CLINICAL TRIALS PROFILE FOR CYSTEAMINE BITARTRATE

All Clinical Trials for cysteamine bitartrate

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00028262 ↗	Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 4	2001-02-01	This study will examine the effectiveness of a drug called Cystagon in treating infantile neuronal ceroid lipofuscinosis (INCL), a progressive neurological disease affecting children. At around 11 to 13 months of age, patients develop slowed head growth, mild brain atrophy (wasting), electroencephalographic (EEG) changes and retinal deterioration, with symptoms worsening over time. The disease results from an enzyme deficiency that causes fatty compounds called ceroid to accumulate in cells. In laboratory experiments, Cystagon has helped remove ceroid from cells of patients with INCL. Children with INCL between 6 months and 3 years of age may be eligible for this study. Participants take Cystagon daily by mouth every 6 hours. They are admitted to the NIH Clinical Center for a 4- to 5-day period every 6 months for the following tests and evaluations: - Review of medical history, including a detailed record of seizures, physical examination, blood tests and clinical photographs. For the initial baseline studies, examinations may also be scheduled with pediatric neurology, ophthalmology and anesthesia services. - Magnetic resonance imaging (MRI) of the brain MRI uses a powerful magnet, radio waves, and computers to provide detailed images of the brain without the use of X-rays. The patient lies on a table that slides inside a donut-shaped machine containing a magnetic field. The child requires general anesthesia for the procedure. - Electroretinogram (ERG) measures the function of the retina, the light-sensitive tissue in the back of the eye. To record the flash ERG, a special contact lens is placed on the eye s surface and the eye is stimulated with flashes of light. Infants and very young children require general anesthesia for the procedure. - Visual evoked potential (VEP) measures the function of the visual pathway from the eye to the brain. To record the VEP, five electrodes are placed on the scalp and the eye is stimulated with flashes of light. Infants and very young children must be anesthetized for the procedure. - Electroencephalogram (EEG) measures brain electrical activity, using electrodes placed on the scalp. The test is useful in defining seizures. The child may need to be sedated to keep still during the test. - Skin biopsy A small piece of skin is removed (usually from the upper arm or shoulder) under local anesthetic to grow cells in the laboratory. This procedure is done at the start of the study and is repeated after 1 year if therapy results are promising. Children s condition may improve, stabilize or worsen during this study. Life may be prolonged without significant improvement in quality. The information gained from the study may help scientists develop more potent drugs to treat INCL.
NCT00359684 ↗	Use of Cysteamine in the Treatment of Cystinosis	Recruiting	National Human Genome Research Institute (NHGRI)	Phase 4	1978-07-01	Cystinosis is an inherited disease resulting in poor growth and kidney failure. There is no known cure for cystinosis, although kidney transplantation may help the renal failure and prolong survival. Both the kidney damage and growth failure are thought to be due to the accumulation of the amino acid cystine within the cells of the body. The cystine storage later damages other organs besides the kidneys, including the thyroid gland, pancreas, eyes, and muscle. The drug cysteamine (Cystagon) is an oral medication given to patients with cystinosis prior to kidney transplantation. The drug works by reducing the level of cystine in the white blood cells and muscle tissue. The drug may also decrease levels of cystine in the kidneys and other tissues. This study has several goals: 1. Long-term surveillance of cysteamine (Cystagon) treated patients. 2. Detection of new non-kidney complications of cystinosis. 3. Maintenance of a patient population for genetic testing (mutational analysis) of the cystinosis gene.
NCT00715559 ↗	Cysteamine Therapy for Major Depressive Disorder	Terminated	Icahn School of Medicine at Mount Sinai	N/A	2008-07-01	The purpose of this study is to determine whether cysteamine bitartrate, an FDA-approved drug for a non-psychiatric condition, is safe and effective for the treatment of major depression.

Clinical Trial Conditions for cysteamine bitartrate

Condition Name

Condition Name for cysteamine bitartrate
Intervention	Trials
Cystinosis	8
Nonalcoholic Fatty Liver Disease (NAFLD)	1
Schizoaffective	1

Condition MeSH

Condition MeSH for cysteamine bitartrate
Intervention	Trials
Cystinosis	8
Fanconi Syndrome	3
Mitochondrial Diseases	2

Clinical Trial Locations for cysteamine bitartrate

Trials by Country

Trials by Country for cysteamine bitartrate
Location	Trials
United States	36
Netherlands	3
France	3
United Kingdom	1
Italy	1

Trials by US State

Trials by US State for cysteamine bitartrate
Location	Trials
California	8
Texas	5
Illinois	5
Georgia	5
Ohio	4

Clinical Trial Progress for cysteamine bitartrate

Clinical Trial Phase

Clinical Trial Phase for cysteamine bitartrate
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	4
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for cysteamine bitartrate
Clinical Trial Phase	Trials
Completed	10
Terminated	3
Recruiting	2

Clinical Trial Sponsors for cysteamine bitartrate

Sponsor Name

Sponsor Name for cysteamine bitartrate
Sponsor	Trials
Horizon Pharma USA, Inc.	7
Raptor Pharmaceuticals Inc.	7
Icahn School of Medicine at Mount Sinai	2

Sponsor Type

Sponsor Type for cysteamine bitartrate
Sponsor	Trials
Industry	16
Other	7
NIH	5

Cysteamine Bitartrate: Clinical Trials Update, Market Analysis, and Projection

Last updated: April 25, 2026

What is cysteamine bitartrate and what is its current clinical position?

Cysteamine bitartrate is a cystine-depleting agent used to treat cystinosis. The drug’s clinical and commercial footprint is tied to the disease-modifying premise: intracellular cystine reduction to slow multi-organ toxicity.

Indications with established clinical use

• Nephropathic (juvenile) cystinosis: Disease-modifying therapy to reduce cystine accumulation in tissues.
• Ocular cystinosis: Treatment supported by the pharmacology-driven rationale that cystine accumulates in ocular tissues and contributes to corneal disease burden.

Clinical-development intensity for cysteamine bitartrate itself is not characterized by broad, late-stage randomized “new entrant” programs typical of novel drugs. Instead, the industry focus has historically concentrated on:

• Formulation improvements (dose delivery and adherence)
• Translational optimization (pediatric dosing regimens, monitoring targets)
• Comparative performance versus alternatives (including other cysteamine formulations)

What are the latest clinical-trials signals for cysteamine bitartrate?

No complete and definitive, current “trial-by-trial” update can be produced from the information provided. A true clinical-trials update requires a current registry pull (e.g., ClinicalTrials.gov) with trial statuses, dates, phases, endpoints, and results. That is not supplied in the prompt.

Given the constraint, no trial table, phase-level update, or date-stamped activity log is provided.

How large is the cysteamine bitartrate market and how is it evolving?

Cysteamine bitartrate market size and growth depend on:

• Prevalence of cystinosis (juvenile nephropathic and ocular burden)
• Diagnosis rates and early initiation of therapy
• Adherence and persistence (lifelong treatment in a chronic condition)
• Conversion of patients across care settings (pediatrics, nephrology, ophthalmology)
• Competitive pressure from alternative cysteamine delivery systems (oral and topical variations)

No numerical market sizing, TAM/SAM/SOM breakdown, or segmentation is included in the prompt. Without cited demand estimates and adoption assumptions, a complete and accurate projection cannot be constructed to a standard suitable for investment or R&D decision-making.

Accordingly, no hard market-sizing figures, growth rates, or forecast curves are stated.

What drives demand at the patient and payer level?

For cystinosis, demand is driven by chronicity and treatment continuity rather than episodic usage.

Demand drivers

• Lifelong therapy behavior: Sustained dosing is required to manage cystine accumulation.
• Pediatric treatment: Treatment starts early, with ongoing monitoring in nephrology and ophthalmology.
• Disease awareness and diagnostic throughput: Earlier identification expands the addressable pool of treated patients.
• Clinical monitoring requirements: Treatment value is assessed through cystine measures and organ outcomes, supporting continued use when targets are met.

Demand dampeners

• Formulation and tolerability constraints: Cysteamine dosing can be burdensome; persistence depends on tolerability and regimen fit.
• Coverage dynamics: Specialty drug utilization can be constrained by prior authorization and regimen documentation requirements.
• Competition within class: Alternative cysteamine formulations can shift share even when the active drug class remains cysteamine.

What is the likely revenue trajectory, and what does it depend on?

A defensible projection needs:

• baseline treated patient counts
• utilization per patient (dose, dosing frequency, formulation split)
• pricing (net of rebates/discounts)
• geography mix and reimbursement changes
• share shift due to competitive formulations
• pipeline substitution risk

None of these numeric inputs are provided, and therefore no revenue curve is generated.

Competitive landscape: how cysteamine bitartrate typically competes

Cysteamine bitartrate competes on:

• Therapeutic parity (cystine depletion effect)
• Dosing practicality (oral regimen burden)
• Adherence outcomes in real-world use
• Ocular disease management fit depending on availability of topical options
• Contracted pricing and formulary placement

In practice, share is influenced by the availability of alternative cysteamine products with distinct dosing schedules and patient-specific tolerability profiles.

Key decision implications for R&D and investment

1) The differentiation battleground is formulation and access

For established disease-modifying drugs in rare genetic disorders, new entrants and incumbents win through:

• improved tolerability
• simpler dosing
• better adherence support
• reimbursement and channel access

2) Evidence strategy should anchor to measurable cystine and organ outcomes

Trial endpoints and post-marketing evidence are typically expected to connect:

• cystine reduction metrics
• organ function stabilization
• ocular surface outcomes
• patient-reported tolerability and persistence

3) Watch substitution risk from competing cysteamine formats

Even without new mechanism innovation, shifts can occur when alternatives deliver:

• fewer administration burdens
• better tolerability
• more convenient titration schedules
• stronger payer positioning

Key Takeaways

• Cysteamine bitartrate is an established cystinosis therapy with demand anchored to chronic, lifelong treatment patterns.
• A complete “clinical trials update” requires current registry data for accuracy; no trial-level update is stated here.
• A defensible market and revenue projection requires numeric inputs on treated prevalence, pricing, utilization, and share shifts; no quantified forecast is stated here.
• Commercial differentiation typically comes from formulation practicality and access rather than mechanism revalidation.

FAQs

1) What diseases does cysteamine bitartrate treat?

It is used for cystinosis, including nephropathic (juvenile) and ocular disease management, consistent with cystine-depleting pharmacology.

2) Is cysteamine bitartrate a one-time treatment or chronic therapy?

It is generally chronic (lifelong) therapy in patients with cystinosis to manage tissue cystine accumulation.

3) What endpoints matter most in cystinosis development?

Commonly used evidence anchors include cystine-related measures and organ function or disease burden outcomes, with ocular measures used for eye involvement.

4) What most influences commercial performance for this class?

Net performance is driven by patient persistence/adherence, coverage and pricing, and formulation convenience versus alternatives.

5) Does the market growth depend on new clinical trial breakthroughs?

In this therapeutic context, growth is more often linked to diagnosis rates, treatment initiation, persistence, and competitive substitution than to new mechanism approvals.

References

[1] ClinicalTrials.gov. (n.d.). Cysteamine bitartrate search results. https://clinicaltrials.gov/
[2] FDA label / prescribing information for cysteamine bitartrate (as available on FDA databases). (n.d.). https://www.accessdata.fda.gov/
[3] EMA product information for cysteamine bitartrate (as available). (n.d.). https://www.ema.europa.eu/