A First in Human Study to Evaluate the Safety of Infusion of MNV-BM-PLC (Autologous CD34+ Cells Enriched With Placenta Derived Allogeneic Mitochondria) in Patients With Primary Mitochondrial Diseases Associated With Mitochondrial DNA Mutation or Deletion
Key facts
- Who runs it
- Minovia Therapeutics Ltd.
- Where
- Israel1 site in 1 countryChoose a country to see its sites
- Trial code
- Status
- Withdrawn
- Phase
- Phase 1
- Started
- 1 Mar 2022
- Ended
- 1 Dec 2024
- Type of study
- Interventional
- Treatments tested
- Bone Marrow mobilization
- Apheresis
- MNV-BM-PLC infusion
- Record updated
- 22 June 2025
- Why it stopped
- No recruitment
Who can take part
- Age
- 4 years to 18 years
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
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Who can take part
- Molecular diagnosis of primary mitochondrial disease
- Age between 4 years and up to 18 years, with a minimum body weight of 20 (+/-1) kilogram on the day of screening visit
- Performance score: Karnofsky ≥40 (or equivalent in children younger than 16 years old
- Patients or Patient's parents or legal guardian (where applicable) has a good understanding of the study and nature of the procedure and is willing and able to provide written informed consent prior to participation in any study-related procedures
- Medical ability to undergo the study procedures safely, as determined by the investigator
Who cannot take part
- Patient has been treated within the last one year prior to IP treatment with a different cell therapy
- Patient has participated in another interventional clinical study and/or received other experimental medication outside of a clinical study within 1 month prior the day of Investigation product (IP) treatment visit
- Chronic severe infection or any other disease or condition that may risk the patient or interfere with the ability to interpret the study results
- Known history of malignancy
- Positive test for pathogenic agents
- Inability to undergo leukapheresis, as determined by the investigator
- A pregnant or lactating woman or a woman who plans to become pregnant during the study. In addition, any woman of childbearing potential (not sterile or postmenopausal), who is unwilling to adhere to the use highly effective contraception method for the duration of the study
- In the opinion of the Investigator, the patient is unsuitable for participating in the study due to safety concerns
This trial has no sites in United States.
It has sites in one country. Choose a country to see its sites.
About the trial
What the trial wants to find out
The study objectives are to evaluate the safety of a single intravenous (IV) infusion of autologous CD34+ cells enriched with placenta-derived allogeneic mitochondria in participant with primary mitochondrial disease associated with mitochondrial DNA mutations or deletions.
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6 participants aged from 4 to 18 years old on the day of screening visit with primary mitochondrial disease associated with mitochondrial DNA mutations or deletions will be enrolled.
The group in the trial
- Cohort 1 & Cohort 2Treatment being tested
3 patients will be administrated with Dose 1 (0.88 mitochondria unit (mU) citrate synthase (CS) activity per million cells). 3 patients will be administrated with Dose 2 (4.4mU mitochondria unit (mU) citrate synthase (CS) activity per million cells).
Treatment: Bone Marrow mobilization, Apheresis, and MNV-BM-PLC infusion
Data and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- A First in Human Phase I, Open Label Dose-escalation Study to Evaluate the Safety of Infusion of MNV-BM-PLC (Autologous CD34+ Cells Enriched With Placenta Derived Allogeneic Mitochondria) in Patients With Primary Mitochondrial Diseases Associated With Mitochondrial DNA Mutation or Deletion
- Record updated
- 22 June 2025