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This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twenty intravenous administrations of deramiocel, each separated by three months, over a period of approximately 60 months. Following completion of the initial open-label phase (Month 60), subjects who have completed all Month 60 assessments will be eligible to continue into a long-term open label extension (LT-OLE) period and can continue to receive deramiocel once every 3 months until deramiocel is commercially available or the sponsor terminates the study, or the subject withdraws consent or study participation is terminated by the sponsor.
Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of deramiocel.
Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, 60, and all LT-OLE visits. Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, and at all LT-OLE trial visits, unless otherwise indicated.
All deramiocel infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, with continued dosing in the LT-OLE visits. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.
This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twenty intravenous administrations of deramiocel, each separated by three months, over approximately a period of 60 months. Additional analyses of efficacy and safety will be conducted in the subsequent Long Term Open-Label Extension (LT-OLE) phase of the study.
Subjects will undergo a targeted screening during a 30-day screening period to determine eligibility based on protocol inclusion and exclusion criteria.
Eligible subjects will undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of deramiocel. Administration of deramiocel (Day 1) should occur within a maximum of 30 days following confirmation of eligibility.
Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12 (± 14 days, each), 15, 18, 21, 24, 27, 30, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, 60, and all LT-OLE visits (± 21 days, each). Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57 trial visits, unless otherwise indicated, and at all LT-OLE visits.
All deramiocel infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57 trial visits. Continued dosing in the LT-OLE phase will begin at LT-OLE Visit 1, which may overlap with Month 60 of the initial OLE phase/LT-OLE Visit 1, in some cases. Prior to each deramiocel administration, medications will be administered to the subject as determined by the Investigator based on the pre-treatment guidelines as outlined in the protocol and/or institutional protocols to minimize the risk of potential severe allergic reactions such as anaphylaxis. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day if medically cleared by the site Investigator. If clinically indicated, an unscheduled in-person visit will be performed at the investigative site with targeted assessments based on presentation of signs and symptoms following any infusion.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Deramiocel | Other | Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deramiocel (CAP-1002) | Biological | Peripheral infusion of 150 million allogeneic cardiosphere-derived cells administered every three months |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) From Baseline Through Month 12 | Adverse event (AE) is defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. Serious AE: an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAE: AE with onset after start of treatment or with onset date before the treatment start date but worsening after the treatment start date. TEAEs included both serious and non-serious TEAEs. | Baseline up to Month 12 |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 12 | Severity of adverse events (AE) were assessed by the investigator as Grade 1 = Mild (Transient or mild discomfort; no limitation in activity; no medical intervention/therapy required), Grade 2 = Moderate (Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required), Grade 3 = Severe (Marked limitation in activity, some assistance usually required; medical intervention/therapy required and often requiring hospitalization or prolongation of hospitalization), Grade 4 = Life-threatening (Extreme limitation in activity, significant assistance required; significant medical intervention/therapy required; hospitalization, prolongation of hospitalization, or hospice care) and Grade 5 = Death. | Baseline up to Month 12 |
| Change From Baseline in Functional Capacity as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) Total Score. | PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) at Month 24, Month 36, Month 48 and Month 60 | Adverse event (AE) is defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. Serious AE: an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAE: AE with onset after start of treatment or with onset date before the treatment start date but worsening after the treatment start date. TEAEs included both serious and non-serious TEAEs. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Craig McDonald, MD | UC Davis | Principal Investigator |
| Mark Awadalla | Capricor Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, Davis | Sacramento | California | 95817 | United States | ||
| Children's Hospital Colorado |
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| ID | Title | Description |
|---|---|---|
| FG000 | Deramiocel | Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for total of 20 IV infusions. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Safety population included all participants who received investigational product.
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| ID | Title | Description |
|---|---|---|
| BG000 | Deramiocel | Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for a total of 20 IV infusions. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) From Baseline Through Month 12 | Adverse event (AE) is defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. Serious AE: an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAE: AE with onset after start of treatment or with onset date before the treatment start date but worsening after the treatment start date. TEAEs included both serious and non-serious TEAEs. | Safety population included all participants who received investigational product. | Posted | Count of Participants | Participants | Baseline up to Month 12 |
|
Up to Month 12
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Deramiocel | Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for a total of 20 IV infusions. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiac Dysfunction | Cardiac disorders | MedDRA version 23.1 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Mark Awadalla | Capricor, Inc. | 858-727-1755 | clinicaltrialsgov@capricor.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 12, 2024 | Jun 25, 2025 | Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 20, 2024 | Jan 27, 2025 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| D009136 | Muscular Dystrophies |
| ID | Term |
|---|---|
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
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Open Label Extension of the HOPE-2 Trial
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| Baseline, Month 12 |
| Month 24, Month 36, Month 48 and Month 60 |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 60 | Severity of adverse events (AE) were assessed by the investigator as Grade 1 = Mild (Transient or mild discomfort; no limitation in activity; no medical intervention/therapy required), Grade 2 = Moderate (Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required), Grade 3 = Severe (Marked limitation in activity, some assistance usually required; medical intervention/therapy required and often requiring hospitalization or prolongation of hospitalization), Grade 4 = Life-threatening (Extreme limitation in activity, significant assistance required; significant medical intervention/therapy required; hospitalization, prolongation of hospitalization, or hospice care) and Grade 5 = Death. | Baseline up to Month 60 |
| Change From Baseline in Upper Limb Function as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60 | PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. | Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60 |
| Change From Baseline in Distal-Level (Wrist and Hand) Upper Limb Function as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60 | PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. | Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60 |
| Change From Baseline in Mid-Level (Elbow) as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60 | PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. | Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60 |
| Change From Baseline in Cardiac Parameter: Left Ventricular Ejection Fraction (LVEF) at Month, 24, 36, 48, and 60 | LVEF is a measurement of how much blood the left ventricle pumps out with each contraction. Change in LVEF from baseline as measured by Cardiac Magnetic Resonance (cMRI) | Baseline, Month 24, Month 36, Month 48, and Month 60 |
| Change From Baseline in Cardiac Parameter: Left Ventricular End Systolic Volumes-Indexed (LV-ESVI) at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in LV-ESVI as assessed by Cardiac Magnetic Resonance (cMRI). | Baseline, Month 24, Month 36, Month 48, and Month 60 |
| Change From Baseline in Cardiac Parameter: Left Ventricular End Diastolic Volumes-Indexed (LV-EDVI) at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in LV-EDVI as assessed by Cardiac Magnetic Resonance (cMRI. | Baseline, Month 24, Month 36, Month 48, and Month 60 |
| Change From Baseline in Cardiac Parameter: Left Ventricle Mass (LV Mass) at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in LV mass was assessed by Cardiac Magnetic Resonance (cMRI). | Baseline, Month 24, Month 36, Month 48, and Month 60 |
| Change From Baseline in Cardiac Parameter: Unindexed Volumes at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in unindexed volumes as assessed by Cardiac Magnetic Resonance (cMRI) | Baseline, Month 24, Month 36, Month 48, and Month 60 |
| Change From Baseline in Cardiac Parameter: Left Ventricle End Diastolic Wall Thickening (LVEDWT) at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in LVEDWT was assessed by Cardiac Magnetic Resonance (cMRI). | Baseline, Month 24, Month 36, Month 48, and Month 60 |
| Change From Baseline in Cardiac Parameter: Left Ventricle End Systolic Wall Thickening (LVESWT) at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in LVESWT was assessed by Cardiac Magnetic Resonance (cMRI). | Baseline, Month 24, Month 36, Month 48, and Month 60 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Children's Hospital Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for a total of 20 IV infusions. |
|
|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 12 | Severity of adverse events (AE) were assessed by the investigator as Grade 1 = Mild (Transient or mild discomfort; no limitation in activity; no medical intervention/therapy required), Grade 2 = Moderate (Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required), Grade 3 = Severe (Marked limitation in activity, some assistance usually required; medical intervention/therapy required and often requiring hospitalization or prolongation of hospitalization), Grade 4 = Life-threatening (Extreme limitation in activity, significant assistance required; significant medical intervention/therapy required; hospitalization, prolongation of hospitalization, or hospice care) and Grade 5 = Death. | Safety population included all participants who received investigational product. | Posted | Count of Participants | Participants | Baseline up to Month 12 |
|
|
|
| Primary | Change From Baseline in Functional Capacity as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) Total Score. | PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. | Safety population included all participants who received investigational product. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Month 12 |
|
|
|
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) at Month 24, Month 36, Month 48 and Month 60 | Adverse event (AE) is defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. Serious AE: an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAE: AE with onset after start of treatment or with onset date before the treatment start date but worsening after the treatment start date. TEAEs included both serious and non-serious TEAEs. | Not Posted | May 2027 | Month 24, Month 36, Month 48 and Month 60 | Participants |
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 60 | Severity of adverse events (AE) were assessed by the investigator as Grade 1 = Mild (Transient or mild discomfort; no limitation in activity; no medical intervention/therapy required), Grade 2 = Moderate (Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required), Grade 3 = Severe (Marked limitation in activity, some assistance usually required; medical intervention/therapy required and often requiring hospitalization or prolongation of hospitalization), Grade 4 = Life-threatening (Extreme limitation in activity, significant assistance required; significant medical intervention/therapy required; hospitalization, prolongation of hospitalization, or hospice care) and Grade 5 = Death. | Not Posted | May 2027 | Baseline up to Month 60 | Participants |
| Secondary | Change From Baseline in Upper Limb Function as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60 | PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. | Not Posted | May 2027 | Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60 | Participants |
| Secondary | Change From Baseline in Distal-Level (Wrist and Hand) Upper Limb Function as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60 | PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. | Not Posted | May 2027 | Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60 | Participants |
| Secondary | Change From Baseline in Mid-Level (Elbow) as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60 | PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. | Not Posted | May 2027 | Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60 | Participants |
| Secondary | Change From Baseline in Cardiac Parameter: Left Ventricular Ejection Fraction (LVEF) at Month, 24, 36, 48, and 60 | LVEF is a measurement of how much blood the left ventricle pumps out with each contraction. Change in LVEF from baseline as measured by Cardiac Magnetic Resonance (cMRI) | Not Posted | May 2027 | Baseline, Month 24, Month 36, Month 48, and Month 60 | Participants |
| Secondary | Change From Baseline in Cardiac Parameter: Left Ventricular End Systolic Volumes-Indexed (LV-ESVI) at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in LV-ESVI as assessed by Cardiac Magnetic Resonance (cMRI). | Not Posted | May 2027 | Baseline, Month 24, Month 36, Month 48, and Month 60 | Participants |
| Secondary | Change From Baseline in Cardiac Parameter: Left Ventricular End Diastolic Volumes-Indexed (LV-EDVI) at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in LV-EDVI as assessed by Cardiac Magnetic Resonance (cMRI. | Not Posted | May 2027 | Baseline, Month 24, Month 36, Month 48, and Month 60 | Participants |
| Secondary | Change From Baseline in Cardiac Parameter: Left Ventricle Mass (LV Mass) at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in LV mass was assessed by Cardiac Magnetic Resonance (cMRI). | Not Posted | May 2027 | Baseline, Month 24, Month 36, Month 48, and Month 60 | Participants |
| Secondary | Change From Baseline in Cardiac Parameter: Unindexed Volumes at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in unindexed volumes as assessed by Cardiac Magnetic Resonance (cMRI) | Not Posted | May 2027 | Baseline, Month 24, Month 36, Month 48, and Month 60 | Participants |
| Secondary | Change From Baseline in Cardiac Parameter: Left Ventricle End Diastolic Wall Thickening (LVEDWT) at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in LVEDWT was assessed by Cardiac Magnetic Resonance (cMRI). | Not Posted | May 2027 | Baseline, Month 24, Month 36, Month 48, and Month 60 | Participants |
| Secondary | Change From Baseline in Cardiac Parameter: Left Ventricle End Systolic Wall Thickening (LVESWT) at Month 24, Month 36, Month 48, and Month 60 | Change from baseline in LVESWT was assessed by Cardiac Magnetic Resonance (cMRI). | Not Posted | May 2027 | Baseline, Month 24, Month 36, Month 48, and Month 60 | Participants |
| 0 |
| 13 |
| 0 |
| 13 |
| 11 |
| 13 |
| Supraventricular Extrasystoles | Cardiac disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Ventricular Extrasystoles | Cardiac disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Secondary Hypogonadism | Endocrine disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Pyrexia | General disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Eyelid Infection | Infections and infestations | MedDRA version 23.1 | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA version 23.1 | Non-systematic Assessment |
|
| Pharyngitis Streptococcal | Infections and infestations | MedDRA version 23.1 | Non-systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA version 23.1 | Non-systematic Assessment |
|
| Ligament Injury | Injury, poisoning and procedural complications | MedDRA version 23.1 | Non-systematic Assessment |
|
| Lower Limb Fracture | Injury, poisoning and procedural complications | MedDRA version 23.1 | Non-systematic Assessment |
|
| Lumbar Vertebral Fracture | Injury, poisoning and procedural complications | MedDRA version 23.1 | Non-systematic Assessment |
|
| Procedural Pain | Injury, poisoning and procedural complications | MedDRA version 23.1 | Non-systematic Assessment |
|
| Heart Rate Increased | Investigations | MedDRA version 23.1 | Non-systematic Assessment |
|
| Respiratory Rate Increased | Investigations | MedDRA version 23.1 | Non-systematic Assessment |
|
| Decreased Appetite | Metabolism and nutrition disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Deformity Thorax | Musculoskeletal and connective tissue disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Muscular Weakness | Musculoskeletal and connective tissue disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Musculoskeletal Pain | Musculoskeletal and connective tissue disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Dysgeusia | Nervous system disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Throat Irritation | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Mechanical Urticaria | Skin and subcutaneous tissue disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Skin Disorder | Skin and subcutaneous tissue disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Deep Vein Thrombosis | Vascular disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
| Flushing | Vascular disorders | MedDRA version 23.1 | Non-systematic Assessment |
|
Not provided
Not provided
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| Title | Measurements |
|---|
|
| Grade 4 |
|
| Grade 5 |
|