CLINICAL TRIALS PROFILE FOR LIQUID PRED

505(b)(2) Clinical Trials for LIQUID PRED

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Dosage	NCT00858936 ↗	Reduction of Ischemia-Reperfusion Mediated Cardiac Injury in Subjects Undergoing Coronary Artery Bypass Graft Surgery	Terminated	Mallinckrodt	Phase 2	2009-05-01	This clinical trial will investigate the safety and effectiveness of IK-1001 (the liquid form of sodium sulfide) when used in Coronary Artery Bypass Graft (CABG) patients to potentially reduce the damage done to the heart during surgery. This study has 2 parts. Part 1 will first test 36 subjects at different doses (amount) of the study drug. There will be 6 different groups of 6 subjects each that will receive the study drug or a placebo. A placebo is a substance that will be prepared to look like the study drug but will contain no active ingredients. In Part 1, five subjects from each group will receive study drug (IK-1001) and one will receive a placebo. This first part of this study is also a dose (amount) escalation. This means that each group will be receiving a different dose of the study drug. The first group will receive the lowest dose, the second group will receive a slightly higher dose, and the third group a slightly higher dose until all six groups has been tested. You can not choose which group you will be in but prior to starting each new dose level, the data (information) from the previous dose level will have been reviewed by a group of qualified individuals to determine if it is safe to proceed to the next highest dose level. Part 2 will expand the study and will treat at least 158 (and up to 632) more subjects at a dose level that has been deemed safe from information collected from Part 1. Subjects in Part 2 of the study will have a 1 in 2 (50%) chance of receiving the study drug or placebo. Whether the subject gets study drug or the placebo will be randomly assigned (like the toss of a coin). The study drug or placebo will be given as an intravenous infusion (into the vein) for six hours while the subject is having their CABG surgery. The subjects will be followed up for 6 months after their CABG surgery.
OTC	NCT00894634 ↗	Study Evaluating Brompheniramine Maleate Liquid in Children and Adolescents	Completed	Wyeth is now a wholly owned subsidiary of Pfizer	Phase 1	2009-03-21	The objective of this study is to characterize the pharmacokinetic (PK) profile of brompheniramine maleate (BROM) in children and adolescents, ages 2 to less than 18 years following dosing in accordance with current weight-age dosing guidelines. Once characterized, the PK data will be pooled with adult PK data from other studies and analyzed under a separate analysis plan to confirm or refine the existing OTC doses in children aged 2 to <12 yrs and adolescents aged 12 to <18 yrs.
OTC	NCT00262145 ↗	Ability of a Tea Leaf Extracts Preparation to Slow Down Carbohydrate and Fat Absorption	Completed	NatureGen	Phase 1	2005-10-01	Objective - A variety of herbal, over-the-counter preparations of tea leaves are said to reduce the rate of absorption of fat ( allegedly via inhibition of pancreatic lipase) and carbohydrate (via inhibition of carbohydrate digestion and blocking of glucose transport by the intestinal mucosa). There has been some study of the ability of these products to reduce the blood glucose increase observed after a carbohydrate meal and to reduce blood cholesterol levels in chronic studies. The purpose of the present study is to objectively determine if one cup of "tea" made from a combination of three types of tea leaves (mulberry, black and green tea) can cause malabsorption of carbohydrate and fat taken in conjunction with the tea. Research Design - The study will consist of a double blind, placebo controlled crossover study in 20 healthy subjects. On one of two days (one week apart) the subjects will ingest a standard meal consisting of 30 g of sucrose (in the tea) and 30 g of starch in the form of white rice plus 10 g of fat as butter. To measure triglyceride absorption, each meal will also contain 250 mg of 13-C labeled triolein. Triolein is a commonly ingested fat consisting of glycerol bound to three oleic acids. 13-C is a stable (non-radioactive) isotope of carbon. On one of the test days the subjects (randomly) will concurrently consume the active preparation, a tea containing extracts of the three types of tea leave described above plus the meal, and on the other test day they will consume the meal with a liquid placebo preparation (warm water, sugar and food coloring). Subjects will provide a breath sample before and at hourly intervals for 8 hours after ingestion of the meal. Carbohydrate malabsorption will be determined by the hydrogen concentration in the breath samples and fat malabsorption by the concentration of 13-CO2 in the breath samples. Clinical Significance - An increase in breath hydrogen indicates carbohydrate malabsoption and a low 13-CO2 indicates lipid malabsorption. Objective evidence that the tea leaf extract actually induces carbohydrate and/or fat malabsorption could provide the basis for further studies.

All Clinical Trials for LIQUID PRED

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000320 ↗	Buprenorphine Formulation Comparison: Sublingual Tablet vs. Solution - 1	Completed	National Institute on Drug Abuse (NIDA)	Phase 1/Phase 2	1997-10-01	The purpose of this study is to compare subject response to liquid vs. tablet formulations, to assess bioequivalency of liquid vs. tablet, to compare subject preference, and to evaluate if dose response curve for tablet is equal to liquid form."
NCT00000140 ↗	The Silicone Study	Completed	National Eye Institute (NEI)	Phase 3	1985-09-01	To compare, through a randomized, multicenter surgical trial, the postoperative tamponade effectiveness of intraocular silicone oil with that of an intraocular long-acting gas (initially sulfur hexafluoride [SF 6 ], later perfluoropropane [C 3 F 8 ]) for the management of retinal detachment complicated by proliferative vitreoretinopathy (PVR), using vitrectomy and associated techniques. To evaluate the ocular complications that result from the use of silicone oil and gas.
NCT00000302 ↗	Study Comparing Liquid and Tablet Buprenorphine Formulations - 5	Completed	National Institute on Drug Abuse (NIDA)	Phase 3	1969-12-31	The purpose of this study is to compare liquid and tablet buprenorphine formulations.

Clinical Trial Conditions for LIQUID PRED

Condition Name

Condition Name for LIQUID PRED
Intervention	Trials
Healthy	79
Breast Cancer	26
Healthy Volunteers	26

Condition MeSH

Condition MeSH for LIQUID PRED
Intervention	Trials
Diabetes Mellitus	50
Infections	46
Infection	40

Clinical Trial Locations for LIQUID PRED

Trials by Country

+

−

Trials by Country for LIQUID PRED
Location	Trials
Italy	99
Germany	93
France	79
Spain	79
Brazil	59

Trials by US State

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−

Trials by US State for LIQUID PRED
Location	Trials
California	169
Texas	143
New York	110
Maryland	101
Ohio	99

Clinical Trial Progress for LIQUID PRED

Clinical Trial Phase

Clinical Trial Phase for LIQUID PRED
Clinical Trial Phase	Trials
Phase 4	265
Phase 3	163
Phase 2/Phase 3	47

Clinical Trial Status

Clinical Trial Status for LIQUID PRED
Clinical Trial Phase	Trials
Completed	853
Recruiting	225
Not yet recruiting	141

Clinical Trial Sponsors for LIQUID PRED

Sponsor Name

Sponsor Name for LIQUID PRED
Sponsor	Trials
National Cancer Institute (NCI)	89
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	27
M.D. Anderson Cancer Center	27

Sponsor Type

Sponsor Type for LIQUID PRED
Sponsor	Trials
Other	1699
Industry	715
NIH	225

Clinical Trials, Market Analysis, and Projections for Rett Syndrome Treatments: A Focus on Trofinetide and Other Candidates

Introduction

Rett syndrome, a rare genetic disorder primarily affecting girls, has seen significant advancements in recent years through clinical trials and research. This article will delve into the current state of clinical trials, market analysis, and projections for treatments like trofinetide (DAYBUE) and other promising candidates.

Understanding Rett Syndrome

Rett syndrome is a neurodevelopmental disorder caused by mutations in the MECP2 gene. It leads to severe symptoms including intellectual disability, seizures, and loss of motor skills. The need for effective treatments is critical, and several companies are actively involved in clinical trials.

Clinical Trials Update

Trofinetide (DAYBUE)

Trofinetide, marketed as DAYBUE, has been a significant breakthrough in Rett syndrome treatment. Here are some key updates:

• Approval and Expansion: DAYBUE was approved by Health Canada in October 2024, following its approval in the U.S. Acadia Pharmaceuticals acquired global rights from Neuren Pharmaceuticals in July 2023 and plans to expand access to Europe, Asia, and other regions[1].
• Clinical Trials: The LOTUS Phase 4 clinical study is ongoing, with six-month interim findings showing improvements in nonverbal communication, alertness, and social interaction. Common side effects included diarrhea and formed/normal stool. Over 90% of patients were on the full labeled dose by week 10[1].

Anavex Life Sciences - ANAVEX®2-73 (Blarcamesine)

Anavex Life Sciences is conducting several clinical trials for ANAVEX®2-73:

• EXCELLENCE Phase 2/3 Study: Although the study did not meet its co-primary endpoints at 12 weeks, it showed no new serious adverse events (SAEs), and over 91% of participants continued into the 48-week open-label study[1].
• AVATAR Phase 3 Trial: This trial met its primary and secondary efficacy endpoints, indicating promising results for blarcamesine in treating Rett syndrome[1].

Taysha Gene Therapy - TSHA-102

Taysha Gene Therapy is advancing with its gene therapy candidate, TSHA-102:

• Clinical Development: The first adult patient was dosed in June 2023, and interim data from the first two patients showed clinical improvements in motor skills, communication, socialization, autonomic function, and seizures without serious adverse events[1].
• Expansion and Recruitment: The trial is currently recruiting female participants aged 12 and older in Montreal, Canada, and the U.S., with plans to evaluate patients over the next 60+ months[1].

Market Analysis

Global Clinical Trials Market

The global clinical trials market is growing significantly, driven by the increasing prevalence of chronic diseases and government initiatives:

• Market Size: The global clinical trials market was valued at USD 84.61 billion in 2024 and is projected to reach USD 146.60 billion by 2033, growing at a CAGR of 6.07% during the forecast period (2025–2033)[3].
• Drivers: The rise in chronic diseases such as cancer, diabetes, and cardiovascular disorders is a major driver. Government funding and initiatives also support the expansion of clinical trials[3].

Rett Syndrome Treatment Market

While specific market size data for Rett syndrome treatments is not readily available, the approval and ongoing clinical trials indicate a growing market:

• Trofinetide Impact: The approval of DAYBUE has opened up new treatment avenues for Rett syndrome patients. Its global expansion plans suggest a potential increase in market share and revenue[1].
• Competitive Landscape: Companies like Anavex Life Sciences and Taysha Gene Therapy are also making significant strides, which could further diversify the treatment options and market competition[1].

Projections and Future Outlook

Trofinetide (DAYBUE)

Given its recent approvals and ongoing studies, DAYBUE is expected to dominate the Rett syndrome treatment market in the near future:

• Global Expansion: As Acadia Pharmaceuticals expands access to other regions, the market reach and revenue for DAYBUE are likely to increase[1].
• Long-term Efficacy: The LOTUS study's positive interim findings suggest long-term benefits, which could solidify DAYBUE's position in the market[1].

Emerging Treatments

Other treatments, such as ANAVEX®2-73 and TSHA-102, are in various stages of clinical trials and show promising results:

• Anavex Life Sciences: Despite the EXCELLENCE study's mixed results, the AVATAR trial's success and ongoing open-label studies indicate potential for blarcamesine in the market[1].
• Taysha Gene Therapy: The positive interim data from TSHA-102 trials suggest a potential breakthrough in gene therapy for Rett syndrome, which could significantly impact the market if approved[1].

Key Takeaways

• Trofinetide (DAYBUE): Approved in the U.S. and Canada, with global expansion plans, showing improvements in nonverbal communication and social interaction.
• Anavex Life Sciences: ANAVEX®2-73 has mixed trial results but continues to show promise in ongoing studies.
• Taysha Gene Therapy: TSHA-102 gene therapy shows clinical improvements without serious adverse events, indicating a potential future treatment.
• Market Growth: The global clinical trials market is growing, driven by chronic diseases and government initiatives, which will support the development of Rett syndrome treatments.

FAQs

What is the current status of DAYBUE (trofinetide) in treating Rett syndrome?

DAYBUE has been approved by Health Canada and is undergoing a Phase 4 clinical study (LOTUS) which has shown positive interim results in nonverbal communication, alertness, and social interaction.

Which companies are actively conducting clinical trials for Rett syndrome treatments?

Companies like Acadia Pharmaceuticals, Anavex Life Sciences, and Taysha Gene Therapy are actively involved in clinical trials for Rett syndrome treatments.

What are the promising candidates besides trofinetide for Rett syndrome?

ANAVEX®2-73 (blarcamesine) by Anavex Life Sciences and TSHA-102 gene therapy by Taysha Gene Therapy are promising candidates in various stages of clinical trials.

How is the global clinical trials market impacting Rett syndrome treatment development?

The growing global clinical trials market, driven by chronic diseases and government initiatives, supports the development and funding of clinical trials for Rett syndrome treatments.

What are the potential side effects of DAYBUE (trofinetide) in Rett syndrome patients?

Common side effects of DAYBUE include diarrhea and formed/normal stool, with over 90% of patients on the full labeled dose by week 10.

Sources

1. Rett Research & Clinical Pipeline - International Rett Syndrome Foundation
2. Petroleum Liquid Feedstocks Market Analysis 2025-2032| Forecast - Wicz
3. Global Clinical Trials Market Size, Top Share, Trends, Forecast by ... - Straits Research
4. REPLENISH - UF Health
5. Liquid Synthetic Rubber Market Size & Trends Report, 2025 - Grand View Research