
Search Clinical Trials
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Utilizing Electronic Modules to Educate on Sexual Health Practices During Pregnancy
Loyola University
Sexual Health Education During Pregnancy
Communication About Sexual Health in Prenatal Care
Sexual Function and Intimacy in Pregnancy
This study looks at whether an interactive online education module can improve knowledge,
comfort, and communication about sexual health during pregnancy. Many pregnant
individuals experience changes in sexual function and intimacy but may feel uncomfortable
discussing these topics or may have misc1 expand
This study looks at whether an interactive online education module can improve knowledge, comfort, and communication about sexual health during pregnancy. Many pregnant individuals experience changes in sexual function and intimacy but may feel uncomfortable discussing these topics or may have misconceptions about what is safe during pregnancy. Participants who are pregnant and receiving routine prenatal care will be randomly assigned to one of two groups. One group will complete an interactive, evidence-based educational module focused on sexual health and intimacy during pregnancy. The other group will review a standard educational article from the American College of Obstetricians and Gynecologists (ACOG) about sexual activity during pregnancy. Participants will complete surveys before and after reviewing the educational material, as well as a follow-up survey two weeks later. These surveys will measure knowledge about sexual health during pregnancy, comfort discussing sexual concerns with healthcare providers, and sexual function. The goal of this study is to determine whether an interactive educational approach can better support pregnant individuals' understanding of sexual health and encourage open communication with healthcare providers during pregnancy. Type: Interventional Start Date: Feb 2026 |
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A Study to Test Whether Nerandomilast Helps People With Systemic Sclerosis
Boehringer Ingelheim
Systemic Sclerosis
Nerandomilast is being developed to help people with systemic sclerosis by potentially
improving symptoms and slowing disease progression. This study is open to adults who are
at least 18 years old and have systemic sclerosis (SSc). People can join the study if
they have limited or diffuse cutaneou1 expand
Nerandomilast is being developed to help people with systemic sclerosis by potentially improving symptoms and slowing disease progression. This study is open to adults who are at least 18 years old and have systemic sclerosis (SSc). People can join the study if they have limited or diffuse cutaneous SSc with disease onset within 7 years of the first non-Raynaud's symptom. The purpose of this study is to find out whether a medicine called nerandomilast helps people with systemic sclerosis. This study also aims to find out how well nerandomilast is tolerated in people with systemic sclerosis. Participants are put into 2 groups randomly, which means by chance. One group takes nerandomilast tablets and the other group takes placebo tablets. Placebo tablets look like nerandomilast tablets but do not contain any medicine. Participants take the tablets twice a day. Participants are in the study for 1 to about 4 years. During this time, they visit the study site regularly and get phone calls from the site staff. During study visits participants regularly have blood samples taken and doctors check changes in skin thickening, lung function, and internal organs, overall health and the safety and tolerability of study treatment in people with SSc. The results are compared between the groups to see whether the treatment works. The doctors also regularly check participants' health and take note of any unwanted effects. Type: Interventional Start Date: Jul 2026 |
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Continuous Dual Aspiration Technique With Zoom System for Stroke
Imperative Care, Inc.
Ischemic Stroke
Acute Stroke
This study is designed to evaluate the effectiveness, safety and clinical performance of
ADAPT 2.0, first-line aspiration neurothrombectomy with Zoom System with Continuous Dual
Aspiration Technique (CDAT). expand
This study is designed to evaluate the effectiveness, safety and clinical performance of ADAPT 2.0, first-line aspiration neurothrombectomy with Zoom System with Continuous Dual Aspiration Technique (CDAT). Type: Observational Start Date: Mar 2026 |
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Nuestro Valor: Increasing Healthier Food Access for Rural Latino Communities Through a Food Retail1
University of Washington
Hispanic Americans
Chronic Disease Prevention
Cancer Prevention
Fruit and Vegetable Consumption
The goal of this clinical trial is to adapt and implement The Value of Our Health (Our
Value), a program to promote eating fruit and vegetables, for people living in rural
areas. The program will be offered in small, independent grocery stores and delivered by
community health workers. The main que1 expand
The goal of this clinical trial is to adapt and implement The Value of Our Health (Our Value), a program to promote eating fruit and vegetables, for people living in rural areas. The program will be offered in small, independent grocery stores and delivered by community health workers. The main question this study will answer is: Do customers who shop at stores receiving Our Value eat more fruits and vegetables than customers of other stores? Type: Interventional Start Date: Apr 2026 |
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Symbiotic-Lung-10: A Study to Learn About PF-08634404 Alone or in Combination in Early-stage or Loc1
Pfizer
Carcinoma
Non-Small-Cell Lung
Lung Cancer (NSCLC)
Lung Neoplasms
Carcinoma, Non-Small-Cell Lung (NSCLC)
This study is being done to learn more about a new medicine called PF-08634404. The study
team wants to understand how well it works when given alone or with chemotherapy. The
study is for adults with early stage or locally advanced non-small cell lung cancer
(NSCLC) that may or may not be removabl1 expand
This study is being done to learn more about a new medicine called PF-08634404. The study team wants to understand how well it works when given alone or with chemotherapy. The study is for adults with early stage or locally advanced non-small cell lung cancer (NSCLC) that may or may not be removable with surgery. The study is seeking participants who: - Are aged 18 years or older - Have either: - Early-stage or locally advanced (Stage II or IIIA/B) NSCLC and are a candidate for neoadjuvant therapy, followed by surgical removal of the tumor. Neoadjuvant therapy is a treatment given as a first step to shrink the tumor before surgery. - Early-stage or locally advanced (Stage II or IIIA/B) NSCLC and are a candidate for adjuvant therapy and did not achieve a pathological complete response (pCR) from approved treatment that was administered before surgery. Adjuvant therapy is an additional cancer treatment given after the primary treatment to lower the risk that the cancer will come back. pCR is defined as absence of viable tumor in all surgically removed samples. - Locally advanced (Stage III) NSCLC that may not be removable with surgery, was treated with concurrent chemoradiotherapy (cCRT), and is a candidate for additional treatment, otherwise known as consolidation therapy. cCRT is chemotherapy and radiation given simultaneously. - Be in good physical condition and have healthy organs based on medical tests. - Do not have known actionable changes in DNA The study has 3 parts and each participant will be assigned to one part by their doctor based on their disease diagnosis: - Part A will test PF-08634404 given with chemotherapy in the neoadjuvant setting, followed by surgery. - Part B will test PF-08634404 alone in adults who already were treated with neoadjuvant chemo-immunotherapy, underwent surgery, and did not achieve pCR per tumor tissue pathology analysis. Neoadjuvant chemo-immunotherapy refers to the combination of chemotherapy with immunotherapy per local standard-of-care, given before surgical removal of the tumor. - Part C will test PF-08634404 alone in adults with unresectable disease who received cCRT and did not have progressive disease. Progressive disease refers to a condition that grows, spreads, or worsens. All treatments will be done at clinical study sites, where a trained medical team will monitor adults during and after each visit. Type: Interventional Start Date: Jun 2026 |
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Identifying Effective and Cost-Conscious Maintenance Daratumumab Dosing
Eden Biltibo
Multiple Myeloma
This phase II trial tests daratumumab given at a reduced frequency with lenalidomide for
maintenance therapy for the cost effective treatment of patients with multiple myeloma
post stem cell transplant. Darzalex Faspor (also known as Daratumumab-hyaluronidase) is a
combination of two drugs used alo1 expand
This phase II trial tests daratumumab given at a reduced frequency with lenalidomide for maintenance therapy for the cost effective treatment of patients with multiple myeloma post stem cell transplant. Darzalex Faspor (also known as Daratumumab-hyaluronidase) is a combination of two drugs used alone or with other drugs to treat adults with certain types of multiple myeloma or light chain amyloidosis. Daratumumab binds to a protein called CD38, which is found on some types of immune cells and cancer cells, including myeloma cells. Daratumumab may block CD38 and help the immune system kill cancer cells. Hyaluronidase allows daratumumab to be given by injection under the skin. Daratumumab and hyaluronidase can be given in less time than daratumumab alone, which is given as an infusion. Lenalidomide may stop or slow cancer cells by blocking the growth of new blood vessels necessary for tumor growth. Daratumumab-hyaluronidase is typically given every 4 weeks per standard of care. Giving it every 8 weeks for the first year followed by every 16 weeks for years 2 through 4 in combination with lenalidomide may be equally as effective and reduce costs and treatment visits for patients with multiple myeloma post stem cell transplant. Type: Interventional Start Date: Apr 2026 |
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Efficacy and Safety Study to Evaluate SD-101 in Epidermolysis Bullosa
Paradigm Therapeutics
Epidermolysis Bullosa (EB)
The upcoming trial is for EB patients is a topically applied whole-body treatment of
patients with either Simplex, RDEB or Junctional (nH) ages 1 month to 12 years old at
study entry. There are only 4 site visits by patients with minimal assessments over a
2-month period, and patients completing th1 expand
The upcoming trial is for EB patients is a topically applied whole-body treatment of patients with either Simplex, RDEB or Junctional (nH) ages 1 month to 12 years old at study entry. There are only 4 site visits by patients with minimal assessments over a 2-month period, and patients completing this study will have the ability to continue receiving SD-101-6.0 at home in an open-label extension study. The drug product and placebo require no special preparation or storage conditions (room temperature). Type: Interventional Start Date: Jul 2026 |
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A Study to Evaluate the Efficacy and Safety of Once Daily Treprostinil Palmitil Inhalation Powder (1
Insmed Incorporated
Pulmonary Arterial Hypertension
The primary objective of this study is to evaluate the effect of 24-weeks of once daily
treatment with TPIP compared with placebo on exercise capacity in adults with PAH. expand
The primary objective of this study is to evaluate the effect of 24-weeks of once daily treatment with TPIP compared with placebo on exercise capacity in adults with PAH. Type: Interventional Start Date: Jul 2026 |
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Symbiotic-Lung-14: A Study to Learn About the Study Medicine Called PF08634404 in Combination With1
Pfizer
Small Cell Lung Cancer
Small Cell Lung Cancer ( SCLC )
Transformed Small Cell Lung Cancer
Lung Neoplasms
Carcinoma, Small Cell Lung
This study is being done to learn more about a new medicine called PF-08634404. The study
team wants to understand how well PF-08634404 works when given alone or with chemotherapy
. Chemotherapy is a type of cancer treatment that uses medicines to destroy cancer cells
or stop them from growing. The1 expand
This study is being done to learn more about a new medicine called PF-08634404. The study team wants to understand how well PF-08634404 works when given alone or with chemotherapy . Chemotherapy is a type of cancer treatment that uses medicines to destroy cancer cells or stop them from growing. The study is for adults with Transformed Small Cell Lung Cancer (T-SCLC ). T SCLC is a rare lung cancer that happens when one type of lung cancer changes into a more aggressive type after treatment stops working. To join the study, participants must meet the following conditions: - Are aged 18 years or older - Diagnosed with T-SCLC and have not received treatment for this type of lung cancer (a single cycle of chemotherapy may be permitted) - Prior diagnosis of epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer treated with tyrosine kinase inhibitors (TKIs) - Have healthy organs based on medical tests and are in good physical condition After joining the study, adults will be given chemotherapy in addition to the study medicine. After this combination treatment is finished, the study medicine will be continued alone. Adults will receive the treatment through IV infusions (medicine given directly into a vein). All treatments will be done at clinical study sites, where a trained medical team will monitor adults during and after each visit. Type: Interventional Start Date: Jul 2026 |
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Implementation Study of Lenacapavir Pre-exposure Prophylaxis for HIV Prevention
Gilead Sciences
HIV Infections
The goal of this observational study is to generate real-life information on the use of
lenacapavir (LEN, YEZTUGO®, (YTG)) for pre-exposure prophylaxis (PrEP) across diverse
clinical settings in the United States. The study will characterize how PrEP is
initiated, used, and discontinued in routine1 expand
The goal of this observational study is to generate real-life information on the use of lenacapavir (LEN, YEZTUGO®, (YTG)) for pre-exposure prophylaxis (PrEP) across diverse clinical settings in the United States. The study will characterize how PrEP is initiated, used, and discontinued in routine clinical practice when LEN is added as PrEP option and will evaluate persistence on LEN PrEP. The primary objective of this study is to evaluate real-life persistence on LEN PrEP at Week 52 in diverse clinical settings in the United States. Type: Observational Start Date: Mar 2026 |
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Integrating a Prescription Produce Program Within a Diabetes Prevention Program
Virginia Commonwealth University
Prediabetes
Type 2 Diabetes remains a major chronic disease among adults in the United States. A way
to prevent Type 2 Diabetes is to engage in a diabetes prevention program. In the diabetes
prevention program, individuals at risk of Type 2 Diabetes meet with a health coach to
learn effective ways to build hea1 expand
Type 2 Diabetes remains a major chronic disease among adults in the United States. A way to prevent Type 2 Diabetes is to engage in a diabetes prevention program. In the diabetes prevention program, individuals at risk of Type 2 Diabetes meet with a health coach to learn effective ways to build health behaviors around diet and physical activity. Individuals who participate in the diabetes prevention program are more likely to lose weight and eat a healthy diet. Type: Interventional Start Date: May 2026 |
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Synchronization of Theta to Influence Memory
Florida International University
Cognitive Control
Memory
This study aims to clarify relations between brain oscillations and two cognitive
functions: cognitive control and memory. expand
This study aims to clarify relations between brain oscillations and two cognitive functions: cognitive control and memory. Type: Interventional Start Date: Mar 2026 |
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A Study Comparing Higher Dose Chemotherapy Over a Shorter Amount of Time to Lower Dose Chemotherapy1
Children's Oncology Group
Rhabdomyosarcoma
This phase III trial compares higher dose chemotherapy, with vincristine, dactinomycin
and cyclophosphamide, over a shorter amount of time to lower dose chemotherapy plus
maintenance, with vincristine, dactinomycin, cyclophosphamide, irinotecan and
vinorelbine, over a longer amount of time, along w1 expand
This phase III trial compares higher dose chemotherapy, with vincristine, dactinomycin and cyclophosphamide, over a shorter amount of time to lower dose chemotherapy plus maintenance, with vincristine, dactinomycin, cyclophosphamide, irinotecan and vinorelbine, over a longer amount of time, along with standard of care surgery and radiation, in patients with newly diagnosed intermediate risk rhabdomyosarcoma. Vincristine and vinorelbine are in a class of medications called vinca alkaloids. They work by stopping tumor cells from growing and dividing and may kill them. Dactinomycin is a type of antibiotic that is only used in cancer chemotherapy (antineoplastic antibiotic). It works by damaging the cell's DNA and may kill tumor cells. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's DNA and may kill tumor cells. It may also lower the body's immune response. Irinotecan is in a class of antineoplastic medications called topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and DNA repair and may kill tumor cells. It is not yet known if the higher dose chemotherapy over a shorter amount of time or the lower dose chemotherapy with maintenance over a longer amount of time is more effective in the treatment of patient with newly diagnosed, intermediate risk rhabdomyosarcoma. Type: Interventional Start Date: Jul 2026 |
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Transcriptomic Profile Changes in Benign Tracheal Stenosis: Wedge Resection vs. Radial Incision
University of Maryland, Baltimore
Tracheal Stenosis
Some people develop a narrowing of their windpipe (trachea), called benign tracheal
stenosis, which can make it hard to breathe. Doctors often treat this by using a
bronchoscope-a thin, flexible tube with a camera-to open up the airway or remove scar
tissue. While these procedures help patients bre1 expand
Some people develop a narrowing of their windpipe (trachea), called benign tracheal stenosis, which can make it hard to breathe. Doctors often treat this by using a bronchoscope-a thin, flexible tube with a camera-to open up the airway or remove scar tissue. While these procedures help patients breathe better, we do not fully understand why the narrowing occurs or how the tissue heals afterward. The purpose of this study is to better understand the biological changes in the airway tissue before and after these standard medical procedures. During the procedure, small samples of tissue that would already be collected as part of normal care will be analyzed in the laboratory. The results may help doctors learn more about airway healing and could guide better treatments in the future. Type: Interventional Start Date: May 2026 |
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A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Ocular My1
UCB Biopharma SRL
Ocular Myasthenia Gravis
The purpose of the study is to demonstrate the efficacy, safety and tolerability of
rozanolixizumab compared with placebo in the treatment of adult study participants with
Ocular Myasthenia Gravis. expand
The purpose of the study is to demonstrate the efficacy, safety and tolerability of rozanolixizumab compared with placebo in the treatment of adult study participants with Ocular Myasthenia Gravis. Type: Interventional Start Date: May 2026 |
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A Study to Evaluate Efficacy and Safety of MK-8690 in Participants With Moderately to Severely Acti1
Merck Sharp & Dohme LLC
Colitis Ulcerative
Ulcerative Colitis
The purpose of this protocol is to evaluate the efficacy of MK-8690 in participants with
moderately to severely active ulcerative colitis. The primary hypothesis is that MK-8690
is superior to placebo with respect to the proportion of participants achieving clinical
remission per Modified Mayo Scor1 expand
The purpose of this protocol is to evaluate the efficacy of MK-8690 in participants with moderately to severely active ulcerative colitis. The primary hypothesis is that MK-8690 is superior to placebo with respect to the proportion of participants achieving clinical remission per Modified Mayo Score at Week 12. Type: Interventional Start Date: Mar 2026 |
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A Long-Term Open-Label Study of ML-007C-MA in Adults With Schizophrenia
MapLight Therapeutics
Schizophrenia
ML-007C-MA-212 is a 52-week open-label study designed to evaluate the long-term safety,
tolerability, and effectiveness of ML-007C-MA in participants with schizophrenia who have
recently completed an antecedent study (ML-007C-MA-211) or enroll directly (De Novo
Cohort). expand
ML-007C-MA-212 is a 52-week open-label study designed to evaluate the long-term safety, tolerability, and effectiveness of ML-007C-MA in participants with schizophrenia who have recently completed an antecedent study (ML-007C-MA-211) or enroll directly (De Novo Cohort). Type: Interventional Start Date: Mar 2026 |
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A Phase 2 Safety, Tolerability, PK, and Efficacy Study of CS-1103 Following Fentanyl Challenge With1
Clear Scientific, Inc.
Fentanyl Overdose
Fentanyl Poisoning
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and
efficacy of CS-1103 (1,000 mg), given by intravenous (IV) infusion in healthy
participants in the presence of a clinically relevant dose of fentanyl (200 μg, IV), with
naloxone blockade (400 μg, IV). expand
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of CS-1103 (1,000 mg), given by intravenous (IV) infusion in healthy participants in the presence of a clinically relevant dose of fentanyl (200 μg, IV), with naloxone blockade (400 μg, IV). Type: Interventional Start Date: May 2026 |
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A Phase 1 Study of D3S-003 as Monotherapy in Participants With Advanced Solid Tumors With a KRAS p.1
D3 Bio (Wuxi) Co., Ltd
KRAS P.G12D
This is a first-in-human (FIH) multicenter, open-label, dose-escalation Phase 1 clinical
trial to evaluate safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and
preliminary efficacy of D3S-003 in participants with advanced KRAS p.G12D mutant solid
tumors. expand
This is a first-in-human (FIH) multicenter, open-label, dose-escalation Phase 1 clinical trial to evaluate safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of D3S-003 in participants with advanced KRAS p.G12D mutant solid tumors. Type: Interventional Start Date: May 2026 |
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CAPRI: A Phase 3 Randomized, Double-Masked Study Comparing the Efficacy of EYP-1901 Against Afliber1
EyePoint Pharmaceuticals, Inc.
Diabetic Macular Edema
DME
Diabetic Macular Edema (DME)
This is a phase 3 randomized, double -masked study comparing the efficacy of EYP-1901
against Aflibercept. expand
This is a phase 3 randomized, double -masked study comparing the efficacy of EYP-1901 against Aflibercept. Type: Interventional Start Date: Feb 2026 |
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Study of AZD4956 as Monotherapy and in Combination With Anti-Cancer Agents in Participants With Adv1
AstraZeneca
Solid Tumours
The purpose of this modular, first trial in human study is to assess the safety,
tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of
ascending dose levels (DLs) of AZD4956 monotherapy and in combination with other
anti-cancer agents in participants with advanced/1 expand
The purpose of this modular, first trial in human study is to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of ascending dose levels (DLs) of AZD4956 monotherapy and in combination with other anti-cancer agents in participants with advanced/metastatic solid tumours with homologous recombination repair (HRR) deficiencies. Type: Interventional Start Date: Mar 2026 |
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Study Evaluating the Safety and Efficacy of HWK-007, a PTK7-directed Antibody Drug Conjugate in Par1
Whitehawk Therapeutics, Inc.
Endometrial Cancer
Ovarian Cancer
Ovarian Cancer Metastatic
Ovarian Cancer Metastatic Recurrent
Non-squamous EGFR Wt NSCLC
HWK-007-101 is a multicenter, open-label, first-in-human (FIH) Phase 1 study evaluating
HWK-007, a protein tyrosine kinase 7 (PTK7)-targeted antibody drug conjugate (ADC), in
adult participants with advanced or metastatic solid tumors known to be expressing PTK7.
The study employs a sequential dose1 expand
HWK-007-101 is a multicenter, open-label, first-in-human (FIH) Phase 1 study evaluating HWK-007, a protein tyrosine kinase 7 (PTK7)-targeted antibody drug conjugate (ADC), in adult participants with advanced or metastatic solid tumors known to be expressing PTK7. The study employs a sequential dose escalation and dose expansion design without a control group. Type: Interventional Start Date: Dec 2025 |
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PrP-targeting siRNA Safety & Mechanism Study
Broad Institute of MIT and Harvard
Prion Disease
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and
pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients. expand
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients. Type: Interventional Start Date: May 2026 |
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Roll-over Study for Participants Who Have Completed a Previous Clinical Study With Benralizumab (Fa1
AstraZeneca
Asthma
Eosinophilic Granulomatosis With Polyangiitis (EGPA)
Hypereosinophilic Syndrome (HES)
The rationale of the roll-over study (ROSY) is to provide continuous access to study
treatment for participants who have completed or exited a parent study and are deemed
appropriate for continued benralizumab treatment, as judged by the Investigator, while
monitoring long-term safety and tolerabil1 expand
The rationale of the roll-over study (ROSY) is to provide continuous access to study treatment for participants who have completed or exited a parent study and are deemed appropriate for continued benralizumab treatment, as judged by the Investigator, while monitoring long-term safety and tolerability of benralizumab. Type: Interventional Start Date: Jul 2026 |
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A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spi1
Biogen
Muscular Atrophy, Spinal
In this study, researchers will learn more about the safety and effects of BIIB115, also
known as salanersen. Specifically, researchers will learn more about how salanersen works
in babies who have already been treated with onasemnogene abeparvovec (OA) after being
diagnosed with SMA.
Most people1 expand
In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have already been treated with onasemnogene abeparvovec (OA) after being diagnosed with SMA. Most people with SMA have changes in a gene called survival motor neuron 1, also known as SMN1. These changes lower the amount of SMN protein in their bodies. Without enough of this protein, motor neurons and muscles cannot work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen works by helping the SMN2 gene to make more SMN protein. OA works by replacing the missing or abnormal SMN1 gene. Sometimes, OA treatment may not work as well as expected. As a result, researchers are exploring whether giving another drug after OA could lead to better outcomes for people with SMA. In this study, participants will have 2 SMN2 copies. The higher the copy number, the less severe the participant's SMA is. They will also have received treatment with OA by the time they were 42 days old and before showing any symptoms of SMA. The main goal of the study is to learn more about the safety of giving salanersen to babies after OA treatment. Researchers will also learn more about whether salanersen can help make SMA symptoms less serious. The main question researchers want to answer in this study is: • How many participants have adverse events and serious adverse events after treatment? Researchers will also learn more about: - The effects on participants' motor symptoms and how many new movement milestones participants achieve. - How many participants stay free of SMA symptoms. - How much neurofilament protein is found in the blood after treatment. - How much salanersen gets into the fluid surrounding the brain and spinal cord. - How much salanersen gets into the blood. Researchers will use different tests to learn if motor symptoms are changing, including the World Health Organization (WHO) motor milestones and Hammersmith Infant Neurological Examination (HINE) Section 2 motor milestones. The study will be done in 2 parts. Part A will last 1 year while Part B will last up to 4 years. The study will be done as follows: - First, participants will be screened to check if they can join the study. The screening period will be up to 6 months. Participants must have received OA treatment before the age of 42 days and started screening within 6 months of the OA dose. - Participants will be assigned to 1 of 2 treatment groups by chance. This is a "double blind" study which means neither the participants, study doctor, nor site staff will know which treatment group the participants are assigned to. - In this study, salanersen will be given as an intrathecal injection, which is an injection into the fluid surrounding the spine. This is done by a procedure called a lumbar puncture (LP) which involves inserting a needle into the lower back into the space around the spinal cord. - During Part A, one group will receive 80 milligrams (mg) of salanersen while another group receives a sham (fake) procedure. This means that a small needle prick will be done, but no injection will be given. - For each participant, the first visit of Part A will be 6 months after they receive OA treatment. - Part A will have up to 6 clinic visits and 2 phone calls and last up to 1 year. - During Part B, both groups of participants will receive 80 mg of salanersen once a year. - Part B will have up to 12 clinic visits and 14 phone calls and last up to 4 years. - In total, participants will be in the study for up to 5 and a half years. Type: Interventional Start Date: Sep 2026 |