CLINICAL TRIALS PROFILE FOR CYCLOSPORINE

505(b)(2) Clinical Trials for CYCLOSPORINE

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 Combination	NCT00373815 ↗	Everolimus in Combination With Cyclosporine A and Prednisolone for the Treatment of Graft Versus Host Disease	Terminated	University Hospital Tuebingen	Phase 1	2006-09-01	The present protocol is a dose-finding and toxicity study in preparation of a randomised study comparing current standard treatment CSA/prednisolone with the new combination CSA/prednisolone/everolimus.
New Formulation	NCT02961608 ↗	Conversion Pharmacodynamic Study in Stable Renal Transplant Patients Receiving Tacrolimus Two Times a Day to a New Formulation of Tacrolimus - LCP Tacro - 1 Time a Day.	Completed	Hospital Universitari de Bellvitge	Phase 4	2016-05-01	LCP-Tacro is an extended-release formulation of tacrolimus designed for once-daily dosing. Phase 1 studies demonstrated greater bioavailability than twice-daily tacrolimus capsules and no new safety concerns. - Stable kidney transplant patients can be safely converted from Adoport® twice-daily to LCP-Tacro®. - The greater bioavailability of LCP-Tacro after once-daily dosing results in similar (AUC) exposure, at a dose approximately 30% less, than the total daily dose of Adoport®. - LCP-Tacro provides a slow drug release and this results in flatter kinetics characterized by significantly lower peak-trough fluctuations. - CN is the major cellular target of the calcineurin inhibitors (CNIs) cyclosporine A (CsA) and tacrolimus. The ability of these drugs to inhibit CN activity is dependent on their binding to the respective immunophilins, cyclophilins A and B for CsA and FKBP12 for tacrolimus. - CN inhibition is a rate limiting phenomenon. Over concentrations of tacrolimus does not correlate with an increase in the CN activity.
OTC	NCT04515329 ↗	Tear Film Markers in Dry Eye Syndrome	Not yet recruiting	Sun Pharma Global FZE	Phase 4	2021-12-01	Dry eye is the most common reason for visit to an ophthalmologist's office. The prevalence is on the rise and is mainly attributed to factors such as increased environmental pollution and contact lens use. The current management options are limited to over the counter artificial tear drops and three FDA-approved drugs. Of these, cyclosporine has been used worldwide for treating mild to moderate dry eyes. The earlier version consisted of 0.05% cyclosporine which worked well for a limited number of inflammatory dry eye conditions. Recently, 0.09% cyclosporine was approved by the FDA. The nearly double concentration is expected to be more beneficial for severe inflammation which is often seen in Sjögren syndrome and other Rheumatological conditions associated with dry eyes. In this pilot project, the investigator proposes to evaluate the change in expression of SLURP1 and other markers of ocular surface inflammation before and after treatment with 0.09% cyclosporine eye drops.
OTC	NCT04515329 ↗	Tear Film Markers in Dry Eye Syndrome	Not yet recruiting	Vishal Jhanji	Phase 4	2021-12-01	Dry eye is the most common reason for visit to an ophthalmologist's office. The prevalence is on the rise and is mainly attributed to factors such as increased environmental pollution and contact lens use. The current management options are limited to over the counter artificial tear drops and three FDA-approved drugs. Of these, cyclosporine has been used worldwide for treating mild to moderate dry eyes. The earlier version consisted of 0.05% cyclosporine which worked well for a limited number of inflammatory dry eye conditions. Recently, 0.09% cyclosporine was approved by the FDA. The nearly double concentration is expected to be more beneficial for severe inflammation which is often seen in Sjögren syndrome and other Rheumatological conditions associated with dry eyes. In this pilot project, the investigator proposes to evaluate the change in expression of SLURP1 and other markers of ocular surface inflammation before and after treatment with 0.09% cyclosporine eye drops.
New Formulation	NCT07033858 ↗	Evaluaion the Short Term Effects of Advograf Plus Rapamiune After Kidney Transplantation	RECRUITING	Shahid Beheshti University of Medical Sciences	NA	2025-03-01	Cornerstone immunosuppressive therapy currently relies on immediate-release tacrolimus, a calcineurin inhibitor (CNI) that is potentially nephrotoxic and is more diabetogenic than cyclosporine A. A new formulation of tacrolimus has been launched: an extended-release formulation (Advagraf/Astagraf XL, Astellas company).

All Clinical Trials for CYCLOSPORINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000524 ↗	Myocarditis Treatment Trial	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	1986-07-01	To determine whether immunosuppressive treatment improved cardiac function in patients with biopsy-proven myocarditis.
NCT00000524 ↗	Myocarditis Treatment Trial	Completed	University of Utah	Phase 2	1986-07-01	To determine whether immunosuppressive treatment improved cardiac function in patients with biopsy-proven myocarditis.
NCT00000880 ↗	A Study to Test the Effect of Cyclosporine on the Immune System of Patients With Early HIV Disease	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	The purpose of this study is to determine the safety and effectiveness of low doses of cyclosporine (CsA) in patients with early HIV infection and to evaluate its effect on the immune system. Activation of T cells (cells of the immune system) leads to HIV replication. Inhibition of immune activation is therefore a potentially important area of therapy for patients with early HIV infection. CsA is capable of decreasing T cell activation, which in turn may decrease HIV replication.
NCT00000936 ↗	A Study To Test An Anti-Rejection Therapy After Kidney Transplantation	Terminated	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1999-11-01	Kidney transplantation is often successful. However, despite aggressive anti-rejection drug therapy, some patients will reject their new kidney. This study is designed to test two anti-rejection approaches. Two medications in this study are currently used in children, but there is no information regarding which drug is safer or more effective. Survival rates in renal transplantation are unacceptably low. Therefore, there is a need for an improved post-transplant treatment, such as the induction therapy used in this study.
NCT00001302 ↗	A Phase I Study of Infusional Chemotherapy With the P-Glycoprotein Antagonist PSC 833	Completed	National Cancer Institute (NCI)	Phase 1	1992-09-01	The clinical study entitled "A Phase I Study of Infusional Chemotherapy with the P-glycoprotein Antagonist PSC 833" seeks to determine the maximum tolerated dose for a proposed P-glycoprotein antagonist, PSC 833. PSC 833 is a cyclosporine analogue which is purportedly non-nephrotoxic and non-immunosuppressive. It has been shown in in-vitro studies to enhance chemosensitivity as well as cyclosporine and to be far better at increasing intracellular drug accumulation than the concentrations of verapamil which are clinically achievable. The purpose of this study is to define the maximum tolerated dose in combination with vinblastine, and to determine how the drug affects the pharmacokinetics of vinblastine. PSC 833 will most likely reduce the clearance of vinblastine, as reported for the parent compound, cyclosporine. This effect will increase the area under the curve (AUC) of vinblastine, may increase toxicity, and requires that the escalation scheme for PSC 833 be a conservative one. Initially, a 120 hour infusion of vinblastine will be given alone. Then 8 days of PSC 833 will follow to allow monitoring of adverse effects of PSC 833 alone. This first cycle of vinblastine will be given in the absence of PSC 833; in second and subsequent cycles both agents will be combined. Escalation of the PSC 833 will continue until a target concentration is reached, or until the maximum tolerated dose is reached. Clinical responses will be monitored in order to provide the best possible medical care to our patients.
NCT00001383 ↗	A Phase I Study of Infusional Paclitaxel With the P-Glycoprotein Antagonist PSC 833	Completed	National Cancer Institute (NCI)	Phase 1	1994-03-01	This is a dosage escalation study to estimate the maximum tolerated dose of drug resistance inhibitor PSC 833 given in combination with paclitaxel. Groups of 3 to 6 patients receive continuous-infusion paclitaxel for 5 days and oral PSC 833 for 6-7 days, following paclitaxel on the first course, then beginning 3 days prior to paclitaxel on subsequent courses. Stable and responding patients are re-treated every 21 days, with paclitaxel dose adjusted to maintain an absolute neutrophil count less than 500 for no more than 4 days.

Clinical Trial Conditions for CYCLOSPORINE

Condition Name

Condition Name for CYCLOSPORINE
Intervention	Trials
Leukemia	108
Myelodysplastic Syndromes	74
Lymphoma	68
Kidney Transplantation	60

Condition MeSH

Condition MeSH for CYCLOSPORINE
Intervention	Trials
Leukemia	175
Syndrome	144
Myelodysplastic Syndromes	143
Preleukemia	138

Clinical Trial Locations for CYCLOSPORINE

Trials by Country

Trials by Country for CYCLOSPORINE
Location	Trials
Canada	163
Italy	143
China	120
Germany	94
France	82

Trials by US State

Trials by US State for CYCLOSPORINE
Location	Trials
California	138
Washington	115
Maryland	100
New York	97
Texas	89

Clinical Trial Progress for CYCLOSPORINE

Clinical Trial Phase

Clinical Trial Phase for CYCLOSPORINE
Clinical Trial Phase	Trials
PHASE4	16
PHASE3	4
PHASE2	19

Clinical Trial Status

Clinical Trial Status for CYCLOSPORINE
Clinical Trial Phase	Trials
Completed	649
Recruiting	125
Unknown status	102

Clinical Trial Sponsors for CYCLOSPORINE

Sponsor Name

Sponsor Name for CYCLOSPORINE
Sponsor	Trials
National Cancer Institute (NCI)	175
Fred Hutchinson Cancer Research Center	82
National Heart, Lung, and Blood Institute (NHLBI)	63

Sponsor Type

Sponsor Type for CYCLOSPORINE
Sponsor	Trials
Other	1186
Industry	418
NIH	293

CycloSPORINE Clinical Trials Update, Market Analysis, and Launch-Projections (2026)

Last updated: July 25, 2026

Cyclosporine remains a high-value immunosuppressant with ongoing lifecycle work focused on chronic use, transplant maintenance, and formulation differentiation. Market growth is constrained by long-established generics in most mature geographies, but demand persists from transplant volumes, autoimmune-treatment cohorts, and new patient starts where branded formulations maintain pricing and adherence advantages.

What is the latest clinical trial landscape for cyclosporine?

Answer: Clinical activity is concentrated in topical ophthalmology and select systemic indications with long-term endpoints (graft survival, renal function preservation proxies, ocular surface disease control) plus formulation-bioavailability work intended to support differentiation in markets where generics dominate systemic cyclosporine.

Which cyclosporine formulations are most represented in trials?

The trial mix is typically split across:

• Ophthalmic cyclosporine (notably emulsion and solution formats) for chronic dry eye and related ocular inflammatory conditions.
• Oral cyclosporine (capsules/modified-release variants) for transplant maintenance and steroid-sparing regimens.
• Topical dermatologic cyclosporine where applicable for inflammatory skin disease cohorts in specific regions.
• New delivery and exposure-optimization efforts (controlled release, microemulsion technologies) that target adherence and steady-state exposure consistency.

What endpoints dominate cyclosporine studies?

• Transplant maintenance: kidney function metrics (eGFR/serum creatinine), acute rejection rates, graft survival time-to-event.
• Ocular: corneal staining, tear production, symptom scores, and responder analyses at fixed timepoints (weeks to months).
• Safety: nephrotoxicity proxies, hypertension incidence, infection signals, and laboratory monitoring burdens.

How do recent trial patterns affect development strategy?

For any entrant, the most defensible clinical path usually requires:

• A differentiated patient population (subset response, earlier disease stage, steroid-sparing endpoints).
• A formulation or exposure profile advantage with clinically meaningful endpoints.
• An evidence package aligned to label-expansion or replacement of a competitor’s regimen rather than duplicate indication chasing in saturated segments.

Which indications have the highest remaining growth leverage for cyclosporine?

Answer: Ophthalmic cyclosporine in chronic ocular inflammation has the clearest commercial leverage versus systemic categories where branded pricing power is more limited by generic penetration.

Ocular surface disease and dry eye

• Chronic inflammatory drivers create sustained demand, and cyclosporine remains a cornerstone option in multiple treatment algorithms.
• Trials in this area often focus on durability and responder rate improvements, which can support persistence and incremental market share even without large headroom in overall prevalence.

Transplant maintenance

• Demand is stable and volume-linked: transplant schedules do not reset quickly, which supports baseline utilization.
• Competitive pressure is high because cyclosporine is a mature active ingredient with multiple approved products and long generic history.

Autoimmune and dermatology (region-dependent)

• Growth is more tied to local guideline positions, reimbursement, and tolerability than to global prevalence increases.
• Trial selection tends to emphasize treatment tolerability and reduced need for corticosteroids.

How does the competitive landscape look for cyclosporine by product segment?

Answer: Competition is structurally different by route: systemic cyclosporine faces broad generic substitution; ocular cyclosporine has more room for branded differentiation through formulation and patient experience, even where generics exist.

Systemic cyclosporine: branded vs generic economics

• Branded systemic products typically lose incremental share where interchangeability is high and payer formularies prefer low-cost options.
• Modified-release and exposure-stable claims can preserve some pricing and restrict substitution, but the active ingredient is long commoditized.

Ophthalmic cyclosporine: persistent brand differentiation

• Ocular dosing regimens (twice daily vs alternative schedules), emulsion stability, and tolerability profile drive formulary and retention decisions.
• Clinical differentiation often tracks into label language that payers accept as medical-need rationale.

What is the cyclosporine market size outlook and growth drivers to 2030?

Answer: The market outlook is “steady-state with pockets of growth.” Total demand is supported by transplant volumes and chronic ocular inflammation, while growth rates are moderated by generic competition in systemic segments.

Key drivers

• Transplant volumes: stable and linked to healthcare capacity and eligibility.
• Ocular inflammatory disease burden: chronicity drives recurring prescriptions and refill persistence.
• Treatment algorithms: steroid-sparing strategies and guideline inclusion maintain cyclical demand.

Key constraints

• Generic substitution: limits branded price erosion risk but also compresses total revenue growth in systemic lines.
• Safety monitoring friction: nephrotoxicity and hypertension monitoring reduce physician and patient willingness to switch without clear benefit.
• Therapeutic competition: other immunomodulators and anti-inflammatory agents compete for ocular and autoimmune share.

What clinical evidence would most impact cyclosporine market share next?

Answer: Evidence that improves durable response or reduces monitoring and adverse events has the highest chance of shifting payer behavior and prescribing habits.

Evidence types that move commercial outcomes

• Rescue strategy superiority: improved outcomes for “inadequate response” patients.
• Tolerability improvements: fewer discontinuations or reduced lab monitoring intensity with validated exposure stability.
• Adherence and persistence: fewer missed doses via better dosing schedules or less sting in ophthalmic formulations.
• Switch studies: data showing acceptable transition from other cyclosporine products or comparators.

What launch projections are realistic for a new cyclosporine entrant?

Answer: A new entrant’s probability-weighted trajectory is strongest when it targets ophthalmic or a formulation-adjacent claim with clear persistence advantages. In systemic transplant, adoption typically requires either a differentiated regulatory route, a payer-supported niche, or a total cost of therapy argument.

Probability-weighted adoption model (framework)

• Year 0–1: limited uptake driven by formulary conversions, starter packs, and clinician trial usage.
• Year 2–3: share gain if switching outcomes are positive and adverse events are manageable within routine monitoring.
• Year 4+: stabilization depends on price, interchangeability restrictions, and persistence among responders.

What “success” looks like in ophthalmic cyclosporine

• Improved responder rates at clinically meaningful timepoints.
• Demonstrated persistence without increased discontinuations from tolerability issues.
• Payer acceptance of evidence-backed outcomes tied to chronic disease management.

What “success” looks like in systemic transplant

• Comparable rejection outcomes with improved renal function proxies.
• Clear clinical positioning for steroid minimization or specific maintenance subgroups.
• Retention depends on physician confidence in exposure stability and lab monitoring workflow.

How do clinical trial timelines typically shape cyclosporine regulatory and market timing?

Answer: For chronic indications, the clinical package must support durable endpoints. That drives longer readouts and can delay commercialization even if enrollment is efficient.

Typical sequencing

• Phase 3 or late Phase 2: chronic outcomes can require months to a year.
• Label language expansion: depends on endpoint hierarchy and regulatory interpretation.
• Commercial ramp: starts only after final approval and supply chain readiness.

Market timing impact

• For ophthalmic products, commercial ramp can start faster if the evidence package supports earlier endpoint readouts.
• For systemic transplant, endpoint timing and safety follow-up often lengthen approval timelines.

What regulatory and IP factors can constrain cyclosporine market entry?

Answer: Cyclosporine’s active ingredient is mature, and competitive entry is primarily shaped by brand lifecycle IP (formulations, methods-of-use) and regulatory exclusivity periods on specific products, not by the base molecule.

Practical implications for developers

• Brand longevity can be protected by formulation-specific patents and labeling differentiation.
• Generic and authorized generic competition can emerge quickly once exclusivity and patent barriers clear for the specific product presentation.

How does cyclosporine compare with competing immunosuppressants in the same space?

Answer: Cyclosporine competes on long-term tolerability experience and algorithm fit, while newer agents compete on convenience and reduced monitoring burden in specific niches.

Ocular immunomodulator competition

• Competes with other chronic dry eye inflammatory treatments where onset and tolerability drive patient preferences.
• Cyclosporine retains strength in chronic anti-inflammatory frameworks and established prescriber familiarity.

Transplant immunosuppression competition

• Competes with other calcineurin inhibitors and steroid-sparing regimens.
• Choice is shaped by patient-specific risk profiles, prior tolerability, and center protocols.

Key commercial scenarios for cyclosporine (base, bull, bear)

Answer: Systemic revenue is vulnerable to generic pricing pressure; ophthalmic segment performance depends on persistence and payer acceptance of formulation differentiation.

Base case

• Stable demand, modest value growth limited by generic penetration in systemic segments.
• Ophthalmic growth continues as a contribution offset.

Bull case

• A differentiated ophthalmic formulation captures share through superior durability and tolerability.
• Better-than-expected persistence reduces discontinuations and increases refill rates.

Bear case

• Aggressive substitution where generics or alternative therapies expand within payer formularies.
• Lower durability or higher discontinuation rates cap share gains for differentiated entrants.

Where are cyclosporine revenues most exposed?

Answer: Revenue exposure concentrates in branded systemic formulations and any product presentations where generics are available at scale. Ophthalmic presentations show comparatively better resilience due to perceived patient experience and chronic dosing persistence dynamics.

Exposure map by segment

• Systemic: highest exposure to price compression and rapid substitution.
• Ophthalmic: moderate exposure tied to formulation differentiation, persistence, and label scope.
• Other topical/autoimmune niches: regionally variable exposure tied to guideline inclusion and reimbursement.

What are the most important due diligence items for cyclosporine investment or licensing?

Answer: Focus on product presentation, jurisdiction-specific exclusivity/IP status, evidence strength for label scope, and persistence metrics in the target segment.

Due diligence checklist

• Label scope and responder definitions tied to endpoints used in payer coverage.
• Switching and adherence evidence.
• Pricing power assumptions adjusted for generic availability.
• Supply and manufacturing readiness for chronic dispensing requirements.
• Litigation and regulatory history tied to the specific presentation.

Key Takeaways

• Cyclosporine’s growth outlook is steady-state, supported by chronic indications and transplant maintenance demand.
• Market share gains for a new entrant are most realistic in ophthalmic formulations with evidence of durability and tolerability that translate into persistence.
• Systemic cyclosporine is structurally exposed to generic substitution, limiting branded value growth unless a presentation has defensible differentiation.
• Commercial outcomes hinge on clinical endpoints that payers and clinicians act on: durability of effect, discontinuation rates, and practical safety management.

FAQs

1. What is driving cyclosporine demand in dry eye over the next 3 years?
2. How quickly can generics displace branded systemic cyclosporine after exclusivity ends?
3. What clinical endpoints best predict long-term payer adoption for ophthalmic cyclosporine?
4. Which cyclosporine formulation attributes most affect adherence and persistence?
5. How do transplant center protocols influence cyclosporine switching and patient outcomes?

References

No cited sources were provided in the prompt.