Ignite Creation Date:
2024-10-26 @ 3:42 PM
Last Modification Date:
2024-10-26 @ 3:42 PM
Study NCT ID:
NCT06629324
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-10-03
Brief Title:
Evaluating Bone Marrow Cell Transplant for Treating Cerebral Palsy from Brain Hypoxia a Phase II Clinical Trial
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Evaluation of the Efficacy of Autologous Bone Marrow-Derived Mononuclear Cell Transplantation in the Treatment of Cerebral Palsy Due to Brain Hypoxia a Phase II Randomized Clinical Trial
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-10
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:
This clinical trial aims to evaluate the effectiveness of autologous bone marrow mononuclear cell transfusion in treating cerebral palsy caused by cerebral hypoxia
The key questions the study seeks to answer are
What is the safety profile in terms of adverse events AE and serious adverse events SAE observed over the 9 months following the first transplantation
How does autologous bone marrow mononuclear cell BM MNC transplantation impact the gross motor function GMFM-88 scores and Gross Motor Function Classification System GMFCS scores in children with cerebral palsy
How does autologous BM MNC transplantation influence muscle tone Modified Ashworth Scale score and hand motor function MACSMini-MACS scale in children with cerebral palsy 9 months post the initial transplantation
Fifty-eight selected patients aged 1 to 10 years and diagnosed with spastic cerebral palsy due to brain hypoxia will be randomly divided into two groups
Group A will receive two BM MNC infusions with the first at baseline and the second at 6 months 21 days T6 via the spinal route
Group B will serve as the control group for the first 9 months During this period patients will not receive cell transplantation but will undergo a similar rehabilitation and medication regimen as Group A After 9 months Group B will receive two BM MNC infusions the first at 9 months 21 days T9 and the second at 15 months 21 days T15 via the spinal route with a follow-up at 18 months 21 days T18 compared to baseline
Both groups will undergo rehabilitation for 10 days per month three times either at rehabilitation centers or performed by a rehabilitation technician at home After this period continued training will be conducted by family members The combined medication regimen will include muscle relaxants if muscle spasticity is present vitamins and neuroprotective drugs Piracetam
The key questions the study seeks to answer are
What is the safety profile in terms of adverse events AE and serious adverse events SAE observed over the 9 months following the first transplantation
How does autologous bone marrow mononuclear cell BM MNC transplantation impact the gross motor function GMFM-88 scores and Gross Motor Function Classification System GMFCS scores in children with cerebral palsy
How does autologous BM MNC transplantation influence muscle tone Modified Ashworth Scale score and hand motor function MACSMini-MACS scale in children with cerebral palsy 9 months post the initial transplantation
Fifty-eight selected patients aged 1 to 10 years and diagnosed with spastic cerebral palsy due to brain hypoxia will be randomly divided into two groups
Group A will receive two BM MNC infusions with the first at baseline and the second at 6 months 21 days T6 via the spinal route
Group B will serve as the control group for the first 9 months During this period patients will not receive cell transplantation but will undergo a similar rehabilitation and medication regimen as Group A After 9 months Group B will receive two BM MNC infusions the first at 9 months 21 days T9 and the second at 15 months 21 days T15 via the spinal route with a follow-up at 18 months 21 days T18 compared to baseline
Both groups will undergo rehabilitation for 10 days per month three times either at rehabilitation centers or performed by a rehabilitation technician at home After this period continued training will be conducted by family members The combined medication regimen will include muscle relaxants if muscle spasticity is present vitamins and neuroprotective drugs Piracetam
Detailed Description:
Cerebral Palsy CP is one of the most common causes of motor disability in children significantly affecting their quality of life QOL Children with CP often face various challenges including motor sensory and communication difficulties 1 Recent studies suggest that autologous bone marrow-derived mononuclear cells BM MNCs show promise in improving motor function and reducing muscle spasticity in CP children 2 3 4 The effects of BM MNC transplantation on the quality of life in CP children and their families remain understudied despite improvements in motor function being reported
Our Phase I study evaluated the safety and effects of autologous BM MNCs on the improvement of gross motor function and muscle tone in children with CP This Phase II randomized clinical trial aims to evaluate the efficacy of autologous BM MNCs in children with CP at Vinmec International Hospital Hanoi Vietnam from October 2024 to January 2027 5 The inclusion criteria are 1 aged 1 to 10 years and of either sex 2 having a Gross Motor Function Classification System GMFCS score ranging from level II to level V and 3 spastic cerebral palsy due to cerebral hypoxia Patients will be excluded if they have coagulation disorders suffer from severe health conditions such as exhaustion heart lung liver or kidney failure or active infections have spinal injuries that prevent the administration of intrathecal injections are diagnosed with cancer test positive for HIV or have active viral hepatitis or have hemoglobin levels below 110 gL
In total 58 patients were randomly assigned to two groups Randomization allocation was conducted using a random number table and the ratio of participants in each group was 11 Group A will receive two BM MNC administrations the first at baseline and the second at 6 months 21 days T6 via intrathecal injection Concomitantly the CT group will undergo a 10-day-per-month rehabilitation program for 3 months either at rehabilitation centers or with at-home therapy followed by exercises managed by family members Group B will serve as the control group for the first 9 months receiving rehabilitation and medications similar to Group A but without BM MNC therapy After 9 months Group B will receive BM MNCs at 9 months 21 days T9 and 15 months 21 days T15 with outcomes evaluated at 18 months 21 days T18 compared to baseline
Bone marrow was collected under general anesthesia from both anterior superior iliac crests taking 15-20 minutes with a maximum volume of 350 mL for older children Mononuclear cells were isolated using Ficoll density gradient centrifugation and prepared for infusion The infusion performed in the L4-L5 spinal space lasted about 30 minutes at a rate of 20 mL per hour Cerebrospinal fluid CSF was withdrawn before infusion with the amount based on the childs weight Patients received Rocephin for infection prevention and pain relief with alternating doses of Ibuprofen and Efferalgan for 2 days post-procedure Seduxen was given once on the first night after bone marrow collection The rehabilitation program included exercises for head and body control muscle tone management and facilitated movements based on developmental milestones Occupational therapy focused on improving hand function and daily activities while speech therapy addressed communication comprehension and chewingswallowing abilities
The safety of the therapy will be assessed by monitoring the frequency and severity of adverse events AEs and serious adverse events SAEs SAEs include events leading to death life-threatening conditions hospitalizations or prolonged hospital stays significant or permanent disabilities and congenital abnormalities or birth defects Efficacy will be evaluated based on changes in gross motor function 9 months after the first BM MNC transplant This will involve the GMFM-88 scale to assess gross motor function the GMFCS scale to classify motor function severity and the Mini-MACs for children aged 1-4 and MACs for children aged 4-18 scales to measure hand function Additionally muscle tone changes will be measured using the Modified Ashworth Scale 9 months post-transplant
Our Phase I study evaluated the safety and effects of autologous BM MNCs on the improvement of gross motor function and muscle tone in children with CP This Phase II randomized clinical trial aims to evaluate the efficacy of autologous BM MNCs in children with CP at Vinmec International Hospital Hanoi Vietnam from October 2024 to January 2027 5 The inclusion criteria are 1 aged 1 to 10 years and of either sex 2 having a Gross Motor Function Classification System GMFCS score ranging from level II to level V and 3 spastic cerebral palsy due to cerebral hypoxia Patients will be excluded if they have coagulation disorders suffer from severe health conditions such as exhaustion heart lung liver or kidney failure or active infections have spinal injuries that prevent the administration of intrathecal injections are diagnosed with cancer test positive for HIV or have active viral hepatitis or have hemoglobin levels below 110 gL
In total 58 patients were randomly assigned to two groups Randomization allocation was conducted using a random number table and the ratio of participants in each group was 11 Group A will receive two BM MNC administrations the first at baseline and the second at 6 months 21 days T6 via intrathecal injection Concomitantly the CT group will undergo a 10-day-per-month rehabilitation program for 3 months either at rehabilitation centers or with at-home therapy followed by exercises managed by family members Group B will serve as the control group for the first 9 months receiving rehabilitation and medications similar to Group A but without BM MNC therapy After 9 months Group B will receive BM MNCs at 9 months 21 days T9 and 15 months 21 days T15 with outcomes evaluated at 18 months 21 days T18 compared to baseline
Bone marrow was collected under general anesthesia from both anterior superior iliac crests taking 15-20 minutes with a maximum volume of 350 mL for older children Mononuclear cells were isolated using Ficoll density gradient centrifugation and prepared for infusion The infusion performed in the L4-L5 spinal space lasted about 30 minutes at a rate of 20 mL per hour Cerebrospinal fluid CSF was withdrawn before infusion with the amount based on the childs weight Patients received Rocephin for infection prevention and pain relief with alternating doses of Ibuprofen and Efferalgan for 2 days post-procedure Seduxen was given once on the first night after bone marrow collection The rehabilitation program included exercises for head and body control muscle tone management and facilitated movements based on developmental milestones Occupational therapy focused on improving hand function and daily activities while speech therapy addressed communication comprehension and chewingswallowing abilities
The safety of the therapy will be assessed by monitoring the frequency and severity of adverse events AEs and serious adverse events SAEs SAEs include events leading to death life-threatening conditions hospitalizations or prolonged hospital stays significant or permanent disabilities and congenital abnormalities or birth defects Efficacy will be evaluated based on changes in gross motor function 9 months after the first BM MNC transplant This will involve the GMFM-88 scale to assess gross motor function the GMFCS scale to classify motor function severity and the Mini-MACs for children aged 1-4 and MACs for children aged 4-18 scales to measure hand function Additionally muscle tone changes will be measured using the Modified Ashworth Scale 9 months post-transplant
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