Related Experiment Video

Updated: Jun 4, 2026

Tumor Treating Field Therapy in Combination with Bevacizumab for the Treatment of Recurrent Glioblastoma
06:15

Tumor Treating Field Therapy in Combination with Bevacizumab for the Treatment of Recurrent Glioblastoma

Published on: October 27, 2014

Novel therapies for resistant focal segmental glomerulosclerosis (FONT) phase II clinical trial: study design

Howard Trachtman1, Suzanne Vento, Debbie Gipson

  • 1Cohen Children's Medical Center of New York, North Shore Long Island Jewish Health System, New Hyde Park, NY, USA. trachtma@lij.edu

BMC Nephrology
|February 12, 2011
PubMed
Abstract

Insights

This study introduces a flexible Phase II trial design to identify effective antifibrotic therapies for focal segmental glomerulosclerosis (FSGS). The Novel Therapies for Resistant Focal Segmental Glomerulosclerosis (FONT) project aims to find promising treatments for future Phase III trials.

Area of Science:

  • Nephrology
  • Clinical Trial Design
  • Pharmacology

Background:

  • Primary focal segmental glomerulosclerosis (FSGS) lacks sufficient randomized clinical trials (RCTs) for novel therapies, particularly for corticosteroid-resistant cases.
  • Advances in basic science offer new antifibrotic strategies targeting fibrosis pathways relevant to FSGS progression.
  • There is a critical need for adaptable Phase II study designs to evaluate these novel antifibrotic agents.

Purpose of the Study:

  • To investigate the efficacy of novel therapies for resistant FSGS using a multicenter Phase I/II RCT.
  • To identify agents with superior response rates for further evaluation in Phase III trials.
  • To efficiently select promising treatments and exclude ineffective ones for FSGS management.

Main Methods:

  • The Novel Therapies for Resistant Focal Segmental Glomerulosclerosis (FONT) project is a multicenter Phase I/II RCT.
  • Adalimumab and galactose will be compared against a conservative therapy (lisinopril, losartan, atorvastatin).
  • A two-step outcome analysis will be employed to expedite the selection of agents for Phase III testing.

Main Results:

  • The study is designed to have high probability of selecting a superior treatment if one exists.
  • Comparison of primary and secondary endpoints across study arms will guide the choice of treatments for future Phase III RCTs.
  • The sample size is calculated to ensure statistical power for treatment selection.

Conclusions:

  • The FONT II RCT utilizes a two-step outcome analysis to accelerate the identification of effective FSGS therapies.
  • This phased approach efficiently identifies promising agents for Phase III testing while discarding ineffective ones.
  • The study design aims to prevent significant investment in non-efficacious treatments for rare kidney diseases like FSGS.

More Related Videos

Evaluation of the Feasibility, Safety, and Accuracy of an Intraoperative High-intensity Focused Ultrasound Device for Treating Liver Metastases05:56

Evaluation of the Feasibility, Safety, and Accuracy of an Intraoperative High-intensity Focused Ultrasound Device for Treating Liver Metastases

Published on: January 9, 2019

Related Concept Videos

Clinical Trials: Overview01:11

Clinical Trials: Overview

Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
Clinical Trials01:16

Clinical Trials

Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
Nephrotic Syndrome II : Assessment and Medical Management01:26

Nephrotic Syndrome II : Assessment and Medical Management

IntroductionNephrotic syndrome is a kidney disorder marked by excessive protein loss in the urine, leading to various systemic complications. This condition often results from damage to the glomeruli—the kidney's filtering units—causing proteinuria, low blood protein levels, and fluid retention. Understanding the assessment, diagnosis, and management of nephrotic syndrome is essential for effective treatment and prevention of further kidney damage.AssessmentPatient History: Document any history...
Heart Failure V: Medical Management01:30

Heart Failure V: Medical Management

Medical Management of Acute Decompensated Heart Failure (ADHF)The primary goals of therapy for patients hospitalized with acute decompensated heart failure (ADHF) include:Relieving symptomsOptimizing volume statusSupporting oxygenation and ventilationMaintaining cardiac output (CO) and end-organ perfusionIdentifying and addressing the cause of ADHFPreventing complicationsProviding patient education on factors precipitating HF exacerbationPlanning for dischargeOngoing monitoring and assessment...
Phase II Reactions: Glucuronidation01:24

Phase II Reactions: Glucuronidation

Glucuronidation, a pivotal phase II biotransformation process, involves the coupling of glucuronic acid to a drug or xenobiotic. Given its widespread occurrence and critical role in drug metabolism, it's considered the most crucial phase II reaction. It enhances the water solubility of substances, aiding their expulsion from the body. The driving force behind these reactions is a group of enzymes known as UDP-glucuronosyltransferases (UGTs). UGTs facilitate the transfer of a glucuronic acid...
Phase II Conjugation Reactions: Overview01:14

Phase II Conjugation Reactions: Overview

Conjugation, a key component of phase II biotransformation reactions, is a vital process in drug detoxification. It involves transferring endogenous substances like glucuronic acid, sulfate, and glycine to drugs or their metabolites formed in phase I reactions. These conjugation reactions, often catalyzed by specific enzymes, transform potentially harmful metabolites into inactive, water-soluble forms easily excreted in urine or bile. By enhancing polarity and eliminating pharmacological...