- ICH GCP
- Rejestr badań klinicznych w USA
- Badanie kliniczne NCT07934186
LM11A-31 Safety and Tolerability in Aging People Living With HIV and Neurocognitive Impairment (LM11A31HAND)
Study to Establish Safety, Tolerability and Feasibility of LM11A-31 as a Neuroprotective Agent in Aging People Living With HIV and Neurocognitive Impairment on Antiretroviral Therapy
HIV rapidly infects the nervous system resulting in chronic infection that persists even when blood HIV is undetectable. The resulting inflammation causes progressive neuronal damage leading to cognitive decline in up to 50% of people living with HIV. The damage to the nervous system is due in part to a loss of natural processes that protect the brain. There are currently no effective treatments. A novel new drug, LM11A-31, has been shown to restore the protective processes thereby shifting the balance from degenerative to protective activity. Beneficial effects have been seen in patients with Alzheimer's disease and research indicates that it should have similar beneficial effects in people living with HIV. The proposed study will establish the safety and tolerability of LM11A-31 treatment in a small cohort of stable virally-suppressed participants with HIV and mild neurocognitive impairment. The focus will be on individuals 50-75 years of age who are most vulnerable for cognitive decline. To provide sufficient time to stabilize brain activity, participants will take tablets twice daily over a period of 48 weeks. The main focus of the trial will be on safety but will also be supplemented with exploratory characterization of biomarkers to confirm a reduction in degeneration as well as to provide early identification of individuals at risk for neurodegeneration. These studies are expected to show that LM11A-31 is safe to use in people living with HIV and to provide preliminary data indicating protection from neuronal damage and cognitive decline.
Przegląd badań
Status
Interwencja / Leczenie
Szczegółowy opis
As people with HIV (PWH) age, the effects of chronic viral infection and associated inflammation put them at higher risk for central nervous system (CNS) complications, including neurocognitive impairment (NCI). Up to half of PWH have some form of NCI, leading to impaired daily functioning, reduced medical access, and lower quality of life. In addition to the effects of HIV, evidence indicates that PWH are at risk for other age-related degenerative diseases such as Alzheimer's disease (AD)-related pathology. In the normal brain, proteins called neurotrophins protect the brain and support the development and stabilization of memories. An important modulator of the neurotrophins, nerve growth factor (NGF) and Brain Derived Neurotrophic Factor (BDNF), is the p75 neurotrophin receptor (p75NTR). This receptor can enhance pro-growth, pro-survival processes or cause degeneration and death of unfit neurons through an interaction with neurotrophin precursors, proneurotrophins. During development, the balance of mature versus proneurotrophin signaling is used to eliminate poorly connected neurons (low neurotrophin/high p75NTR signaling). In the healthy brain, expression of p75NTR is suppressed and pro-growth, pro-survival processes dominate through the actions of NGF and BDNF. However, when neurons are stressed by disease, damage and inflammation, the precursors to NGF (proNGF) and BDNF (proBDNF) increase indicating reduced availability of the beneficial mature neurotrophins. Under these conditions, p75NTR increases, engages with the pro-neurotrophins and supports degenerative processes.
Inflammation is often one of the first indicators of disease and is responsible for a variety of early neuronal changes that impair function and set the stage for disease. When HIV virions or gp120, interact with macrophages or microglia in the brain they induce the secretion of factors into the medium that alter neural function beginning with a chronic destabilization of intracellular calcium, cytoskeletal modifications and increases in the p75NTR. Studies indicate that the small molecule, p75NTR ligand, LM11A-31, interacts with the p75NTR to shift the balance of neurotrophin activity and inflammation from degeneration to neuroprotective and anti-inflammatory activity that can protect against HIV-associated neurodegeneration. By intervening and stabilizing neural function as pathological processes begin to develop, LM11A-31 has the potential to provide an effective disease-modifying intervention for PWH. Since the actions are expected to suppress disease activity, the intent is to modify the course of disease before the development of more permanent damage. Participants who show early signs of cognitive decline are therefore the best candidates for treatment. To assess the therapeutic potential of LM11A-31, the proposed trial will evaluate safety and tolerability in participants with mild cognitive decline and collect preliminary neurocognitive and biomarker data to evaluate the neuroprotective and anti-inflammatory effects in PWH.
Typ studiów
Zapisy (Szacowany)
Faza
- Faza 2
- Faza 1
Kontakty i lokalizacje
Lokalizacje studiów
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North Carolina
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Chapel Hill, North Carolina, Stany Zjednoczone, 27599
- University of North Carolina School of Medicine, Clinical and Translational Research CenterUnit
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Kontakt:
- Marleny, Clinical Research Coordinator, BS
- E-mail: marleny_nolasco@med.unc.edu
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Kontakt:
- Monica Diaz, Clinical Principle Investigator, MD, MS
- E-mail: monica.diaz@neurology.unc.edu
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Główny śledczy:
- Rick Meeker, PhD
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Kryteria uczestnictwa
Kryteria kwalifikacji
Wiek uprawniający do nauki
Akceptuje zdrowych ochotników
Płeć kwalifikująca się do nauki
Opis
Inclusion Criteria:
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Women and men of any race or ethnicity, aged 50-75 years, inclusive.
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Have capacity to provide informed consent, as demonstrated with a capacity screening tool.
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Sign informed consent form (ICF).
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Evidence of HIV infection. If unavailable in the participant's medical record, HIV infection will be confirmed using a point-of-care antibody test (MedMira Rapid Test, Canada). If the result differs from the participant's self-report, a clinical HIV antigen-antibody assay will be performed.
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Stable antiretroviral therapy (ART) regimen for at least 1 year with plasma HIV RNA ≤ 40 copies/mL at screening and for at least 6 months.
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CD4+ T-cell count > 350/μL.
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Mild to moderate neurocognitive impairment (NCI), defined as a Brain Baseline Assessment of Cognition and Everyday Functioning (BRACE) and global T score from a full 7 domain neurocognitive test battery that is at least 1 standard deviation below age- and sex-standardized norms.
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For women of reproductive potential, negative serum or urine pregnancy test within 48 hours prior to treatment initiation. Women of childbearing potential as well as male study participants with partners of childbearing potential must use a suitable barrier method (e.g., male condom) in conjunction with a highly effective form of contraception, while receiving the protocol-specified treatment and for 90 days after stopping.
- Women of childbearing potential (WOCBP) are defined as women who have experienced menarche and are neither postmenopausal nor permanently sterile. Permanent sterility includes prior hysterectomy, bilateral salpingectomy, or bilateral oophorectomy. Postmenopausal status is defined as at least 12 consecutive months without menstruation, with no alternative medical explanation for the amenorrhea.
- No sperm donations should be made from a participant while on study drug and for 90 days after stopping study drug.
Exclusion Criteria:
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Neurological conditions other than HIV disease that may affect study outcomes. Exclusionary neurological conditions include: head injury with loss of consciousness for more than 30 minutes or resulting in neurologic complications; poorly controlled or untreated seizure disorder; Alzheimer's Disease (AD) and related dementias, demyelinating diseases, a history of stroke, an intracranial mass lesion and other non-HIV neurological disorders that in the opinion of the study clinician would lead to impaired cognition.
o Note: The comprehensive medical evaluations, which includes a careful history and standardized neurologic examination, will collect symptoms and signs of any prior clinically significant cerebrovascular events, such as unilateral upper motor neuron pattern weakness, visual field defect, or hyperreflexia.
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Current or past severe central nervous system (CNS) infection (e.g., cryptococcal meningitis, toxoplasmic encephalitis).
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Clinically significant psychiatric disorder that could make study participation problematic or unsafe. Exclusionary psychiatric disorders include:
- Untreated major depressive disorder, schizophrenia or bipolar disorders and lifetime history of schizophrenia or other psychotic disorders
- Severe depression or suicidal intent on the Patient Health Questionaire-9 (PHQ-9) or Columbia-Suicide Severity Rating Scale (C-SSRS).
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Current substance use disorder (dependence of abuse) as defined by Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V) criteria. Exclusionary substance use includes: DSM-V-diagnosed alcohol use disorder (moderate or severe) within the past 12 months and, for other substances (except nicotine), moderate or severe substance use disorder within the past 5 years, or a mild substance use disorder within the past 6 months.
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Any illicit drug use as demonstrated on drug screening, except cannabis
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Pregnancy or breast-feeding
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Current serious illness requiring systemic treatment or hospitalization
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Any significant medical conditions that would put the individual at risk to participate, based on medical history, clinical examination, or any other protocol screening assessments. Exclusionary medical conditions include: significant renal insufficiency, unstable hypertension, unstable angina, poorly controlled diabetes mellitus, unstable cardiac arrhythmias, severe coronary artery disease, severe peripheral vascular disease, severe hepato-gastrointestinal disease, and oxygen-requiring chronic pulmonary disease.
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Untreated coinfection with hepatitis C virus (HCV), hepatitis B virus (HBV), or Treponema pallidum
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Any of the following laboratory findings at Screening:
- Alanine aminotransferase >2 x upper limit of normal (ULN), aspartate aminotransferase >2 x ULN, and/or total bilirubin >1.5 x ULN
- Note: Isolated bilirubin >1.5 x ULN is acceptable if bilirubin is fractionated and direct bilirubin is <35%.
- Creatinine clearance ≤ 45 min/mL (measured or calculated based on institutional standards)
- Eosinophil count >500 cells/µL
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Pre-existing gastrointestinal conditions:
- Active or poorly controlled inflammatory bowel disease, including Crohn's disease or ulcerative colitis, or active microscopic colitis.
- Ongoing infectious diarrhea, including unresolved Clostridioides difficile infection or another suspected or confirmed gastrointestinal infection.
- Persistent or recurrent diarrhea at screening that limits usual daily activities, requires ongoing treatment but remains inadequately controlled, or is accompanied by dehydration, clinically significant electrolyte abnormalities, or unintentional weight loss.
- Clinically significant malabsorption, short-bowel syndrome, or a high-output ostomy associated with difficulty maintaining hydration, electrolyte balance, or adequate nutrition.
- Active or history of gastrointestinal bleeding, unresolved bowel obstruction, or unresolved complications of gastrointestinal surgery.
- Any other gastrointestinal condition for which the clinical principle investigator (PI) and safety officer document that additional diarrhea would pose an unacceptable risk.
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Screening will include documentation of usual stool frequency and consistency, current gastrointestinal symptoms, relevant diagnoses and surgery, medications, hydration status, and available electrolyte and renal function results. Additional evaluation will be obtained when clinically indicated. Participants with an acute, potentially reversible condition will not be enrolled while the condition remains active. Rescreening may be considered after documented resolution and confirmation that all eligibility criteria are met. A history of a gastrointestinal diagnosis or surgery alone will not automatically exclude a participant if the condition is stable and none of the above criteria apply. Uncertain cases will require documented review by the clinical PI and safety officer before enrollment. These criteria will supplement the existing general medical exclusions
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Participants who are receiving any of the following medications should be excluded due to potential interactions with the study measures (i.e., chronic daily drug intake of ≥ 14 days or expected for ≥14 days):
- Centrally active drugs that in the opinion of the physician may affect cognitive performance (e.g., clonidine, l-methyl dihydroxyphenylalanine (DOPA), guanidine, guanfacine, etc.)
- Opioid containing analgesics
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For optional lumbar puncture (LP) procedure only. These criteria will not exclude participants from the study but will exclude them from undergoing option LP:
- Prior adverse effect (AE) due to an LP (i.e., prior post-LP headache that did not resolve within 3 days or required blood patch)
- Safety contraindications to LP (e.g., bleeding diathesis, anticoagulant use)
- Requirements for LP: platelet count ≥50,000/µL, International Normalized Ratio (INR) <1.5, and Prothrombin Time (PT) within the performing laboratory's normal reference range. An LP will not be performed if any of these requirements is not met.
- For clinically stable participants, these laboratory assessments will be obtained within 14 calendar days before each LP. If an intervening illness, bleeding concern, medication change, or concern about changing laboratory values arises, the participant will undergo clinical reassessment and repeat laboratory testing within 24 hours before the procedure. The LP will be deferred until the results meet eligibility criteria and the procedural clinician determines that it is appropriate to proceed.
- Before each LP, the procedural clinician will document review of the laboratory results, bleeding history, and medications that may increase bleeding risk. Medication-related contraindications and other LP safety exclusions will continue to apply. These requirements will be finalized with the study clinicians at both sites and incorporated consistently throughout the protocol. Participants who are ineligible for the optional LP may remain in the main study if they otherwise meet study eligibility criteria.
- The investigators will exclude participants with suspected intracranial space-occupying lesions from the optional LP procedure. The investigators will review relevant history, neurological findings with a neurological examination performed by a licensed clinician and available neuroimaging. Optional research magnetic resonance imaging (MRI) participation will not substitute for clinically indicated pre-LP imaging.
- A neurological examination, including fundoscopy will be performed by a qualified licensed clinician before every optional LP to assess for signs of raised intracranial pressure or other neurological contraindications. A normal funduscopic examination alone will not be considered sufficient to rule out raised intracranial pressure.
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For optional MRI only: Safety contraindications identified on MRI safety screening (e.g., implanted metal, stents, or other devices), based on institutional guidelines
Plan studiów
Jak projektuje się badanie?
Szczegóły projektu
- Główny cel: Leczenie
- Przydział: Randomizowane
- Model interwencyjny: Zadanie krzyżowe
- Maskowanie: Poczwórny
Broń i interwencje
Grupa uczestników / Arm |
Interwencja / Leczenie |
|---|---|
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Aktywny komparator: LM11A-31, followed by Placebo
LM11A-31 administered orally in the form of tablets for 24 weeks. Then placebo oral tablets identical to the active compound taken for 24 weeks. |
Tablets matched to active drug Tablets matching the active compound |
|
Komparator placebo: Placebo, followed by LM11A-31
Placebo oral tablets identical to the active compound taken for 24 weeks. Then LM11A-31 administered orally in the form of tablets for 24 weeks. |
Tablets matched to active drug Tablets matching the active compound |
Co mierzy badanie?
Podstawowe miary wyniku
Miara wyniku |
Opis środka |
Ramy czasowe |
|---|---|---|
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Adverse effects (AE)
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
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Any clinical signs/symptoms, laboratory toxicities and clinical events that are possibly or definitely related to study treatment. The incidence and severity of adverse effects will be summarized from Baseline to 24 weeks and 24 weeks to 48 weeks. |
From enrollment until the end of treatments at 48 weeks
|
|
Cluster of Differentiation (CD4)+ T cells
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
Participants that show a CD4+ T cell decline >33% from Baseline to Week 24 and week 24 to week 48 |
From enrollment until the end of treatments at 48 weeks
|
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HIV-1 ribonucleic acid (RNA)
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
Participants during each 24 week period with confirmed HIV-1 RNA level >40 copies/mL in the absence of an interruption in antiretroviral therapy |
From enrollment until the end of treatments at 48 weeks
|
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Neurocognitive performance
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
Change in neurocognitive performance from Baseline to Week 24 and week 24 to week 48 based on the neurocognitive test battery for HIV-associated neurocognitive impairment. The neurocognitive test battery contains 7 cognitive domains scored on a sliding scale. An aggregate z-score for all 7 domains will be calculated. Negative z-scores reflect poorer cognitive performance. |
From enrollment until the end of treatments at 48 weeks
|
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Number of Participants with Depressive symptoms or suicidal ideation
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
Depressive symptoms or new suicidal ideation during baseline to 24 weeks and 24 to 48 weeks based on psychiatric assessments, self-report, diary review or during interviews with the study coordinator. |
From enrollment until the end of treatments at 48 weeks
|
Miary wyników drugorzędnych
Miara wyniku |
Opis środka |
Ramy czasowe |
|---|---|---|
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Change in Expression of the Biomarker Neurofilament light chain (NFL)
Ramy czasowe: From enrollment until the end of treatments at weeks 24 and 48
|
Neurofilament light chain (NFL) is a marker of neuronal damage. Increased expression, measured from baseline to 24 weeks and 24 to 48 weeks, indicates degeneration of neurons in the brain. |
From enrollment until the end of treatments at weeks 24 and 48
|
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Change in expression of the biomarker Neurogranin
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
Neurogranin is a protein released from damaged dendritic spines in the brain. Increased expression, measured from baseline to 24 weeks and 24 to 48 weeks, indicates degeneration of neuronal connections in the brain. |
From enrollment until the end of treatments at 48 weeks
|
|
Change in expression of the biomarker Synaptosomal Protein-25 (SNAP-25)
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
Synaptosomal Protein-25 (SNAP-25) is released from damaged brain synapses. Increased expression, measured from baseline to 24 weeks and 24 to 48 weeks, indicates loss of neuronal connections in the brain. |
From enrollment until the end of treatments at 48 weeks
|
|
Change in Expression of the Biomarker phosphoThreonine 181 Tau (pThr181 Tau)
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
PhosphoThreonine 181 Tau (pThr181 Tau) is a protein in neurons that is mistakenly phosphorylated when neurons are stressed. Increased expression, measured from baseline to 24 weeks and 24 to 48 weeks, indicates a loss of normal function of neurons in the brain. |
From enrollment until the end of treatments at 48 weeks
|
|
48-plex cytokine array
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
Cytokines are protein released to regulate immune functions. Measurement of changes in a broad range of cytokines from baseline to 24 weeks and 24 to 48 weeks provides information on the pattern of changes in the immune system that may predict disease processes. |
From enrollment until the end of treatments at 48 weeks
|
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Change in expression of the biomarker C-X-C Motif Chemokine Ligand 10 (CXCL10)
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
C-X-C Motif Chemokine Ligand 10 (CXCL10) is a protein released in response to HIV-associated inflammation. An increase from baseline to 24 weeks and 24 to 48 weeks signals an increase in inflammatory activity that could damage the brain. |
From enrollment until the end of treatments at 48 weeks
|
|
Change in expression of the biomarkerChitinase-3-like protein 1 (YKL-40)
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
Chitinase-3-like protein 1, also known as YKL-40, is a protein released from brain astrocytes in response to inflammation. An increase from baseline to 24 weeks and 24 to 48 weeks signals an increase in inflammatory activity that could damage the brain. |
From enrollment until the end of treatments at 48 weeks
|
|
Change in expression of the biomarker Amyloid beta 1-42 (Aβ1-42)
Ramy czasowe: From enrollment until the end of treatments at 48 weeks
|
Amyloid beta 1-42 (Aβ1-42) is a protein generated by abnormal protein processing under conditions of stress in the brain. Often associated with Alzheimer's disease it can also accumulate in people with HIV. An increase from baseline to 24 weeks and 24 to 48 weeks indicates degenerative processes associated with cognitive decline. |
From enrollment until the end of treatments at 48 weeks
|
Współpracownicy i badacze
Współpracownicy
Śledczy
- Główny śledczy: Monica Diaz, MD, MS, University of North Carolina, Chapel Hill
- Dyrektor Studium: Rick Meeker, PhD, University of North Carolina, Chapel Hill
Daty zapisu na studia
Główne daty studiów
Rozpoczęcie studiów (Szacowany)
Zakończenie podstawowe (Szacowany)
Ukończenie studiów (Szacowany)
Daty rejestracji na studia
Pierwszy przesłany
Pierwszy przesłany, który spełnia kryteria kontroli jakości
Pierwszy wysłany (Rzeczywisty)
Aktualizacje rekordów badań
Ostatnia wysłana aktualizacja (Rzeczywisty)
Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości
Ostatnia weryfikacja
Więcej informacji
Terminy związane z tym badaniem
Słowa kluczowe
Dodatkowe istotne warunki MeSH
Inne numery identyfikacyjne badania
- 24-3168
- 5R01AG082540-02 (Grant/umowa NIH USA)
Plan dla danych uczestnika indywidualnego (IPD)
Planujesz udostępniać dane poszczególnych uczestników (IPD)?
Opis planu IPD
Deidentified individual data that supports the results will be shared beginning 9 and continuing for 36 months following publication provided the investigator who proposes to use the data has approval from an Institutional Review Board (IRB), Independent Ethics Committee (IEC), or Research Ethics Board (REB), as applicable, and executes a data use/sharing agreement with UNC.
Ramy czasowe udostępniania IPD
beginning 9 and continuing for 36 months following publication
Kryteria dostępu do udostępniania IPD
Investigator has approved IRB, IEC, or REB and an executed data use/sharing agreement with UNC.
Typ informacji pomocniczych dotyczących udostępniania IPD
- PROTOKÓŁ BADANIA
Informacje o lekach i urządzeniach, dokumenty badawcze
Bada produkt leczniczy regulowany przez amerykańską FDA
Bada produkt urządzenia regulowany przez amerykańską FDA
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