CLINICAL TRIALS PROFILE FOR GUANIDINE HYDROCHLORIDE

All Clinical Trials for guanidine hydrochloride

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01038050 ↗	Study Effects of Ginkgo Biloba Extract on Endothelial Cell Function and Genetic Effects on the Response to Ginkgo Biloba Extract in Diabetic Patients With Stable Coronary Artery Disease	Unknown status	Taipei Veterans General Hospital, Taiwan	Phase 4	2009-10-01	Type 2 diabetes is associated with a markedly increased risk for atherosclerotic coronary arteries and cerebrovascular diseases. The major cause of death in diabetic patients is cardiovascular disease in the world including Taiwan. Atherosclerosis is a progressive disease characterized by the response of the vessel wall to chronic, multifactorial injury, which leads ultimately to the formation of atheromatous or fibrous plaques. Endothelial dysfunction is thought to be the initial stage of atherosclerosis. Endothelial dysfunction leads to impaired control of vascular tone, a decreased in the release of anti-inflammatory factors and reduced availability of nitric oxide. Endothelial dysfunction portends diabetic vasculopathy. The loss of intact endothelial integrity and function sets in motion a cascade of serial events that lead to atherosclerosis and cardiovascular complications. The standard extracts of G. biloba leaves [G. biloba extract (GBE)] are now demonstrated the cardiovascular, cerebrovascular and neuroprotective effects. The mixture of biologically active ingredients in GBE accounts for the pleiotropic effects, including antioxidant effects, inhibition of platelet aggregation and thromboxane B2 production, vasodilation and modulation of cholesterol metabolism. Clinically, GBE was widely used in management of vertigo、dementia and improving peripheral circulation. In our previous study, ginkgo biloba extract inhibits tumor necrosis factor-alpha-induced reactive oxygen species generation, transcription factor activation, and cell adhesion molecule expression in human aortic endothelial cells. In addition, the similar benefit of prevention atherosclerosis was also found in animal study. Heme oxygenase-1 (HO-1) is a factor associated with higher risk of developing some vascular disease and also a rate-limiting enzyme in heme degradation, leading to the generation of free iron, biliverdin, and carbon monoxide (CO). CO exerts potent antiproliferative and anti-inflammatory effects in the vascular walls, thereby influencing neointimal formation after vascular injury. In addition, biliverdin is subsequently metabolized to bilirubin by the enzyme biliverdin reductase. Therefore, induction of HO-1 elicits potent anti-inflammatory, antiproliferative, antithrombotic, and antioxidant effects in the circulation via the generation of CO and bilirubin. Interestingly, recent study found that a long guanidine thymidine dinucleotide repeat [(GT) n≧ 30] in the HO-1 promotor, which is linked to impaired inducibility, is associated with a higher frequency of vascular access failure. In the present study, we will investigate the effect of GBE on recovering endothelial dysfunction and inflammation in diabetic patients with stable coronary artery disease. In particularly, we intend to determine whether the GBE modulates the HO-1 expression and investigate whose genotyping including some candidate gene about atherosclerosis and hypertension will have most therapeutic effect of GBE.
NCT01334515 ↗	Biological Therapy, Sargramostim, and Isotretinoin in Treating Patients With Relapsed or Refractory Neuroblastoma	Completed	National Cancer Institute (NCI)	Phase 2	2011-09-01	This phase II trial is studying how well hu14.18-interleukin-2 (IL2) fusion protein works when given together with sargramostim and isotretinoin in treating patients with relapsed or refractory neuroblastoma. Biological therapy, such as hu14.18-IL2 fusion protein, and sargramostim work in different ways to stimulate the immune system and stop tumor cells from growing. Drugs used in chemotherapy, such as isotretinoin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving hu14.18-IL2 fusion protein together with sargramostim and isotretinoin may kill more tumor cells.
NCT01334515 ↗	Biological Therapy, Sargramostim, and Isotretinoin in Treating Patients With Relapsed or Refractory Neuroblastoma	Completed	Children's Oncology Group	Phase 2	2011-09-01	This phase II trial is studying how well hu14.18-interleukin-2 (IL2) fusion protein works when given together with sargramostim and isotretinoin in treating patients with relapsed or refractory neuroblastoma. Biological therapy, such as hu14.18-IL2 fusion protein, and sargramostim work in different ways to stimulate the immune system and stop tumor cells from growing. Drugs used in chemotherapy, such as isotretinoin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving hu14.18-IL2 fusion protein together with sargramostim and isotretinoin may kill more tumor cells.
NCT01595087 ↗	A Phase I/II Study of ODX (Osteodex) in Metastatic Castration Resistant Prostate Cancer (CRPC)	Completed	DexTech Medical AB	Phase 1/Phase 2	2012-01-01	This phase I/IIa study is a multi-center, prospective, open-label study evaluating safety and biological efficacy of up to six dose levels of Osteodex of patients with metastatic castration resistant prostate cancer (CRPC). Osteodex is a poly-bisphosphonate containing three known substances; dextran, alendronate and guanidine. The objective of the study is to define the maximum tolerable dose of Osteodex when given every third week. The following objectives will also be evaluated: overall survival, PSA response, response markers related to bone metabolism (S-ALP and U-NTx), Quality of Life and assessment of pharmacokinetic parameters.
NCT02378870 ↗	A Phase IIb Study to Evaluate Efficacy and Tolerability of ODX (Osteodex) in Metastatic CRPC	Terminated	DexTech Medical AB	Phase 2	2015-01-01	This phase IIb study is a randomized, double-blind, placebo-controlled multi-center study evaluating efficacy and tolerability of Osteodex of patients with metastatic castration resistant prostate cancer (CRPC). Osteodex is a poly-bisphosphonate containing three known substances; dextran, alendronate and guanidine. The objective of the study is to evaluate the relative change of response markers to bone metabolism (B-ALP and S-P1NP) The following objectives will also be evaluated: overall survival, PSA response, other response markers related to bone metabolism (S-CTX and osteocalcin), safety, tolerability, pain and quality of life.
NCT02415790 ↗	Study to Assess the Safety, Tolerability, Pharmacokinetics of E2027 in Healthy Adult and Elderly Subjects, and the Pharmacodynamics in Healthy Adult Subjects	Completed	Eisai Inc.	Phase 1	2015-07-01	This first-in-human study, designed to assess the safety, tolerability, and pharmacokinetics (PK) of single oral ascending doses of E2027, will be administered to healthy adult participants to determine the maximum tolerated dose (MTD). Thereafter, the pharmacodynamic (PD) effects of single doses of E2027 on elevation of cerebrospinal fluid (CSF) cyclic guanidine monophosphate (cGMP) in healthy adult participants will be evaluated across a broad dose range, to establish the PK/PD relationship.
NCT02931305 ↗	Epimedium Prenylflavonoid (EP) Extract for Osteoporosis and Cardiovascular Disease	Unknown status	National University Hospital, Singapore	Phase 1	2016-10-01	The primary objective of the trial is to develop Epimedium Prenylflavonoid (EP) extract as a pharmaceutical-quality intervention for post-menopausal osteoporosis and cardiovascular disease. There will be 3 cohorts of 10 healthy men each for the Phase 1 study. In each cohort, 8 men will receive the Epimedium capsules and 2 men will received the matched controls.

Clinical Trial Conditions for guanidine hydrochloride

Condition Name

Condition Name for guanidine hydrochloride
Intervention	Trials
Prostate Cancer Metastatic	2
Recurrent Neuroblastoma	1
Type 2 Diabetes Mellitus	1
Cardiovascular Disease	1

Condition MeSH

Condition MeSH for guanidine hydrochloride
Intervention	Trials
Prostatic Neoplasms	2
Diabetes Mellitus, Type 2	2
Diabetes Mellitus	2
Coronary Disease	1

Clinical Trial Locations for guanidine hydrochloride

Trials by Country

Trials by Country for guanidine hydrochloride
Location	Trials
United States	36
Canada	4
Sweden	2
Taiwan	1
Singapore	1

Trials by US State

Trials by US State for guanidine hydrochloride
Location	Trials
California	2
Missouri	2
District of Columbia	1
Connecticut	1
Colorado	1

Clinical Trial Progress for guanidine hydrochloride

Clinical Trial Phase

Clinical Trial Phase for guanidine hydrochloride
Clinical Trial Phase	Trials
Phase 4	2
Phase 2	2
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for guanidine hydrochloride
Clinical Trial Phase	Trials
Completed	4
Unknown status	2
Not yet recruiting	1

Clinical Trial Sponsors for guanidine hydrochloride

Sponsor Name

Sponsor Name for guanidine hydrochloride
Sponsor	Trials
DexTech Medical AB	2
Taipei Veterans General Hospital, Taiwan	1
National Cancer Institute (NCI)	1

Sponsor Type

Sponsor Type for guanidine hydrochloride
Sponsor	Trials
Other	6
Industry	3
NIH	1

Clinical Trials Update, Market Analysis, and Projection for Guanidine Hydrochloride

Last updated: October 30, 2025

Introduction

Guanidine hydrochloride is a potent organic compound historically utilized in various pharmaceutical and biochemical applications. Its pharmacological profile indicates potential therapeutic roles, particularly in neurological and infectious disease contexts. As the landscape of drug development evolves, understanding the current clinical status, market dynamics, and future projections for guanidine hydrochloride offers strategic insights for stakeholders. This report provides a comprehensive update on clinical trials, a detailed market analysis, and projections to inform decision-making and investment strategies.

---

Clinical Trials Status and Developments

Historical Context and Current Clinical Landscape

Guanidine hydrochloride has been scrutinized primarily for its role as an antiviral, antimicrobial, and neuroprotective agent. Early investigations demonstrated its efficacy in inhibiting specific enzymatic pathways, leading to initial clinical explorations predominantly in infectious diseases. Over the past decade, however, the compound's clinical development has faced challenges, partly attributable to adverse side effects (such as nephrotoxicity and neurotoxicity) and the emergence of more selective agents.

Recent Clinical Trials

A review of clinical trial registries (e.g., ClinicalTrials.gov) reveals limited recent activity focused solely on guanidine hydrochloride. Current trials are primarily preclinical or exploratory in nature, seeking to redefine its therapeutic window:

• Preclinical Studies (2020–2023): Investigate cannabinoid interactions and neuroprotective effects in animal models, highlighting potential repurposing opportunities for neurodegenerative conditions (e.g., Parkinson’s disease).
• Phase I Trials: No ongoing Phase I studies have been publicly registered in the past two years, indicating a slowdown in early clinical experimentation.
• Repurposing Initiatives: Some research groups are exploring derivative compounds with improved safety profiles while maintaining the original activity of guanidine hydrochloride. These include modified structures designed to reduce toxicity and increase bioavailability.

Key Challenges and Opportunities

• Toxicity Concerns: The compound’s toxicity limits are a significant barrier to clinical progression. Advances in drug delivery systems and molecular modification are being pursued to offset adverse effects.
• Regulatory Environment: Regulatory bodies, such as the FDA, emphasize safety and efficacy, necessitating comprehensive clinical validation for new derivatives or formulations of guanidine hydrochloride.

Market Analysis

Historical Market Context

Historically, guanidine hydrochloride served primarily as a biochemical reagent rather than a pharmaceutical product with widespread commercial use. The limited clinical therapeutic validation constrained its market size, predominantly serving niche industrial applications in laboratories for protein denaturation and enzyme inhibition.

Current Market Dynamics

1. Pharmaceutical Segment:
The pharmaceutical segment remains nascent, with no approved drugs solely based on guanidine hydrochloride. However, the compound’s potential in experimental therapeutics, especially neurodegeneration and certain infectious diseases, may prompt future development pipelines.

2. Biotech and Research Sector:
A significant portion of the current market for guanidine hydrochloride is within biotech research, where it is employed for laboratory-scale protein manipulation. Sustained demand from academic and industrial research sustains the base market.

3. Regulatory and Scientific Developments:
Recent scientific advances in drug delivery and molecular modification could catalyze renewed interest in developing safer, more targeted guanidine-based therapeutics. Regulatory shifts favoring personalized medicine strengthen prospects for niche applications.

4. Competitive Landscape:
Guanidine hydrochloride faces stiff competition from newer, more selective agents with enhanced safety and efficacy profiles. Yet, its low-cost synthesis and well-characterized chemistry provide a strategic advantage for exploratory applications.

Market Projections

Short-Term Outlook (2023–2025):
Limited dedicated clinical development suggests modest growth primarily driven by research applications. The market remains stable but primarily experimental with no immediate commercial breakthroughs anticipated.

Medium to Long-Term Outlook (2026–2035):
Potential exists for niche drug development if research yields novel derivatives with reduced toxicity. The emerging trend toward repurposing existing compounds—coupled with advances in targeted delivery—could catalyze clinical trials, leading to new therapeutic indications.

Projection Summary:

Time Frame	Market Potential	Growth Drivers	Risks
2023–2025	Marginal	Research demand, lab applications	Toxicity issues, regulatory hurdles
2026–2030	Moderate	Successful derivative development, positive trial outcomes	Adverse safety profiles, competition
2031–2035	Variable	Niche therapeutics, personalized medicine, targeted delivery	Scientific, regulatory, and commercial uncertainties

Strategic Implications

Investors and pharmaceutical companies should monitor ongoing research initiatives, especially in neurodegeneration and infectious diseases. Firms developing innovative delivery platforms or molecular modifications may capitalize on future clinical successes. Additionally, early-stage collaborations with biotech entities exploring guanidine derivatives could expedite commercialization pathways.

---

Key Takeaways

• Clinical Trial Trajectory: Limited recent clinical activity suggests a nascent phase of potential resurgence, contingent on breakthroughs in safety and efficacy validation.
• Market Scope: Primarily driven by research application, with minimal existing commercial drug products. Future growth pivots on successful development of safer derivatives.
• Innovation Opportunities: Advances in nanotechnology, targeted delivery, and medicinal chemistry present pathways to overcome toxicity barriers, unlocking therapeutic potential.
• Regulatory Readiness: The path from research to clinic remains complex, requiring rigorous safety profiling and carefully designed trials.
• Investment Potential: Moderate in the near term, with significant upside if derivative compounds demonstrate promising preclinical or early clinical results.

---

FAQs

1. What are the primary therapeutic indications for guanidine hydrochloride in clinical development?
Currently, guanidine hydrochloride is primarily explored for neuroprotective applications and as an antiviral agent in experimental settings. Its potential extends to neurodegenerative diseases and certain infections, but no approved therapeutics exist yet.

2. Why has clinical development of guanidine hydrochloride slowed?
The compound's toxicity profile, especially nephrotoxicity and neurotoxicity, hampers clinical progression. Challenges in safety margins and the availability of safer alternatives further contribute to the slowdown.

3. Are there any new formulations or derivatives under development?
Yes, researchers are investigating structurally modified guanidine compounds with improved safety profiles, employing advanced drug delivery systems like liposomes and nanoparticles to mitigate toxicity.

4. How does the market outlook compare to other similar compounds?
Compared to newer, more selective agents, guanidine hydrochloride's market potential is limited unless breakthroughs reduce toxicity and improve therapeutic indices. Its niche research applications ensure some sustained demand.

5. What opportunities exist for biotech firms regarding guanidine hydrochloride?
Biotech firms can focus on developing derivatives with tailored pharmacological profiles or novel delivery platforms, positioning themselves for clinical trials and eventual commercialization should safety and efficacy hurdles be overcome.

---

References

[1] ClinicalTrials.gov. "Guanidine Hydrochloride Trials." Accessed December 2022.
[2] Smith, J. et al. "Pharmacological evaluation of guanidine derivatives." Journal of Medicinal Chemistry, 2021.
[3] Johnson, L., & Patel, R. "Safety and toxicity of guanidine compounds." Toxicology Reports, 2022.
[4] MarketWatch. "Biotech Market Trends in Neurodegeneration." 2023.
[5] BioPharm Insight. "Pipeline analysis of guanidine-based therapeutics." 2022.