CLINICAL TRIALS PROFILE FOR CYSTEAMINE BITARTRATE

All Clinical Trials for cysteamine bitartrate

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00872729 ↗	Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis	Completed	Horizon Pharma USA, Inc.	Phase 1/Phase 2	2009-05-01	Cystinosis is an inheritable disease that if untreated, results in kidney failure as early as the first decade of life. The current marketed therapy is Cystagon® (cysteamine bitartrate) which must be taken every six hours for the rest of the patient's life to prevent complications of cystinosis. RP103 is a formulation of cysteamine bitartrate that is being studied to see if it may be able to be given less frequently, once every 12 hours, and have similar results.
NCT00872729 ↗	Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis	Completed	Raptor Pharmaceuticals Inc.	Phase 1/Phase 2	2009-05-01	Cystinosis is an inheritable disease that if untreated, results in kidney failure as early as the first decade of life. The current marketed therapy is Cystagon® (cysteamine bitartrate) which must be taken every six hours for the rest of the patient's life to prevent complications of cystinosis. RP103 is a formulation of cysteamine bitartrate that is being studied to see if it may be able to be given less frequently, once every 12 hours, and have similar results.
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.
NCT00715559 ↗	Cysteamine Therapy for Major Depressive Disorder	Terminated	Murrough, James, M.D.	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.
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.

Clinical Trial Conditions for cysteamine bitartrate

Condition Name

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

Condition MeSH

Condition MeSH for cysteamine bitartrate
Intervention	Trials
Cystinosis	8
Fanconi Syndrome	3
Mitochondrial Diseases	2
Syndrome	1

Clinical Trial Locations for cysteamine bitartrate

Trials by Country

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

Trials by US State

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−

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, Market Analysis, and Projections

Introduction to Cysteamine Bitartrate

Cysteamine bitartrate is a crucial medication in the treatment of nephropathic cystinosis, a rare genetic disorder characterized by the accumulation of cystine within cells, leading to organ damage. Here, we will delve into the current status of clinical trials, market analysis, and future projections for this drug.

Clinical Trials Update

Ongoing and Completed Trials

One of the significant clinical trials involving cysteamine bitartrate is the Phase III study comparing the safety and efficacy of cysteamine bitartrate delayed-release capsules (RP103) to the immediate-release formulation (Cystagon)[1][2].

• RP103-03 Trial: This trial has shown that RP103, the delayed-release formulation, is noninferior to Cystagon in terms of efficacy. The trial demonstrated that RP103 can maintain white blood cell (WBC) cystine levels below 1 nmol/½ cystine/mg protein during the 12-hour dosing interval, similar to Cystagon. However, the delayed-release formulation offers the advantage of a simplified dosing regimen, reducing the frequency from four times daily to twice daily[2][3].

• Adverse Events: While RP103 was found to be safe and well-tolerated, there was a higher proportion of serious and non-serious adverse events in the delayed-release group compared to the immediate-release group[3].

• Dosing and Formulation: RP103 is formulated in microspheronized beads that are enteric-coated, allowing patients to sprinkle the contents on soft food or liquid if they have difficulty swallowing capsules. The dose is highly individualized and is titrated based on WBC cystine response[2].

Other Clinical Investigations

In addition to its use in nephropathic cystinosis, cysteamine bitartrate is being explored in other conditions. For instance, an ongoing Phase II/III clinical trial is investigating its safety and efficacy in Huntington's disease (HD). Preliminary results indicate that RP103 is safe and well-tolerated in HD patients and has shown a small but significant slowing of disease progression in certain patient groups[4].

Market Analysis

Current Market Size and Trends

The global market for cystinosis treatments, primarily driven by cysteamine bitartrate formulations like Procysbi (RP103) and Cystagon, was valued at USD 130.46 million in 2017 across seven major markets. The United States accounted for the largest share, with a market size of USD 86.52 million in 2017[5].

• Market Drivers: The main drivers of the market include the increasing prevalence of diagnosed cystinosis cases and the need for effective treatments. Procysbi and Cystagon are the dominant players, with other therapies such as Cystaran and Cystadrops also contributing to the market size[5].

• Emerging Therapies: The market is expected to grow due to the potential of new therapies such as AVR-RD-04, ELX-02, and A0003, which are in various stages of development[5].

Economic Impact and Cost-Effectiveness

The pharmacoeconomic analysis of Procysbi (RP103) indicates that it could result in significant health benefits, including an additional 12.98 quality-adjusted life-years (QALYs) and an incremental life-year gain of 16.36 years over the lifetime of a patient. However, this comes at a substantial incremental cost, estimated at $8,770,005, resulting in an incremental cost-utility ratio of $675,605 per QALY compared to no treatment[3].

Future Projections

Market Growth

The cystinosis market is expected to grow at a significant compound annual growth rate (CAGR) from 2017 to 2028. This growth is driven by increasing awareness, better diagnostic tools, and the introduction of new therapies. The market size is projected to increase due to improved treatment adherence and the potential benefits of delayed-release cysteamine formulations[5].

Therapeutic Advancements

The development of new formulations and the exploration of cysteamine bitartrate in other diseases, such as Huntington's disease, are expected to expand the therapeutic landscape. These advancements could lead to better patient outcomes and increased market demand.

Regulatory and Access Considerations

Regulatory approvals and access to these medications will play a critical role in their adoption. The approval of RP103 in various regions and its inclusion in healthcare reimbursement programs will be crucial for its market success.

Key Takeaways

• Clinical Trials: Cysteamine bitartrate delayed-release capsules (RP103) have shown noninferior efficacy to immediate-release formulations and offer a simplified dosing regimen.
• Market Size: The global market for cystinosis treatments was valued at USD 130.46 million in 2017, with the United States being the largest market.
• Economic Impact: RP103 could provide significant health benefits but at a substantial incremental cost.
• Future Growth: The market is expected to grow due to increasing awareness, better diagnostics, and the introduction of new therapies.
• Therapeutic Expansions: Cysteamine bitartrate is being explored in other conditions, such as Huntington's disease, which could expand its therapeutic use.

FAQs

What is the primary indication for cysteamine bitartrate?

Cysteamine bitartrate is primarily used in the treatment of nephropathic cystinosis, a rare genetic disorder.

How does the delayed-release formulation of cysteamine bitartrate differ from the immediate-release formulation?

The delayed-release formulation (RP103) offers a simplified dosing regimen, reducing the frequency from four times daily to twice daily, and allows for the contents to be sprinkled on food or liquid if swallowing capsules is difficult.

What are the potential health benefits of using delayed-release cysteamine bitartrate?

Delayed-release cysteamine bitartrate could result in an additional 12.98 QALYs and an incremental life-year gain of 16.36 years over the lifetime of a patient.

Is cysteamine bitartrate being explored for other conditions?

Yes, cysteamine bitartrate is being investigated in clinical trials for its potential use in Huntington's disease.

What are the economic implications of using delayed-release cysteamine bitartrate?

The use of delayed-release cysteamine bitartrate comes at a substantial incremental cost, estimated at $8,770,005, resulting in an incremental cost-utility ratio of $675,605 per QALY compared to no treatment.

Sources

1. Orphanet: Research and trials - Orphanet.
2. FDA: 203389Orig1s000 - accessdata.fda.gov.
3. CADTH: CDR Pharmacoeconomic Review Report for Procysbi.
4. Huntington Study Group: Meet the Compound: RP103 (cysteamine bitartrate delayed-release).
5. GlobeNewswire: Global Cystinosis Market Study 2017-2019 & 2020-2028.