Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06503367
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-06-21
Brief Title:
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Establishing Clinical Trial Readiness in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy READY CMD LAMA2
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time We will also learn about how this condition impacts other body systems
Participants will undergo
Neuromuscular assessments
Blood collections
Swallowing and breathing assessments
Questionnaires
Participants will undergo
Neuromuscular assessments
Blood collections
Swallowing and breathing assessments
Questionnaires
Detailed Description:
READY CMD LAMA2 is a Pre-Phase 1 single-arm multicenter prospective natural history clinical study to evaluate the natural history and potential early outcome measures in subjects with laminin α2-related dystrophy LAMA2-RD aged 0-5 years The study seeks to enroll 44 subjects across 14 sites NCH will enroll 10 subjects
Subjects will complete in-clinic visits at Baseline Month 6 Month 12 Month 18 and Month 24 Within two weeks of these visits a remote visit will be performed to repeat and compare remote outcomes with in-person outcomes A phone call will also be made by the coordinator to capture adverse events AEs at months 3 9 15 and 21 Remote visits via videoconference may be conducted at Months 3 9 15 21
The total duration for each subject is up to 24 months
All subjects will have functional assessments including the evaluation of motor milestones at each visit Evaluation of milestones lost or gained will be according to the Motor Milestone Checklist unless otherwise specified Assessment of all functional testing will be captured by video
Physical assessments to be performed will depend on the subjects age and may include the following
Assessment of motor milestones maintained lost or acquired
Neuromuscular Gross Motor Outcome GRO
Childrens Hospital of Philadelphia Infant Test of Neuromuscular Disorders CHOP-INTEND
Motor Function Measure MFM-20
Bayley-4
Respiratory assessments transcutaneous capnography and collection of data on use of non- invasive ventilation and other respiratory support
Swalloworomotor assessment To be performed by Speech therapist at bedside
Other assessments
Growth parameters head circumference height weight body mass index BMI
Mode of feeding
Peds Quality of Life PedsQL Neuromuscular Module
Clinical Global Impression of Change CGIC physician and parentcaregiver assessed
Blood samples will be collected from subjects and stored for biomarker and other research purposes
AEs will be collected and assessed for a potential relationship to participation in this study
The total duration of the study for each subject is up to approximately 24 months A subject will be considered to have competed the study if heshe has completed all assessments up to and including Month 24 assessments The end of the study is defined as the date of completion of the last scheduled assessment shown in the Schedule of Assessments and Procedures for the last subject in the study
Subjects who prematurely discontinue study participation will be encouraged to complete an End-of- Study Visit to include assessment of motor milestones and recording of AEs
Subjects will complete in-clinic visits at Baseline Month 6 Month 12 Month 18 and Month 24 Within two weeks of these visits a remote visit will be performed to repeat and compare remote outcomes with in-person outcomes A phone call will also be made by the coordinator to capture adverse events AEs at months 3 9 15 and 21 Remote visits via videoconference may be conducted at Months 3 9 15 21
The total duration for each subject is up to 24 months
All subjects will have functional assessments including the evaluation of motor milestones at each visit Evaluation of milestones lost or gained will be according to the Motor Milestone Checklist unless otherwise specified Assessment of all functional testing will be captured by video
Physical assessments to be performed will depend on the subjects age and may include the following
Assessment of motor milestones maintained lost or acquired
Neuromuscular Gross Motor Outcome GRO
Childrens Hospital of Philadelphia Infant Test of Neuromuscular Disorders CHOP-INTEND
Motor Function Measure MFM-20
Bayley-4
Respiratory assessments transcutaneous capnography and collection of data on use of non- invasive ventilation and other respiratory support
Swalloworomotor assessment To be performed by Speech therapist at bedside
Other assessments
Growth parameters head circumference height weight body mass index BMI
Mode of feeding
Peds Quality of Life PedsQL Neuromuscular Module
Clinical Global Impression of Change CGIC physician and parentcaregiver assessed
Blood samples will be collected from subjects and stored for biomarker and other research purposes
AEs will be collected and assessed for a potential relationship to participation in this study
The total duration of the study for each subject is up to approximately 24 months A subject will be considered to have competed the study if heshe has completed all assessments up to and including Month 24 assessments The end of the study is defined as the date of completion of the last scheduled assessment shown in the Schedule of Assessments and Procedures for the last subject in the study
Subjects who prematurely discontinue study participation will be encouraged to complete an End-of- Study Visit to include assessment of motor milestones and recording of AEs
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None