc)    00902166570606 ext 1628    ayca.evkaya@gmail.com   
Principal Investigator: Ayca Evkaya, PT (MSc)         
Sub-Investigator: Sena Imamoglu, PT         
Sponsors and Collaborators
Marmara University
Investigators
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Study Director: Naime Evrim Karadag Saygi, Prof Department of PM&R, Marmara University School of Medicine
Principal Investigator: Ayca Evkaya, PT (MSc) Department of PM&R, Marmara University School of Medicine

Publications:
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Responsible Party: Marmara University
ClinicalTrials.gov Identifier: NCT04228718    
Other Study ID Numbers: 09.2019.728
First Posted: January 14, 2020    Key Record Dates
Last Update Posted: January 31, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Marmara University:
Spinal Muscular Atrophy
Caregiver Burden
Disabled persons
Caregivers
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases