A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome
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|ClinicalTrials.gov Identifier: NCT03384420|
Recruitment Status : Enrolling by invitation
First Posted : December 27, 2017
Last Update Posted : March 26, 2020
|Condition or disease||Intervention/treatment||Phase|
|Mitochondrial Diseases Pearson Syndrome||Biological: CD34+ cells enriched with MNV-BLD||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||7 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I/II, Open Label, Single Dose Clinical Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD (Autologous cd34+ Cells Enriched With Blood Derived Mitochondria) in Pediatric Patients With Pearson Syndrome|
|Actual Study Start Date :||February 13, 2019|
|Estimated Primary Completion Date :||January 1, 2021|
|Estimated Study Completion Date :||January 1, 2021|
Experimental: Intervention 'CD34+ cells enriched with MNV-BLD'
Intervention 'CD34+ cells enriched with MNV-BLD'
Biological: CD34+ cells enriched with MNV-BLD
Transplantation of autologous stem cell enriched with MNV-BLD (blood-derived mitochondria)
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 following MNV-BM-BLD infusion. [ Time Frame: 1 year ]Toxicities will according to CTCAE, Version 5.0 will be assessed starting enrollment.
- Change in Quality of Life (QoL) questionnaire IPMDS (International Pediatric Mitochondrial Disease Scale) [ Time Frame: 1 year ]To explore Change in International Pediatric Mitochondrial Disease Scale (IPMDS) score during a follow up period of 12 months post treatment. IPMDS total score ranges from 0 to 243. The score is expressed as the percentage of items which were feasible to perform. The lower the score is, the higher the child's function
- Percent of mitochondrial engraftment, per participant, compared to baseline, by serial qPCR in peripheral blood performed monthly. [ Time Frame: 1 years ]We will monitor the engraftment of infused autologous cells enriched with normal mitochondria by measuring the level of the mitochondrial DNA by a qPCR assay designed to detect and separate between the participant's mitochondria and the normal donor mitochondria. Assays will be performed at baseline, monthly during the first year following the intervention, and then every 3 months.
- To measure change from baseline in cognitive status per patient by serial neuro-developmental clinical exams. [ Time Frame: 1 years ]Neuro-developmental assessment will be performed by a board-certified neurologist at baseline and every 6 months
- Number of patients with changes in brain MRI from baseline [ Time Frame: 1 years ]Brain MRI will be done at baseline to detect changes associated with mitochondrial disorders. Follow-up will at 1 year.
- To measure change from baseline in aerobic activity by 5-minute walk test or exercise test, according to baseline ability. [ Time Frame: 1 years ]Aerobic activity will be assessed at baseline and every 6 months
- To measure the change from baseline in weight as compared to age-specific growth charts - from baseline and monthly throughout the study [ Time Frame: 1 years ]Growth is delayed in many mitochondrial disorders; We will follow weight gain of participants in this study and compare to control growth charts
- Number of patients developing anti-mitochondrial antibodies compared to baseline during the study period. [ Time Frame: 1 year ]For potential immune reactions, anti-mitochondrial antibodies will be determined at baseline, and every 3 months till 1 year.
- To measure change from baseline in peripheral blood lactate level. [ Time Frame: 1 years ]Determination of change in basic metabolic parameters in peripheral blood as a result of mitochondrial augmentation, focusing on lactate level. Measurements will be performed at baseline and every 3 months.
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): NCT03384420
|Sheba Medical Center Hospital- Tel Hashomer|
|Ramat Gan, Israel|