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Clinical Trial News

Milk Elimination Diet Shows Promise as First-Line Therapy for Pediatric Eosinophilic Esophagitis

  • A new study suggests that eliminating animal milk alone may be as effective as a four-food elimination diet for children with eosinophilic esophagitis (EoE).
  • The randomized, multi-site clinical trial compared a one-food (milk) elimination diet (1FED) with a four-food (milk, egg, wheat, soy) elimination diet (4FED) in 63 children with EoE.
  • Results showed that 1FED and 4FED achieved similar remission rates and improvements in symptoms, histologic, and endoscopic features over 12 weeks.
  • Starting with milk elimination alone may be an easier and more tolerable initial approach for managing EoE in children, potentially increasing patient compliance.

Florida Cancer Specialists Presents Real-World Evidence Research on Multiple Myeloma at International Meeting

Real World Evidence
  • Florida Cancer Specialists & Research Institute presented research on minimal residual disease testing perspectives among community hematologists treating multiple myeloma patients at the International Myeloma Society meeting.
  • The research utilized real-world data from over one million patients treated by FCS, demonstrating the organization's expanding capabilities in generating clinical insights from large-scale patient datasets.
  • Multiple myeloma represents the second most common hematologic malignancy in the U.S., affecting primarily older patients with two-thirds of newly diagnosed cases occurring in patients 65 years or older.

Click Therapeutics' CT-132 Digital Therapeutic Demonstrates Efficacy in Migraine Prevention

  • CT-132, a prescription digital therapeutic, significantly reduced monthly migraine days (MMDs) in a Phase 3 trial for episodic migraine prevention.
  • The ReMMi-D study showed a statistically significant reduction of 0.9 MMDs relative to the sham control group over a 12-week period (p = 0.005).
  • Patients treated with CT-132 experienced improvements in migraine-specific quality of life and reduced disability, with benefits observed as early as week 4.
  • The digital therapeutic was well-tolerated, with no treatment-related adverse events reported, offering a potential new option for migraine management.

Study of Two Digital Therapeutics for the Prevention of Episodic Migraine Receiving CGRP Therapy (ReMMiD-C)

NCT06004388CompletedPhase 3
Click Therapeutics, Inc.
Posted 7/21/2023

Study of Two Digital Therapeutics for the Prevention of Episodic Migraine

NCT05853900CompletedNot Applicable
Click Therapeutics, Inc.
Posted 3/28/2023

Obeticholic Acid Shows Long-Term Benefit in Primary Biliary Cholangitis

  • COBALT trial data, published in The American Journal of Gastroenterology, shows obeticholic acid (OCA) significantly reduces adverse outcomes in PBC patients.
  • The study compared OCA-treated patients to an external control arm derived from real-world U.S. claims data.
  • OCA treatment was associated with a 61% lower relative risk of death, liver transplant, or hepatic decompensation (HR, 0.39; P=0.0010).
  • Findings support the use of external control arms in rare disease trials, where placebo groups pose ethical and practical challenges.

5-FU Bolus Omission Reduces Toxicity in Advanced GI Cancer Chemotherapy

  • A recent study involving over 11,000 patients suggests that omitting the 5-FU bolus in multidrug chemotherapy regimens for advanced gastrointestinal cancers can reduce side effects.
  • The omission of the 5-FU bolus did not compromise overall survival in patients with colorectal, pancreatic, or gastroesophageal malignancies.
  • Patients receiving the 5-FU bolus experienced significantly higher rates of neutropenia and thrombocytopenia compared to those who received continuous infusion only.
  • This adjustment in chemotherapy administration may improve treatment tolerability and the overall experience for patients with advanced gastrointestinal cancers.

CRISPR Technology Shows Promise Across Diverse Clinical Applications

• CRISPR-Cas9 gene editing has achieved a significant milestone with FDA approval of Casgevy for sickle cell disease and transfusion-dependent beta thalassemia. • Clinical trials are underway for CRISPR-based therapies targeting urinary tract infections, hereditary transthyretin amyloidosis, hereditary angioedema, and cardiovascular diseases. • CRISPR technology is being explored for chronic conditions like type 1 diabetes, systemic lupus erythematosus, and HIV, with early trials showing potential for disease management. • Challenges remain in addressing the high costs of CRISPR therapies and establishing regulatory standards to manage potential off-target effects and ethical implications.

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

NCT04853576Active, Not RecruitingPhase 1
Editas Medicine, Inc.
Posted 5/4/2021

CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy for Relapsed/Refractory B Cell Non-Hodgkin Lymphoma (ANTLER)

NCT04637763RecruitingPhase 1
Caribou Biosciences, Inc.
Posted 5/26/2021

Safety, Tolerability, and PK of LBP-EC01 in Patients With Lower Urinary Tract Colonization Caused by E. Coli

NCT04191148CompletedPhase 1
Locus Biosciences
Posted 12/30/2019

NTLA-2001 in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)

2024-511170-69-00Active, Not RecruitingPhase 1
Intellia Therapeutics Inc.

A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease

NCT05398029CompletedPhase 1
Verve Therapeutics, Inc.
Posted 7/5/2022

A Safety and Efficacy Study Evaluating CTX112 in Subjects With Relapsed or Refractory B-Cell Malignancies

NCT05643742RecruitingPhase 1
CRISPR Therapeutics AG
Posted 3/10/2023

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

NCT03745287CompletedPhase 2
Vertex Pharmaceuticals Incorporated
Posted 11/27/2018

A Study of LBP-EC01 in the Treatment of Acute Uncomplicated UTI Caused by Drug Resistant E. Coli (ELIMINATE Trial)

NCT05488340RecruitingPhase 2
Locus Biosciences
Posted 7/13/2022

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

NCT06128629RecruitingPhase 3
Intellia Therapeutics
Posted 12/13/2023

A Safety and Efficacy Study Evaluating CTX131 in Adult Subjects With Relapsed or Refractory Solid Tumors

NCT05795595RecruitingPhase 1
CRISPR Therapeutics AG
Posted 3/13/2023

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

NCT05456880RecruitingPhase 1
Beam Therapeutics Inc.
Posted 8/30/2022

Study of EBT-101 in Aviremic HIV-1 Infected Adults on Stable ART

NCT05144386CompletedPhase 1
Excision BioTherapeutics
Posted 1/24/2022

An Open-Label, FIH Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With T1D

NCT05565248RecruitingPhase 1
CRISPR Therapeutics AG
Posted 1/20/2023

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCT05120830Active, Not RecruitingPhase 1
Intellia Therapeutics
Posted 12/10/2021

Deuterium-Depleted Water Shows Promise in Regulating Cancer Cell Growth

  • Two review papers analyzing nearly 200 studies suggest that deuterium-depleted water (DDW) can inhibit tumor cell proliferation and suppress cancer cell migration, potentially reducing metastasis.
  • DDW may influence cell cycle regulation by promoting autophagy, apoptosis, and senescence, with observed changes in gene expression related to cancer development.
  • A Phase 2 trial showed that prostate cancer patients receiving DDW alongside conventional therapy experienced a significantly greater reduction in prostate size compared to those receiving a placebo.
  • HYD LLC is seeking partnerships to conduct a Phase II/III clinical study to evaluate DDW's efficacy in treating cancers like glioblastoma, lung, breast, or prostate cancer.

Azithromycin Reduces Childhood Mortality in Niger: Study Shows Twice-Yearly Distribution Effective for Children Aged 1-59 Months

  • A randomized cluster trial in rural Niger showed that twice-yearly azithromycin distribution significantly reduced all-cause mortality in children aged 1-59 months.
  • The study found no significant reduction in mortality when azithromycin distribution was limited to infants aged 1-11 months.
  • The findings suggest that broader azithromycin distribution strategies may be more effective in reducing childhood mortality in high-mortality settings.
  • The research highlights the potential of azithromycin as a tool to combat childhood mortality in Sub-Saharan Africa.

Resmetirom: Duke Research Leads to First FDA-Approved Drug for MASH Liver Disease

• Resmetirom, a novel drug developed with Duke University research, has gained FDA approval as the first treatment for metabolic dysfunction-associated steatohepatitis (MASH). • The drug targets thyroid hormone pathways to stimulate fat breakdown in the liver, addressing a critical need for the estimated 115 million people worldwide affected by MASH. • While only 30% of trial participants benefited after one year and the treatment costs $50,000 annually, resmetirom marks a significant advancement, with hopes for expanded treatment options and reduced costs through further research. • Research indicates that interest in MASH is growing, with resmetirom and similar drugs potentially working alongside other therapies to combat the disease and alleviate the health burden associated with advanced liver and cardiovascular complications.

Lenvatinib and Pembrolizumab Combination Shows Promise in Non-Clear Cell Renal Cell Carcinoma Subtypes

• Lenvatinib plus pembrolizumab demonstrates a 54% overall response rate and a median progression-free survival of 17.4 months in papillary renal cell carcinoma. • For chromophobe RCC, lenvatinib/pembrolizumab shows a 34.5% ORR and a median PFS of 26.2 months, suggesting combination therapy is superior to single-agent treatment. • IO-TKI combinations, including lenvatinib/pembrolizumab, exhibit the highest response rates (around 25%) in translocation RCC, supporting their use in this population.

Randomized Phase-II Study of Nivolumab Plus Ipilimumab vs. Standard of Care in Untreated and Advanced Non-clear Cell RCC

NCT03075423Active, Not RecruitingPhase 2
Goethe University
Posted 11/1/2017

Axitinib +/- Pembrolizumab in First Line Treatment of mPRCC

NCT05096390RecruitingPhase 2
Centre Leon Berard
Posted 6/1/2022

Testing Cabozantinib With or Without Atezolizumab in Patients With Advanced Papillary Kidney Cancer, PAPMET2 Trial

NCT05411081RecruitingPhase 2
National Cancer Institute (NCI)
Posted 3/24/2023

Study of XL092 + Nivolumab vs Sunitinib in Subjects With Advanced or Metastatic Non-Clear Cell Renal Cell Carcinoma

NCT05678673Active, Not RecruitingPhase 3
Exelixis
Posted 1/1/2023

Savolitinib Plus Durvalumab Versus Sunitinib and Durvalumab Monotherapy in MET-Driven, Unresectable and Locally Advanced or Metastatic PRCC

NCT05043090Active, Not RecruitingPhase 3
AstraZeneca
Posted 10/28/2021

Testing of Bevacizumab, Erlotinib, and Atezolizumab in Combination for Advanced-Stage Kidney Cancer

NCT04981509RecruitingPhase 2
National Cancer Institute (NCI)
Posted 6/10/2022

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