A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome

Phase 1

Completed

• Conditions: Mitochondrial Diseases; Pearson Syndrome

• Registration Number: NCT03384420
• Lead Sponsor: Minovia Therapeutics Ltd.

Brief Summary

Mitochondrial diseases are a genetically heterogeneous group of disorders caused by mutations or deletions in mitochondrial DNA (mtDNA) displaying a wide range of severity and phenotypes. These diseases may be inherited from the mother (mitochondrial inheritance) or non-inherited. The latter are ultra-rare pediatric diseases caused by a mutation or deletion of mtDNA, which develop into a systemic multi organ disease and eventually death. MNV-BM-BLD is a therapeutic process for enrichment of patient's peripheral hematopoietic stem cells with normal and healthy mitochondria derived from donor blood cells. The process, called mitochondria augmentation therapy, aims to reduce the symptoms of mitochondrial diseases.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-11-30
• Study First Submitted QC: 2017-12-24
• Study First Posted: 2017-12-27
• Study Start: 2019-02-13
• Status Verified: 2020-06
• Primary Completion: 2021-03-09
• Study Completion: 2021-03-09
• Last Update Submitted: 2021-08-30
• Last Update Posted: 2021-08-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 7

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Number of participants with Treatment-related adverse events as assessed by CTCAE v5.0 following MNV-BM-BLD during a follow up period of 12 months post treatment.	1 year	Severity will graded according to CTCAE, Version 5.0
IPMDS (International Pediatric Mitochondrial Disease Scale)	1 year	To compare the 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

Secondary Outcome Measures

Name	Time	Method
Change in Brain involvement	1 year	Lactate peak as assessed by MRS
Height	1 year	To compare the changes (in meters) to Baseline
Change in renal function	1 year	Measurement of blood creatinine in a serum sample
Monitoring for liver disease	1 year	Measurement of Aspartate Aminotransferase and Alanine aminotransferase level
Weight	1 year	To compare the changes (kilograms) to Baseline
Quantification of levels of normal mtDNA in blood and urine	1 years	To compare the changes to Baseline
Metabolic crisis events occurrence compared to two years prior to the study.	3 Years	To compare the changes during 3 years (2 years prior the study entry and 1 year follow up)
Change in cardiac function	1 year	Assessment of left ventricular ejection fraction via echocardiography

Trial Locations

Locations (1)

Sheba Medical Center Hospital- Tel Hashomer; 🇮🇱 Ramat Gan, Israel