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A Phase I/II Study of IVONESCIMAB in Recurrent Glioblastoma
M.D. Anderson Cancer Center
Glioblastoma
The goal of Phase 1 of this clinical research study is to find the highest tolerable dose
and the recommended Phase 2 dose of ivonescimab that can be given to patients who have
recurrent glioblastoma.
The goal of Phase 2 of this clinical research study is to learn if the recommended Phase
2 dose o1 expand
The goal of Phase 1 of this clinical research study is to find the highest tolerable dose
and the recommended Phase 2 dose of ivonescimab that can be given to patients who have
recurrent glioblastoma.
The goal of Phase 2 of this clinical research study is to learn if the recommended Phase
2 dose of ivonescimab found in Phase 1 can help to control the disease.
Type: Interventional
Start Date: Jan 2025
open study
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A Study of Remternetug (LY3372993) in Early Alzheimer's Disease (TRAILRUNNER-ALZ 3)
Eli Lilly and Company
Alzheimer's Disease
The purpose of this study is to measure the difference in time to developing or worsening
memory, thinking, or functional problems due to Alzheimer's disease occurring in
participants receiving study drug compared to placebo.
Participation could last up to 255 weeks including screening, a double-b1 expand
The purpose of this study is to measure the difference in time to developing or worsening
memory, thinking, or functional problems due to Alzheimer's disease occurring in
participants receiving study drug compared to placebo.
Participation could last up to 255 weeks including screening, a double-blind treatment
period, and a double-blind observation period. In addition, eligible participants who
receive placebo during the double-blind treatment period may choose to extend their study
participation to receive open-label remternetug in an extension period.
Type: Interventional
Start Date: Oct 2024
open study
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A Study to Investigate Safety and Effectiveness of BGB-16673 in Combination With Other Agents in Pa1
BeiGene
B-cell Malignancy
Relapsed Cancer
Refractory Cancer
B-cell Lymphoma
The purpose of this study is to measure the safety, preliminary antitumor activity,
pharmacokinetics, and pharmacodynamics with BGB-16673 in combination with other agents in
participants with relapsed or refractory (R/R) B-cell malignancies. This study is
structured as a master protocol with separa1 expand
The purpose of this study is to measure the safety, preliminary antitumor activity,
pharmacokinetics, and pharmacodynamics with BGB-16673 in combination with other agents in
participants with relapsed or refractory (R/R) B-cell malignancies. This study is
structured as a master protocol with separate substudies. This study currently includes
four substudies, and more substudies may be added as other combination agents are
identified.
Type: Interventional
Start Date: Nov 2024
open study
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Eltrombopag as a Novel Therapeutic Approach for Low-risk MDS and CMML With TET2 Mutations
Abhay Singh, MD MPH
Myelodysplastic Syndromes
Chronic Myelomonocytic Leukemia
The purpose of this study is to evaluate if a study drug called eltrombopag can improve
the blood cell counts in patients with low-risk Myelodysplastic Syndromes (MDS) and
Chronic Myelomonocytic Leukemia (CMML) with mutations in TET2 gene, observe changes in
the TET2 gene over time, and evaluate th1 expand
The purpose of this study is to evaluate if a study drug called eltrombopag can improve
the blood cell counts in patients with low-risk Myelodysplastic Syndromes (MDS) and
Chronic Myelomonocytic Leukemia (CMML) with mutations in TET2 gene, observe changes in
the TET2 gene over time, and evaluate the effectiveness of the treatment. TET2 gene is
one of the most frequently mutated genes (altered parts of the DNA) in MDS and CMML.
Eltrombopag is a Food and Drug Administration (FDA) approved drug for the treatment of
severe aplastic anemia and low levels of platelets in patients with persistent or chronic
immune thrombocytopenia (ITP) and chronic hepatitis C. Eltrombopag is considered
investigational (experimental) in this study because the FDA has not approved its use in
the treatment of low-risk MDS or CMML. Eltrombopag is a drug that helps stimulate the
body's process of making more platelets (small components of blood that help with
clotting) by interacting with specific parts of cells. This interaction starts a series
of signals that encourage the growth and development of the cells that produce platelets.
It was found that this drug could stop the growth of TET2 mutated cells.
Type: Interventional
Start Date: Jun 2025
open study
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A Trial to Test the Safety and Efficacy of TEV-53408 in Treating Vitiligo
Teva Branded Pharmaceutical Products R&D, Inc.
Vitiligo
The primary objective of the trial is to evaluate the safety of TEV-53408 administered
subcutaneously for the treatment of adults with vitiligo.
A secondary objective is to further evaluate the safety of TEV-53408.
The planned study period per participant is 84 weeks including a screening period1 expand
The primary objective of the trial is to evaluate the safety of TEV-53408 administered
subcutaneously for the treatment of adults with vitiligo.
A secondary objective is to further evaluate the safety of TEV-53408.
The planned study period per participant is 84 weeks including a screening period (up to
4 weeks), a 24-week open-label treatment period, a 16-week washout period, and a 40-week
follow-up period.
Type: Interventional
Start Date: Nov 2024
open study
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Testing the Addition of an Anti-cancer Drug, DT2216, to the Usual Chemotherapy Treatment for Relaps1
Children's Oncology Group
Childhood Fibrolamellar Carcinoma
Recurrent Childhood Fibrolamellar Carcinoma
Recurrent Childhood Malignant Solid Neoplasm
Recurrent Fibrolamellar Carcinoma
Recurrent Malignant Solid Neoplasm
This phase I/II trial tests the safety, side effects and best dose of DT2216 in
combination with irinotecan and how well it works in treating children, adolescents and
young adults with solid tumors and fibrolamellar cancer that has come back after a period
of improvement (relapsed) or that has not1 expand
This phase I/II trial tests the safety, side effects and best dose of DT2216 in
combination with irinotecan and how well it works in treating children, adolescents and
young adults with solid tumors and fibrolamellar cancer that has come back after a period
of improvement (relapsed) or that has not responded to previous treatment (refractory).
DT2216 is an anti-apoptotic protein B-cell lymphoma-extra large targeted protein
degrader. It may stop the growth of tumor cells by blocking Bcl-xL, a protein needed for
tumor cell survival. Irinotecan is in a class of antineoplastic medications called
topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and
deoxyribonucleic acid repair and may kill tumor cells. Giving DT2216 in combination with
irinotecan may be safe, tolerable, and/or effective in treating children, adolescents and
young adults with relapsed or refractory solid tumors or fibrolamellar cancer.
Type: Interventional
Start Date: Feb 2026
open study
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Study to Assess the Efficacy of Rina-S Compared to Treatment of Investigator's Choice in Participan1
Genmab
Platinum-resistant Ovarian Cancer
This phase 3 study will be conducted in different countries all over the world.
The purpose of this study is to compare how well Rina-S works against platinum-resistant
ovarian cancer compared to chemotherapy drugs that are already approved and used for
platinum-resistant ovarian cancer.
Treatmen1 expand
This phase 3 study will be conducted in different countries all over the world.
The purpose of this study is to compare how well Rina-S works against platinum-resistant
ovarian cancer compared to chemotherapy drugs that are already approved and used for
platinum-resistant ovarian cancer.
Treatment in this study could be Rina-S or it could be 1 of 4 indicated chemotherapy
agents that are considered standard medical care. There is an equal (50:50) chance of
getting Rina-S or an approved chemotherapy agent as treatment in this study. No one will
know what treatment they are assigned to until the first dose.
All participants will receive active drug; no one will be given placebo.
Type: Interventional
Start Date: Feb 2025
open study
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Ruxolitinib and Enzalutamide for the Treatment of Metastatic Castration-Resistant Prostate Cancer
University of Michigan Rogel Cancer Center
Castration-Resistant Prostate Carcinoma
Metastatic Prostate Adenocarcinoma
Stage IVB Prostate Cancer AJCC v8
This phase I/II tests the safety, side effects and best dose of ruxolitinib in
combination with enzalutamide and how well it works in treating patients with prostate
cancer that remains despite blocking hormone production (castration-resistant) and that
has spread from where it first started to oth1 expand
This phase I/II tests the safety, side effects and best dose of ruxolitinib in
combination with enzalutamide and how well it works in treating patients with prostate
cancer that remains despite blocking hormone production (castration-resistant) and that
has spread from where it first started to other places in the body (metastatic).
Ruxolitinib, a kinase inhibitor, slows down the growth of the tumor by blocking the
proteins, JAK1 and JAK2, tumors use to grow. Enzalutamide, an androgen receptor
inhibitor, works by blocking the effects of androgen (a male reproductive hormone). This
may help stop the growth and spread of tumor cells that need testosterone to grow. Giving
ruxolitinib in combination with enzalutamide may be safe, tolerable, and/or effective in
treating metastatic castration-resistant prostate cancer.
Type: Interventional
Start Date: Jul 2025
open study
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A Study of Tacrolimus/Methotrexate/Ruxolitinib Versus Post-Transplant Cyclophosphamide/Tacrolimus/M1
Incyte Corporation
Graft-versus-host Disease (GVHD)
The purpose of this study is to assess Tacrolimus/Methotrexate/Ruxolitinib versus
Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in
Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell
Transplantation expand
The purpose of this study is to assess Tacrolimus/Methotrexate/Ruxolitinib versus
Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in
Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell
Transplantation
Type: Interventional
Start Date: Apr 2025
open study
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Wearable Evaluation of Ambulatory Readings for Blood Pressure
Stephen Juraschek
Normal Blood Pressure
Controlled Hypertension
Uncontrolled Hypertension
The purpose of this project is to compare the performance and validity of novel wearable
technologies that measure blood pressure (BP) and physical activity with a Spacelabs
Ambulatory Blood Pressure Monitoring (ABPM) device. expand
The purpose of this project is to compare the performance and validity of novel wearable
technologies that measure blood pressure (BP) and physical activity with a Spacelabs
Ambulatory Blood Pressure Monitoring (ABPM) device.
Type: Interventional
Start Date: Jun 2025
open study
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Time Restricted Eating (TRE) in Metastatic Head and Neck Squamous Cell Cancer (mHNSCC)
Duke University
Metastatic Head-and-neck Squamous-cell Carcinoma
The purpose of this study is to evaluate whether Time Restricted Eating (TRE) can improve
responses in participants with metastatic head and neck squamous cell cancer (mHNSCC)
receiving Immune Checkpoint Blockers (ICB) by changing the gut microbiome (the bacteria
and other microorganisms living in1 expand
The purpose of this study is to evaluate whether Time Restricted Eating (TRE) can improve
responses in participants with metastatic head and neck squamous cell cancer (mHNSCC)
receiving Immune Checkpoint Blockers (ICB) by changing the gut microbiome (the bacteria
and other microorganisms living in individual's bodies). A particular focus of this study
is to compare the outcomes of African American participants when compared to the rest of
the participant population. TRE is a form of intermittent fasting where food and drink
intake is limited to a specific time window during the day. The information learned from
this study may help researchers develop new strategies to improve outcomes in patients
with mHNSCC in the future.
Participants will be asked to complete a dietary survey at baseline and week 9 and
provide a baseline stool and blood sample. Two weeks before beginning ICB and after
participants completed the baseline assessments, they will begin TRE. TRE will be defined
as limiting food and drink intake to a 10 hour window during each day and fasting for 14
hours at night. Participants will be asked to complete a daily food log to document the
times they eat and drink. On day 1 of ICB and weeks 3, 6, 9, 26, and 52 after ICB,
participants will be asked to collect a blood sample and a toxicity assessment will be
performed. On day 1 of ICB and weeks 9, 26, and 52 of ICB, participants will be asked to
provide a stool sample. Participants will also undergo tumor imaging throughout the study
as part of their standard of care assessments. If a participant's disease progresses
after ICB, they will repeat all study assessments and be withdrawn from the study.
Type: Interventional
Start Date: Jun 2025
open study
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Safety and Pharmacodynamic Effects of BIIB122 in Participants With LRRK2-Associated Parkinson's Dis1
Denali Therapeutics Inc.
Parkinson Disease
This Phase 2a, multicenter, randomized, 12-week double-blind, placebo-controlled,
parallel-group study, followed by an OLE, is designed to evaluate the safety,
tolerability, and pharmacodynamic effects of BIIB122 in participants with LRRK2-PD.
LRRK2-PD is defined as Parkinson's Disease (PD) in indi1 expand
This Phase 2a, multicenter, randomized, 12-week double-blind, placebo-controlled,
parallel-group study, followed by an OLE, is designed to evaluate the safety,
tolerability, and pharmacodynamic effects of BIIB122 in participants with LRRK2-PD.
LRRK2-PD is defined as Parkinson's Disease (PD) in individuals who are heterozygous or
homozygous carriers of a pathogenic LRRK2 variant that increases LRRK2 kinase activity.
Type: Interventional
Start Date: Oct 2024
open study
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MOONRAY-01, A Study of LY3962673 in Participants With KRAS G12D-Mutant Solid Tumors
Eli Lilly and Company
Pancreatic Ductal Adenocarcinoma
Non-small Cell Lung Cancer
Colorectal Cancer
The main purpose of this study is to assess safety & tolerability and antitumor activity
of LY3962673 as monotherapy and in combination with other chemotherapy agents in
participants with KRAS G12D-mutant advanced solid tumor types. The study is expected to
last approximately 5 years. expand
The main purpose of this study is to assess safety & tolerability and antitumor activity
of LY3962673 as monotherapy and in combination with other chemotherapy agents in
participants with KRAS G12D-mutant advanced solid tumor types. The study is expected to
last approximately 5 years.
Type: Interventional
Start Date: Sep 2024
open study
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A Study to Assess Adverse Events and How Intravenously (IV) Infused Telisotuzumab Vedotin (ABBV-3991
AbbVie
Non-Small Cell Lung Cancer
Cancer is a condition where cells in a specific part of body grow and reproduce
uncontrollably. Non-small cell lung cancer (NSCLC) is a solid tumor, a disease in which
cancer cells form in the tissues of the lung. The purpose of this study is to assess how
safe telisotuzumab vedotin is in adult par1 expand
Cancer is a condition where cells in a specific part of body grow and reproduce
uncontrollably. Non-small cell lung cancer (NSCLC) is a solid tumor, a disease in which
cancer cells form in the tissues of the lung. The purpose of this study is to assess how
safe telisotuzumab vedotin is in adult participants with NSCLC. Change in disease
activity and adverse events will be assessed.
Telisotuzumab vedotin is an investigational drug being developed for the treatment of
NSCLC. Participants will be randomly assigned a treatment of telisotuzumab vedotin in 1
of 3 arms at an 1:1:1 ratio. Each group receives intravenous (IV) infusion of
telisotuzumab vedotin at different doses. Approximately 150 adult participants with c-Met
overexpressing NSCLC will be enrolled in the study at approximately 70 to 80 sites
worldwide.
Participants will receive IV telisotuzumab vedotin at 1 of 3 dose regimens as part of a 3
year study duration.
There may be higher treatment burden for participants in this trial compared to their
standard of care. Participants will attend regular visits during the study at a hospital
or clinic. The effect of the treatment will be checked by medical assessments, blood
tests, checking for side effects and completing questionnaires.
Type: Interventional
Start Date: Jan 2025
open study
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A Study to Test if TEV-56286 is Effective in Relieving Multiple System Atrophy (MSA): A Safety and1
Teva Branded Pharmaceutical Products R&D, Inc.
Multiple System Atrophy
The primary objective of the study is to evaluate the efficacy of TEV-56286 administered
orally for the treatment of adult participants with Multiple System Atrophy (MSA).
A secondary objective of the study is to evaluate specific efficacy parameters of
TEV-56286.
Another secondary objective is t1 expand
The primary objective of the study is to evaluate the efficacy of TEV-56286 administered
orally for the treatment of adult participants with Multiple System Atrophy (MSA).
A secondary objective of the study is to evaluate specific efficacy parameters of
TEV-56286.
Another secondary objective is to evaluate the safety and tolerability of TEV-56286.
The planned study period per participant is 56 weeks including a screening period (up to
4 weeks), a 48-week double-blind treatment period, and a follow-up visit (approximately 4
weeks after the end of the double-blind treatment period). The study duration will be
approximately 27 months.
Type: Interventional
Start Date: Oct 2024
open study
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Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomati1
Eidos Therapeutics, a BridgeBio company
Amyloidosis
Amyloid Cardiomyopathy
Transthyretin Amyloidosis
Cardiomyopathies
Heart Diseases
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin
(TTR) protein falls apart and forms amyloid, a sticky plaque- like substance that
accumulates in different organs in the body and can cause damage to the organ. There are
two ways that the TTR protein can fall1 expand
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin
(TTR) protein falls apart and forms amyloid, a sticky plaque- like substance that
accumulates in different organs in the body and can cause damage to the organ. There are
two ways that the TTR protein can fall apart. One way occurs as a person ages, where the
normal TTR protein can fall apart and form amyloid that may no longer be sufficiently
cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way
occurs when a person inherits a defective TTR gene that causes the TTR protein to
spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and
can be detected in adults by a genetic test of their TTR gene before they age.
Amyloid build-up in the heart causes the heart wall to become thick and stiff and can
result in heart failure and even death. Accumulation of TTR amyloid in the heart is known
as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve
tissues leading to nerve problems. Accumulation of TTR in the nerves is known as
transthyretin amyloid polyneuropathy or ATTR-PN.
Acoramidis is an experimental drug designed to bind tightly to TTR in the blood and
stabilize its structure, so it does not form the harmful amyloid plaques that can cause
damage to organs.
This study is intended to determine if treatment with acoramidis in participants with
ATTRv who have not yet developed any symptoms of disease can prevent or delay the
development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene
are treated early before any of the symptoms of disease have developed, it may be
possible to delay the onset or prevent the disease entirely.
Type: Interventional
Start Date: May 2025
open study
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Study of Targeted Therapies for the Treatment of Adult Participants With Moderate to Severe Crohn's1
AbbVie
Crohn's Disease
Crohn's disease (CD) is a long-lasting disease that causes severe inflammation (redness,
swelling), in the digestive tract, most frequently affecting the bowels. It can cause
many different symptoms including belly pain, diarrhea, tiredness, and weight loss.
Treatments are available but do not work1 expand
Crohn's disease (CD) is a long-lasting disease that causes severe inflammation (redness,
swelling), in the digestive tract, most frequently affecting the bowels. It can cause
many different symptoms including belly pain, diarrhea, tiredness, and weight loss.
Treatments are available but do not work the same for all patients or may stop working
over time. This study will evaluate the effectiveness and adverse events of targeted
therapies (TaTs) for adult participants with moderate to severe CD.
The medicines assessed in this study are risankizumab, ABBV-382 and lutikizumab. When
participants join the study, they will be randomized into available study treatment
groups. Adult participants with CD will be enrolled. Around 500 participants will be
enrolled in the study at approximately 300 sites worldwide.
Risankizumab and ABBV-382 are given as an injection under the skin or as an infusion into
the vein. Lutikizumab is given as an injection under the skin. Each group includes a
12-week induction period, a 12-week maintenance period, and an optional long-term
extension period where medication will be given after the maintenance period.
There may be higher treatment burden for participants in this trial compared to their
standard of care treatment without participating in this study. Participants will attend
regular visits during the study at a hospital or clinic. The effect of the treatment will
be checked by medical assessments, blood tests, endoscopies, checking for side effects
and completing questionnaires and a daily diary.
Type: Interventional
Start Date: Sep 2024
open study
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TReatment of ADC-Refractory Breast CancEr With Dato-DXd or T-DXd: TRADE DXd
Ana C Garrido-Castro, MD
Breast Cancer
HER2-low Breast Cancer
Metastatic Breast Cancer
HER2-negative Breast Cancer
The purpose of this study is to test the safety and effectiveness of the sequence of two
investigational drugs (trastuzumab deruxtecan followed by datopotamab deruxtecan, or
datopotamab deruxtecan followed by trastuzumab deruxtecan) to learn whether the treatment
works in treating HER2-negative (HE1 expand
The purpose of this study is to test the safety and effectiveness of the sequence of two
investigational drugs (trastuzumab deruxtecan followed by datopotamab deruxtecan, or
datopotamab deruxtecan followed by trastuzumab deruxtecan) to learn whether the treatment
works in treating HER2-negative (HER2-low or HER2-0) metastatic breast cancer.
The names of the study drugs involved in this study are:
- Datopotamab deruxtecan (a type of antibody drug conjugate)
- Trastuzumab deruxtecan (a type of antibody drug conjugate)
Type: Interventional
Start Date: Oct 2024
open study
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A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Place1
Novartis Pharmaceuticals
Generalized Myasthenia Gravis
The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III
study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+
gMG who are on stable SOC treatment. Participants who meet the eligibility criteria will
be randomized in a ratio of 1:1, to r1 expand
The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III
study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+
gMG who are on stable SOC treatment. Participants who meet the eligibility criteria will
be randomized in a ratio of 1:1, to receive either iptacopan or matching placebo, for 6
months (180 days) while continuing on a stable SOC treatment. The randomization will be
stratified based on region.
Type: Interventional
Start Date: Jul 2024
open study
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Global Real World Data in Patients With Advanced Thyroid Cancer on Standard of Care and Specialized1
M.D. Anderson Cancer Center
Thyroid Cancer
The goal of this clinical research study is to learn about the barriers in the real world
to accessing treatments for ATC. And to learn about how patients with ATC tolerate and
respond to the commercially available medications for treatment of this disease, outside
of a clinical study. expand
The goal of this clinical research study is to learn about the barriers in the real world
to accessing treatments for ATC. And to learn about how patients with ATC tolerate and
respond to the commercially available medications for treatment of this disease, outside
of a clinical study.
Type: Observational
Start Date: Dec 2024
open study
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A Safety and Efficacy Study Evaluating CTX131 in Adult Subjects With Relapsed/Refractory Hematologi1
CRISPR Therapeutics
T Cell Lymphoma
B Cell Lymphoma
Acute Myeloid Leukemia
This is an open label, multicenter, phase 1/2 dose evaluation and cohort expansion study
evaluating the safety and efficacy of CTX131 in subjects with Relapsed/Refractory
Hematologic Malignancies expand
This is an open label, multicenter, phase 1/2 dose evaluation and cohort expansion study
evaluating the safety and efficacy of CTX131 in subjects with Relapsed/Refractory
Hematologic Malignancies
Type: Interventional
Start Date: Aug 2024
open study
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Multi-Center Project: Building Electronic Tools To Enhance and Reinforce CArdiovascular REcommendat1
NYU Langone Health
Heart Failure
This study will test an automated, electronic health record (EHR-)embedded alert to
improve prescribing of guideline-directed medical therapy for patients with heart failure
and reduced ejection fraction (HFrEF). The investigators have previously tested and
implemented this alert at NYU Langone Hea1 expand
This study will test an automated, electronic health record (EHR-)embedded alert to
improve prescribing of guideline-directed medical therapy for patients with heart failure
and reduced ejection fraction (HFrEF). The investigators have previously tested and
implemented this alert at NYU Langone Health (NYULH), and will now test and implement
this alert across three other health systems.
Type: Interventional
Start Date: Apr 2025
open study
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Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limb1
Claris Biotherapeutics, Inc.
Limbal Stem Cell Deficiency
This study will enroll subjects with qualifying limbal stem cell deficiency (LSCD). All
subjects will receive CSB-001 investigational drug in either one or both study eyes. The
study is comprised of two identical phases (Dosing Phase I and II) of test article dosing
separated by a 31- to 40-day per1 expand
This study will enroll subjects with qualifying limbal stem cell deficiency (LSCD). All
subjects will receive CSB-001 investigational drug in either one or both study eyes. The
study is comprised of two identical phases (Dosing Phase I and II) of test article dosing
separated by a 31- to 40-day period, the Dosing Holiday, where no test article is
administered. Dosing Phase II is followed by an observational, noninterventional phase
(Observation Phase).
Type: Interventional
Start Date: May 2024
open study
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Echography-guided Surfactant THERapy (ESTHER) For Preterm Infants With Respiratory Failure
Connecticut Children's Medical Center
Respiratory Distress Syndrome, Newborn
Pulmonary Surfactants
Intensive Care Units, Neonatal
Point-of-care ultrasound (POCUS) is the use of ultrasound by the bedside provider in real
time to answer a specific question and guide medical management. POCUS can be used to
diagnose the severity of neonatal respiratory distress syndrome (RDS) through a lung
ultrasound score. Lung ultrasound scor1 expand
Point-of-care ultrasound (POCUS) is the use of ultrasound by the bedside provider in real
time to answer a specific question and guide medical management. POCUS can be used to
diagnose the severity of neonatal respiratory distress syndrome (RDS) through a lung
ultrasound score. Lung ultrasound scores have also been shown to predict if an infant is
treated with an initial dose of surfactant. Therefore, using lung ultrasound scores to
guide surfactant therapy for RDS will likely lead to earlier surfactant therapy and may
improve short-term respiratory outcomes. This study will test this theory by comparing
lung ultrasound score-guided surfactant therapy for premature infants with RDS with our
current surfactant administration guidelines.
Type: Interventional
Start Date: Jun 2024
open study
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Phase I Study of Q702 With Azacitidine and Venetoclax for Relapsed or Refractory Acute Myeloid Leuk1
M.D. Anderson Cancer Center
Acute Myeloid Leukemia
To learn about the safety and tolerability of the drug combination of Q702, azacitidine,
and venetoclax when given to participants with relapsed/refractory AML. expand
To learn about the safety and tolerability of the drug combination of Q702, azacitidine,
and venetoclax when given to participants with relapsed/refractory AML.
Type: Interventional
Start Date: Feb 2025
open study
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