A Proof of Concept Study to Evaluate Exosomes From Human Mesenchymal Stem Cells in Women With Premature Ovarian Insufficiency (POI)
Overview
- Phase
- Phase 1
- Intervention
- VL-PX10
- Conditions
- Premature Ovarian Insufficiency
- Sponsor
- Vitti Labs, LLC
- Enrollment
- 9
- Locations
- 1
- Primary Endpoint
- Incidence of Treatment-Emergent Adverse Events
- Status
- Recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
The VL-POI-01 study is designed to evaluate the safety and efficacy of human placental mesenchymal stem cell derived exosome treatment in patients with premature ovarian insufficiency (POI) and diminished ovarian reserve.
Detailed Description
Premature ovarian insufficiency (POI) is a devastating disease for young women who have not yet completed childbearing. The prevalence of this condition is on the rise due to the increasing number of cancer survivors and the delay in childbearing age. Current treatment options available are very limited. Stem cell therapy has been shown to be beneficial and effective in various disease processes and the safety has been assessed in multiple clinical trials. The regenerative potential of mesenchymal stem cells is increasingly attributed to the paracrine effects of exosomes. Exosomes consist of bioactive lipids, nucleic acids, and proteins which play a key role in intercellular communication. Exosome therapy is considered a safe and effective therapy since it offers a cell-free approach. VL-PX10 is a decellularized exosome product derived from human placental derived mesenchymal stem cells. This interventional pilot study will investigate the ability of intravenous injection of VL-PX10 to restore steroidogenesis, folliculogenesis, and support quality of life improvement, resumption of menstruation, and reversal of infertility in patients with POI and diminished ovarian reserve. This is an open label study. All patients entering this study will be treated with VL-PX10. Participant duration will be approximately 12 months.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Able to understand and communicate in English language
- •Female of age 18-43 years
- •Diagnosis of premature ovarian insufficiency based on ESHRE Guidelines (i) oligo/amenorrhea for at least 4 months, and (ii) an elevated FSH level \>25 IU/L on two occasions \>4 weeks apart, or diagnosis of low ovarian reserve defined as: Basal FSH value \>10 IU/L or failure of prior attempts of assisted reproductive techniques due to limited ovarian response (poor responders)
- •Normal karyotype 46, XX, and no known history FMR1 premutation (fragile X syndrome)
- •Baseline AMH levels ≤ 1.0 ng/mL
- •Presence of at least one ovary
- •Normal uterine anatomy (by any clinically and/or imaging acceptable methods)
- •Normal thyroid function as evident by normal serum Thyroid Stimulating Hormone (TSH) levels
- •For subjects who had contraception before, the duration of amenorrhea should be more than 3 months after discontinuation of the oral contraception pill (OCP) or more than 6 months after discontinuation of Depo Provera (or similar) therapies
- •Agree to report any pregnancy to the research staff immediately
Exclusion Criteria
- •Currently pregnant or breast-feeding
- •Has a history of, or evidence of current gynecologic malignancy, breast cancer or other estrogen responsive cancer or any other malignancy within the past five years
- •Subjects with FMR1 premutation (fragile X syndrome), a BMP15 mutation or family history of a first degree relative with POI
- •Presence of adnexal masses indicating the need for further evaluation
- •Major mental health disorder that precludes participation in the study
- •Active substance abuse or dependence
- •Current or recent (within the past 2 weeks) use of the following medications: Oral or systemic corticosteroids, Hormones (estrogen, progestin, oral contraceptives), Danazol, anticoagulants, herbal or botanical supplements with possible hormonal effects. Washout will be allowed (2 weeks from screening)
- •Subjects under hormonal treatments including hormone replacement therapy (HRT) for osteoporosis, cardiovascular disease, or recalcitrant vasomotor symptomatology within 3 months from screening
- •Subjects with a history of breast cancer or other estrogen responsive cancer within 5 years from screening
- •Subjects with existing malignant neoplasm, under active management for malignant neoplasm or under active surveillance for malignant neoplasm within 5 years from screening
Arms & Interventions
Treatment Arm
Intervention: VL-PX10
Outcomes
Primary Outcomes
Incidence of Treatment-Emergent Adverse Events
Time Frame: 2 years
The number of treatment-emergent adverse events.
Secondary Outcomes
- Estradiol Levels(2 years)
- Antral Follicle Counts(2 years)
- Follicle Stimulating Hormone Levels(2 years)
- Anti-Müllerian Hormone Levels(2 years)