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Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome

Primary Purpose

Mitochondrial Diseases, Pearson Syndrome

Status
Recruiting
Phase
Phase 1
Locations
Israel
Study Type
Interventional
Intervention
MNV-201
Sponsored by
Minovia Therapeutics Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mitochondrial Diseases focused on measuring Autologous, Mitochondrial, Pearson, Transplantation, Stem cell

Eligibility Criteria

1 Year - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Male or female participants aged from 1 to 18 years old. Diagnosis of Pearson Syndrome, mtDNA deletion verified by NGS. Body weight ≥ 10 kg. Participant has anemia or thrombocytopenia, or leukopenia and/or blood transfusion dependent (receives blood transfusions every 6 weeks or less). Participant is medically able to undergo the study interventions, as determined by the investigator. Participant's living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent. Exclusion Criteria: History of infection with HIV-1, HIV-2, or HTLV I/II. Current active infection with HBV (including HBcore and HBsAg positive), HCV, HTLV I/II, Treponema Pallidum or HIV I-II Participant has been diagnosed with Myelodysplastic Syndrome, by FISH and/or karyotype. Participant is unable to undergo leukapheresis. Total number of CD34+ cells collected is lower than 20x106 cells Participant has known hypersensitivity to murine proteins or iron-dextran. Participant has chronic severe infection. Participant has disease or condition that may risk the participant or interfere with the ability to interpret the study results. History of malignancy History of treatment with gene therapy, bone marrow or allogeneic cord blood transplantation. Currently participating in another clinical trial, or participation in another clinical trial within 1 year prior to study enrollment. In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.

Sites / Locations

  • Sheba Medical CenterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria

Arm Description

Outcomes

Primary Outcome Measures

Occurrence of treatment-related adverse events
Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion.

Secondary Outcome Measures

Stabilization or improvement in Quality of Life (QoL) questionnaire IPMDS (International Pediatric Mitochondrial Disease Scale) scores
The score is expressed as the percentage of items which were feasible to perform. The lower the score, the better is the child performance.
Reduction in frequency and lengths of hospitalization during the 12 months after MAT compared to the 12 months period before MAT
Changes in anemia during a follow up period of 12 months post treatment.
Changes since baseline in anemia during a follow up period of 12 months post treatment measured by Complete Blood Count.
Changes in thrombocytopenia during a follow up period of 12 months post treatment
Changes since baseline in thrombocytopenia during a follow up period of 12 months post treatment measured by Complete Blood Count.
Changes in leukopenia during a follow up period of 12 months post treatment
Changes since baseline in leukopenia during a follow up period of 12 months post treatment measured by Complete Blood Count.
Changes in frequency of blood transfusions during a follow up period of 12 months post treatment.
Changes in frequency of blood transfusion will be assessed based on the number of blood transfusions received by the participant since baseline compared to the number of blood transfusions the participant received during the 6 months period pre screening.

Full Information

First Posted
August 24, 2023
Last Updated
October 19, 2023
Sponsor
Minovia Therapeutics Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT06017869
Brief Title
Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome
Official Title
A Phase I, Open Label, Single Dose Clinical Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 (Autologous CD34+ Cells Enriched With Allogenic Placenta Derived Mitochondria) in Pediatric Patients With Pearson Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 31, 2023 (Actual)
Primary Completion Date
December 2026 (Anticipated)
Study Completion Date
December 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Minovia Therapeutics Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Primary Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders caused by mutations in genes encoded by nuclear Deoxyribonucleic Acid (DNA) or by mutations and/or deletions in the mitochondrial DNA (mtDNA). While some mitochondrial disorders only affect a single organ (e.g., the eye in Leber hereditary optic neuropathy [LHON]), many involve multiple organs. Mitochondrial disorders may present at any age and a frequent feature is the increasing number of organs involved in the course of the disease. Minovia Therapeutics Ltd. ("Minovia") is a biotech company developing novel therapeutics based on its mitochondrial augmentation technology (MAT). MNV-201 is a cell therapy produced by MAT that consists of the participant's autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) enriched with allogeneic placental-derived mitochondria, manufactured in Minovia's GMP facility.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mitochondrial Diseases, Pearson Syndrome
Keywords
Autologous, Mitochondrial, Pearson, Transplantation, Stem cell

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
MNV-201
Other Intervention Name(s)
CD34+ cells enriched with allogeneic placenta derived mitochondria
Intervention Description
Autologous CD34+ cells are isolated from the participant's peripheral blood after mobilization by leukapheresis. Allogeneic mitochondria are isolated under aseptic conditions from healthy donor placenta, cryopreserved and qualified before use.
Primary Outcome Measure Information:
Title
Occurrence of treatment-related adverse events
Description
Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion.
Time Frame
12 months post treatment.
Secondary Outcome Measure Information:
Title
Stabilization or improvement in Quality of Life (QoL) questionnaire IPMDS (International Pediatric Mitochondrial Disease Scale) scores
Description
The score is expressed as the percentage of items which were feasible to perform. The lower the score, the better is the child performance.
Time Frame
12 months post treatment
Title
Reduction in frequency and lengths of hospitalization during the 12 months after MAT compared to the 12 months period before MAT
Time Frame
12 months post treatment
Title
Changes in anemia during a follow up period of 12 months post treatment.
Description
Changes since baseline in anemia during a follow up period of 12 months post treatment measured by Complete Blood Count.
Time Frame
12 months post treatment.
Title
Changes in thrombocytopenia during a follow up period of 12 months post treatment
Description
Changes since baseline in thrombocytopenia during a follow up period of 12 months post treatment measured by Complete Blood Count.
Time Frame
12 months post treatment
Title
Changes in leukopenia during a follow up period of 12 months post treatment
Description
Changes since baseline in leukopenia during a follow up period of 12 months post treatment measured by Complete Blood Count.
Time Frame
12 months post treatment
Title
Changes in frequency of blood transfusions during a follow up period of 12 months post treatment.
Description
Changes in frequency of blood transfusion will be assessed based on the number of blood transfusions received by the participant since baseline compared to the number of blood transfusions the participant received during the 6 months period pre screening.
Time Frame
12 months post treatment.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female participants aged from 1 to 18 years old. Diagnosis of Pearson Syndrome, mtDNA deletion verified by NGS. Body weight ≥ 10 kg. Participant has anemia or thrombocytopenia, or leukopenia and/or blood transfusion dependent (receives blood transfusions every 6 weeks or less). Participant is medically able to undergo the study interventions, as determined by the investigator. Participant's living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent. Exclusion Criteria: History of infection with HIV-1, HIV-2, or HTLV I/II. Current active infection with HBV (including HBcore and HBsAg positive), HCV, HTLV I/II, Treponema Pallidum or HIV I-II Participant has been diagnosed with Myelodysplastic Syndrome, by FISH and/or karyotype. Participant is unable to undergo leukapheresis. Total number of CD34+ cells collected is lower than 20x106 cells Participant has known hypersensitivity to murine proteins or iron-dextran. Participant has chronic severe infection. Participant has disease or condition that may risk the participant or interfere with the ability to interpret the study results. History of malignancy History of treatment with gene therapy, bone marrow or allogeneic cord blood transplantation. Currently participating in another clinical trial, or participation in another clinical trial within 1 year prior to study enrollment. In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Natalie Yivgi Ohana, PhD
Phone
+ 972 54 5833727
Email
natalie@minoviatx.com
First Name & Middle Initial & Last Name or Official Title & Degree
Lea Bensoussan, MSc
Phone
+972 58 6101291
Email
lea@minoviatx.com
Facility Information:
Facility Name
Sheba Medical Center
City
Ramat Gan
ZIP/Postal Code
5266202
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elad Jacoby, MD
Phone
+972 526668355
Email
elad.jacoby@sheba.health.gov.il
First Name & Middle Initial & Last Name & Degree
Moran Levin
Phone
+972523923147
Email
moran.levin@sheba.health.gov.il

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome

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