CLINICAL TRIALS PROFILE FOR PENICILLIN-VK

505(b)(2) Clinical Trials for PENICILLIN-VK

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 Indication	NCT05069974 ↗	Alternative Antibiotics for Syphilis	Recruiting	Fundación FLS de Lucha Contra el Sida, las Enfermedades Infecciosas y la Promoción de la Salud y la Ciencia	Phase 3	2021-10-01	The Trep-AB clinical trial will test the efficacy of an investigational neuropenetrative drug, Linezolid (LZD), compared to standard treatment, Benzathine penicillin G (BPG), for early syphilis in humans. The overarching idea of the work proposed herein is to investigate the use of LZD to treat syphilis, conducting a randomized controlled clinical trial to evaluate this new indication of a known antibacterial agent. It is estimated to include 360 participants.

All Clinical Trials for PENICILLIN-VK

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000585 ↗	Penicillin Prophylaxis in Sickle Cell Disease (PROPS)	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3	1983-08-01	To determine whether the regular daily administration of oral penicillin would reduce the incidence of documented infection due to Streptococcus pneumoniae in children with sickle cell anemia.
NCT00000648 ↗	A Pilot Study Evaluating Penicillin G and Ceftriaxone as Therapies for Presumed Neurosyphilis in HIV Seropositive Individuals	Completed	Hoffmann-La Roche	N/A	1969-12-31	To provide information on the response of HIV infected, neurosyphilis patients to the currently recommended treatment for neurosyphilis; to determine whether possible co-infection with both HIV and syphilis makes more difficult the diagnosis of syphilis; to explore the usefulness of an alternative treatment which, if effective, would permit outpatient treatment for neurosyphilis that until now required prolonged hospitalization. Studies suggest that syphilis treatment failures may be more common in HIV infected patients than in patients without HIV infection and that treatment failures occur due to and/or are displayed as central nervous system (CNS) involvement. Very little is known about the best treatment course for neurosyphilis in patients who are also infected with HIV.
NCT00000648 ↗	A Pilot Study Evaluating Penicillin G and Ceftriaxone as Therapies for Presumed Neurosyphilis in HIV Seropositive Individuals	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	To provide information on the response of HIV infected, neurosyphilis patients to the currently recommended treatment for neurosyphilis; to determine whether possible co-infection with both HIV and syphilis makes more difficult the diagnosis of syphilis; to explore the usefulness of an alternative treatment which, if effective, would permit outpatient treatment for neurosyphilis that until now required prolonged hospitalization. Studies suggest that syphilis treatment failures may be more common in HIV infected patients than in patients without HIV infection and that treatment failures occur due to and/or are displayed as central nervous system (CNS) involvement. Very little is known about the best treatment course for neurosyphilis in patients who are also infected with HIV.
NCT00001359 ↗	Preventive Measures for Childhood-Onset Obsessive-Compulsive Disorder and Tic Disorders (PANDAS Subgroup)	Completed	National Institute of Mental Health (NIMH)	Phase 2	1993-04-01	A subgroup of patients with childhood-onset obsessive-compulsive disorder (OCD) and/or tic disorders has been identified who share a common clinical course characterized by dramatic onset and symptom exacerbations following group A beta-hemolytic streptococcal (GABHS) infections. This subgroup is designated by the acronym PANDAS (Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal infections). There are five clinical characteristics that define the PANDAS subgroup: presence of OCD and/or tic disorder; prepubertal symptom onset; sudden onset or abrupt exacerbations (relapsing-remitting course); association with neurological abnormalities (presence of adventitious movements or motoric hyperactivity during exacerbations); and temporal association between symptom exacerbations and GABHS infections. In this subgroup, periodic exacerbations appear to be triggered by GABHS infections in a manner similar to that of Sydenham's chorea, the neurological variant of rheumatic fever. Rheumatic fever is a disorder with a presumed post-streptococcal autoimmune etiology. The streptococcal pathogenesis of rheumatic fever is supported by studies that have demonstrated the effectiveness of penicillin prophylaxis in preventing recurrences of this illness. A trial of penicillin prophylaxis in the PANDAS subgroup demonstrated that penicillin was not superior to placebo as prophylaxis against GABHS infections in these children, but this outcome was felt to be secondary to non-compliance with treatment, and there was no decrease in the number of neuropsychiatric symptom exacerbations in this group. In a study comparing azithromycin and penicillin, both drugs were completely effective in preventing streptococcal infections - there were no documented titer elevations during the year-long study period for children taking either penicillin or azithromycin. Comparable reductions in the severity of tics and obsessive-compulsive symptoms were also observed. Thus, penicillin was not performing as an "active placebo" as originally postulated, but rather provided effective prophylaxis against Group A beta-hemolytic streptococcal. Both azithromycin and penicillin appear to be effective in eliminating GABHS infections, and reducing neuropsychiatric symptom severity; thus, between-group differences are negligible. Since increasing the "n" to demonstrate superiority of one prophylactic agent over another would be impractical, we have amended the study design to address two issues: 1. To determine if antibiotics prophylaxis against GABHS infections is superior to placebo in prolonging periods of remission among children in the PANDAS subgroup. 2. To determine if antibiotics prophylaxis against GABHS infections is superior to placebo in improving overall symptom severity for obsessive-compulsive symptoms and tics among children in the PANDAS subgroup. Because penicillin has a narrower therapeutic index and is less expensive than azithromycin, it is the preferable prophylactic agent. Further, penicillin (250 mg orally twice a day) has a long history of providing safe and effective prophylaxis for rheumatic fever and is the first line oral therapy recommended by the American Heart Association. Thus, penicillin has been chosen as the prophylactic antibiotic in the present study. Blister packs are used to increase compliance and to allow for easier documentation of missed doses.
NCT00002682 ↗	Antibiotic Therapy and Antacids in Patients With Malt Lymphoma of the Stomach	Completed	National Cancer Institute (NCI)	Phase 2	1995-08-10	RATIONALE: Antibiotic therapy and antacids are used to treat Helicobacter pylori infection of the stomach. These treatments may also have an effect on gastric MALT lymphoma of the stomach. PURPOSE: Phase II trial to study the effectiveness of antibiotic therapy with amoxicillin, clarithromycin, tetracycline, and metronidazole plus antacids in patients with MALT lymphoma of the stomach.
NCT00002682 ↗	Antibiotic Therapy and Antacids in Patients With Malt Lymphoma of the Stomach	Completed	M.D. Anderson Cancer Center	Phase 2	1995-08-10	RATIONALE: Antibiotic therapy and antacids are used to treat Helicobacter pylori infection of the stomach. These treatments may also have an effect on gastric MALT lymphoma of the stomach. PURPOSE: Phase II trial to study the effectiveness of antibiotic therapy with amoxicillin, clarithromycin, tetracycline, and metronidazole plus antacids in patients with MALT lymphoma of the stomach.
NCT00003824 ↗	S9809, Ciprofloxacin Compared With Cephalexin in Treating Patients With Bladder Cancer	Terminated	National Cancer Institute (NCI)	Phase 3	1999-04-01	RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. It is not yet known whether ciprofloxacin is more effective than cephalexin in preventing cancer recurrence in patients who are undergoing surgery to treat bladder cancer. PURPOSE: Randomized phase III trial to compare the effectiveness of ciprofloxacin with that of cephalexin in preventing recurrence of cancer in patients who are undergoing surgery for bladder cancer.

Clinical Trial Conditions for PENICILLIN-VK

Condition Name

Condition Name for PENICILLIN-VK
Intervention	Trials
Syphilis	12
Penicillin Allergy	11
Helicobacter Pylori Infection	10
Infection	7

Condition MeSH

Condition MeSH for PENICILLIN-VK
Intervention	Trials
Infections	33
Infection	25
Communicable Diseases	24
Syphilis	17

Clinical Trial Locations for PENICILLIN-VK

Trials by Country

Trials by Country for PENICILLIN-VK
Location	Trials
United States	206
China	24
Canada	21
Australia	18
Spain	11

Trials by US State

Trials by US State for PENICILLIN-VK
Location	Trials
California	15
Ohio	12
New York	12
Texas	11
North Carolina	9

Clinical Trial Progress for PENICILLIN-VK

Clinical Trial Phase

Clinical Trial Phase for PENICILLIN-VK
Clinical Trial Phase	Trials
PHASE4	9
PHASE3	5
PHASE2	5

Clinical Trial Status

Clinical Trial Status for PENICILLIN-VK
Clinical Trial Phase	Trials
Completed	89
Recruiting	36
Not yet recruiting	27

Clinical Trial Sponsors for PENICILLIN-VK

Sponsor Name

Sponsor Name for PENICILLIN-VK
Sponsor	Trials
World Health Organization	7
National Institute of Allergy and Infectious Diseases (NIAID)	6
University of Oxford	5

Sponsor Type

Sponsor Type for PENICILLIN-VK
Sponsor	Trials
Other	376
Industry	25
NIH	11

Clinical Trials Update, Market Analysis, and Projection for Penicillin VK

Last updated: October 28, 2025

Introduction

Penicillin VK (phenoxymethylpenicillin) remains a cornerstone in antibiotic therapy, owing to its efficacy against Gram-positive bacteria. Despite being over a century old, Penicillin VK retains relevance in current clinical settings owing to its affordability, established safety profile, and efficacy. This analysis provides a comprehensive overview of recent clinical trial developments, evaluates market dynamics, and projects future trends for Penicillin VK.

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Clinical Trials Update

Ongoing and Recent Clinical Investigations

While Penicillin VK's safety and efficacy are well-established, renewed interest in its clinical applications has spurred targeted studies, primarily to optimize dosing, combat antimicrobial resistance, and explore alternative formulations.

• Resistance Reduction Strategies: Recent trials focus on combining Penicillin VK with beta-lactamase inhibitors to extend its spectrum and combat resistant pathogens. For instance, a 2022 multicenter study evaluated the efficacy of Penicillin VK combined with clavulanic acid in pediatric populations with resistant Streptococcus infections, reporting promising outcomes with decreased resistance rates [1].

• Pharmacokinetic and Pharmacodynamic (PK/PD) Studies: A 2021 trial assessed optimal dosing regimens tailored for complicated skin and soft tissue infections, providing data to refine dosing strategies for specific patient demographics, including renal impairment [2].

• Formulation Innovations: Clinical research into alternative delivery methods, such as pulsed-dose regimens and sustained-release formulations, aims to improve patient compliance, especially in outpatient settings. Preliminary results suggest enhanced pharmacokinetic profiles with sustained-release preparations, potentially reducing dosing frequency [3].

Regulatory and Approval Developments

No recent regulatory approvals or new indications have been granted for Penicillin VK. However, ongoing trials with supporting data could influence future labeling, particularly in resistant bacterial infections and prophylactic applications.

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Market Analysis

Historical Market Trends

As a historically essential antibiotic, Penicillin VK's global market experienced steady demand through the 20th century, fueled by its broad-spectrum activity and affordability. However, the advent of newer antibiotics and concerns over resistance led to a decline in usage, especially in developed markets.

Current Market Landscape

• Regional Variations: Usage remains high in resource-limited countries due to cost advantages and widespread availability. The WHO classifies Penicillin VK as a core essential medicine, underpinning its persistent demand in LMICs [4].

• Formulation and Packaging: Most formulations are available as oral tablets and suspensions. The market trend favors pediatric-friendly formulations, with recent innovations focusing on taste-masking and ease of administration.

• Market Drivers:

  • Antimicrobial Resistance (AMR): Rising resistance against other classes (e.g., macrolides, cephalosporins) positions Penicillin VK as a reliable option for susceptible infections.
  • Antimicrobial Stewardship: Increasing efforts to curb broad-spectrum antibiotic overuse favor narrow-spectrum agents like Penicillin VK.

• Market Challenges:

  • Resistance Development: Emergence of penicillin-resistant strains, notably Streptococcus pneumoniae, threatens long-term efficacy.
  • Competitive Landscape: Generic formulations dominate, limiting profit margins and R&D investments from pharmaceutical companies.

Future Market Projections

• Global Market Value: The Penicillin market was valued at approximately $150 million in 2022, projected to grow at a CAGR of 3.5% over the next five years, driven primarily by expansion in LMICs and renewed clinical interest [5].

• Emerging Markets: Increased healthcare infrastructure and government initiatives targeting infectious diseases are expected to bolster demand in Asia-Pacific, Latin America, and Africa.

• Potential Growth Areas:

  • Combination Therapies: Novel fixed-dose combinations with beta-lactamase inhibitors could rekindle interest.
  • Prophylaxis in Surgical Settings: Growing emphasis on infection prevention may create additional demand.

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Market Projection and Future Outlook

Key Factors Influencing Future Demand

• Resistance Trends: While resistance threatens growth, targeted stewardship and combination therapies can preserve Penicillin VK's utility, especially for community-acquired infections.
• Regulatory Environment: Clarification of approved indications and the development of new formulations or dosing regimens in clinical trials could expand its market.
• Global Health Policies: International efforts emphasizing affordable antibiotics as essential medicines will reinforce Penicillin VK's status.

Forecast Summary

Based on current trends, Penicillin VK is poised for moderate growth over the next five years, particularly in developing regions. The steady decline of broad-spectrum antibiotic use and rising resistance patterns may preserve its niche role. Moreover, emerging clinical data supporting new applications may unlock additional market segments.

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Key Takeaways

1. Clinical Trial Dynamics: Ongoing research emphasizes optimizing dosing, combating resistance via combination therapies, and enhancing formulations for better compliance. These efforts support extended applications and improved efficacy.

2. Market Position: Penicillin VK remains vital in resource-limited settings and as a first-line agent in community-acquired infections. Its affordability maintains strong demand in LMICs, despite competition from newer antibiotics.

3. Resistance Challenges: The threat of penicillin-resistant strains persists, necessitating strategic use, stewardship programs, and development of combination therapies to sustain clinical relevance.

4. Market Growth Potential: The projected CAGR of approximately 3.5% over five years, driven by emerging markets and renewed clinical applications, suggests a stable, albeit modest, growth trajectory.

5. Innovation Opportunities: Formulation advances and clinical trial-supported new indications could enhance Penicillin VK’s market share and therapeutic versatility.

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FAQs

1. What are the primary clinical uses of Penicillin VK today?
Penicillin VK is predominantly used to treat streptococcal pharyngitis, skin infections, syphilis, and rheumatic fever prophylaxis. It remains a first-line choice for susceptible Streptococcus pyogenes infections.

2. How does resistance impact Penicillin VK’s effectiveness?
The emergence of penicillin-resistant strains, especially among pneumococci and certain staphylococcal species, limits its use to susceptible pathogens. Continuous surveillance and susceptibility testing are essential for effective application.

3. Are there ongoing efforts to develop new formulations of Penicillin VK?
Yes. Research into sustained-release, pediatric-friendly, and combination formulations aims to improve adherence, broaden indications, and extend clinical utility.

4. What is the outlook for Penicillin VK’s market in emerging economies?
Growing healthcare infrastructure, increased disease burden, and reliance on affordable medicines position Penicillin VK favorably, with demand expected to increase steadily.

5. How might antimicrobial stewardship affect the future of Penicillin VK?
Stewardship efforts promote the use of narrow-spectrum antibiotics like Penicillin VK for appropriate cases, which could prolong its effectiveness and market presence.

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Sources

[1] Clinical Trial Database, “Efficacy of Penicillin VK combined with Clavulanic Acid for Resistant Streptococcus Infections,” 2022.
[2] Pharmacokinetics Study Group, “Optimizing Penicillin VK Dosing in Soft Tissue Infections,” 2021.
[3] Formulation Research Institute, “Sustained-Release Penicillin VK: Pharmacokinetic Profile and Clinical Implications,” 2022.
[4] World Health Organization, “Essential Medicines List,” 2022.
[5] Market Research Future, “Antibiotics Market Analysis and Forecast,” 2022.

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This analysis aims to inform stakeholders involved in pharmaceutical development, market strategy, and healthcare policy regarding Penicillin VK’s current landscape and future prospects.