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

NCT03384420 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: MNV-BM-BLD-001-IL
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

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.

Conditions

Mitochondrial Diseases; Pearson Syndrome

Keywords

Autologous stem cell transplantation; Mitochondrial Diseases; Pearson Syndrome; Rare Diseases

Outcome Measures

Primary Outcomes (2)

• 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.

  Severity will graded according to CTCAE, Version 5.0

  1 year

• IPMDS (International Pediatric Mitochondrial Disease Scale)

  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

  1 year

Secondary Outcomes (8)

• Weight

  1 year

• Quantification of levels of normal mtDNA in blood and urine

  1 years

• Metabolic crisis events occurrence compared to two years prior to the study.

  3 Years

• Change in renal function

  1 year

• Change in Brain involvement

  1 year

Other Outcomes (7)

• Hospitalization events

  1 year

• Change in functional status

  1 year

• Change in hematological parameter

  1 year

Study Arms (1)

Intervention CD34+ cells enriched with MNV-BLD

EXPERIMENTAL

Intervention CD34+ cells enriched with MNV-BLD

Biological: CD34+ cells enriched with MNV-BLD

Interventions

CD34+ cells enriched with MNV-BLD BIOLOGICAL

Transplantation of autologous stem cell enriched with MNV-BLD (blood-derived mitochondria)

Intervention CD34+ cells enriched with MNV-BLD

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patient diagnosed with Pearson Syndrome, as verified by molecular identification of a defect in the mitochondrial DNA.
• Normal maternal mitochondria as verified by mtDNA sequencing.
• Males and females between 3 years or older and up to 18th birthday.
• Patient is transfusion independent.
• Patient has at least one of the following systematic involvements:
• High baseline lactate levels
• Episodes of metabolic crisis in the last year before pre-screening
• Renal failure (not dependent on dialysis) or evidence of proximal tubulopathy
• Growth retardation or failure to thrive

You may not qualify if:

• Absence of detectable mitochondria mutation or deletion.
• Patient or patient's mother have a positive test for microbiologic
• Patient is unable to undergo leukapheresis.
• Patient suffers from chronic severe infection, malignant disease or any other disease or condition that may risk the patient or interfere with the ability to interpret the study results.
• Patient has been treated previously with any cell or gene therapy.
• Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study.

Sponsors & Collaborators

• Minovia Therapeutics Ltd.: lead

Study Sites (1)

Sheba Medical Center Hospital- Tel Hashomer

Ramat Gan, Israel

Location

MeSH Terms

Conditions

Mitochondrial Diseases; VLCAD deficiency; Rare Diseases

Condition Hierarchy (Ancestors)

Metabolic Diseases; Nutritional and Metabolic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 30, 2017
• First Posted: December 27, 2017
• Study Start: February 13, 2019
• Primary Completion: March 9, 2021
• Study Completion: March 9, 2021
• Last Updated: August 31, 2021

Record last verified: 2020-06

Locations

IL (1)