Mitochondrial DiseasesPhase 1/2NCT03384420
A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome
A Phase 1/2 study testing CD34+ cells enriched with MNV-BLD in people with Mitochondrial Diseases and Pearson Syndrome, run by Minovia Therapeutics Ltd. at 1 site in Israel.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Mitochondrial Diseases, Pearson Syndrome
- What is being tested?
- CD34+ cells enriched with MNV-BLD
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteIsrael
- Study dates
- 13 Feb 2019to 9 Mar 2021
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Show all 9Show fewer
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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
- Being tested
- CD34+ cells enriched with MNV-BLD
- Keywords
- Autologous stem cell transplantation, Mitochondrial Diseases, Pearson Syndrome, Rare Diseases
- Medical subject headings
- Mitochondrial Diseases, VLCAD deficiency, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD34+ cells enriched with MNV-BLDBiological
Transplantation of autologous stem cell enriched with MNV-BLD (blood-derived mitochondria)
| Study group | Role | What they receive |
|---|---|---|
| Intervention CD34+ cells enriched with MNV-BLD | Experimental | Intervention CD34+ cells enriched with MNV-BLD Receives: Biological: CD34+ cells enriched with MNV-BLD |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 1 year
Secondary outcomes (8)
Weight
To compare the changes (kilograms) to Baseline
Measured: 1 year
Quantification of levels of normal mtDNA in blood and urine
To compare the changes to Baseline
Measured: 1 years
Metabolic crisis events occurrence compared to two years prior to the study.
To compare the changes during 3 years (2 years prior the study entry and 1 year follow up)
Measured: 3 Years
Change in renal function
Measurement of blood creatinine in a serum sample
Measured: 1 year
Change in Brain involvement
Lactate peak as assessed by MRS
Measured: 1 year
Height
To compare the changes (in meters) to Baseline
Measured: 1 year
Change in cardiac function
Assessment of left ventricular ejection fraction via echocardiography
Measured: 1 year
Monitoring for liver disease
Measurement of Aspartate Aminotransferase and Alanine aminotransferase level
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Dec 2017
- Study start13 Feb 2019
- Primary completion9 Mar 2021
- Study completion9 Mar 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Minovia Therapeutics Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03384420
- Sponsor's protocol ID
- MNV-BM-BLD-001-IL
- Status last verified
- Jun 2020
- First submitted
- 30 Nov 2017
- Last updated
- 31 Aug 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03384420
- Acronym / sponsor ID
- MNV-BM-BLD-001-IL
- Conditions
- Mitochondrial Diseases, Pearson Syndrome
- Keywords
- Autologous stem cell transplantation, Mitochondrial Diseases, Pearson Syndrome, Rare Diseases
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Minovia Therapeutics Ltd.Industry (pharma, biotech, devices)
- Study start
- 13 Feb 2019 (actual)
- Primary completion
- 9 Mar 2021 (actual)
- Study completion
- 9 Mar 2021 (actual)
- First posted
- 27 Dec 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intervention CD34+ cells enriched with MNV-BLD | Experimental | Biological: CD34+ cells enriched with MNV-BLDIntervention CD34+ cells enriched with MNV-BLD |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- 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:
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CD34+ cells enriched with MNV-BLDTransplantation of autologous stem cell enriched with MNV-BLD (blood-derived mitochondria) | Biological | Intervention CD34+ cells enriched with MNV-BLD |
Primary outcome
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.
Time frame: 1 year
Severity will graded according to CTCAE, Version 5.0
IPMDS (International Pediatric Mitochondrial Disease Scale)
Time frame: 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
8 secondary outcomes
Weight
Time frame: 1 year
Quantification of levels of normal mtDNA in blood and urine
Time frame: 1 years
Metabolic crisis events occurrence compared to two years prior to the study.
Time frame: 3 Years
Change in renal function
Time frame: 1 year
Change in Brain involvement
Time frame: 1 year
Height
Time frame: 1 year
Change in cardiac function
Time frame: 1 year
Monitoring for liver disease
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2021.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CD34+ cells enriched with MNV-BLD in people with Mitochondrial Diseases and Pearson Syndrome, run by Minovia Therapeutics Ltd. at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Aug 2021.