CLINICAL TRIALS PROFILE FOR CYSTEAMINE HYDROCHLORIDE
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505(b)(2) Clinical Trials for CYSTEAMINE HYDROCHLORIDE
| Trial Type | Trial ID | Title | Status | Sponsor | Phase | Start Date | Summary |
|---|---|---|---|---|---|---|---|
| New Formulation | NCT00001736 ↗ | New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis | Completed | National Eye Institute (NEI) | Phase 1 | 1998-05-01 | This study will evaluate the safety and effectiveness of a new formulation of eye drops used to treat cystine crystals that form in the corneas of patients with cystinosis. Cystinosis is an inherited disease caused by a defective enzyme, in which excessive amounts of the amino acid cystine accumulate in the body. Among others, symptoms include poor growth and development of kidney failure. In addition, after 10 to 20 years, the cornea-the outside covering of the eye over the iris and pupils-becomes so packed with cystine crystals that small, painful breaks may develop. This corneal condition is treated with cysteamine eye drops. This study is designed to provide additional information about this medication that the Food and Drug Administration requires before approving it for marketing. The study will examine, in two separate but simultaneous investigations, the safety and effectiveness of a new cysteamine formulation. In both studies, before treatment begins, patients will have a complete eye examination, and photographs of the eye will be taken using a bright flash. Safety Study Children and adults currently enrolled in a cystinosis study at the National Institutes of Health may participate in this trial. They will receive the current cysteamine formulation in one eye and the new preparation in the other eye. The drops will be given every hour during waking hours. Patients will be observed daily for the first week of treatment and will be called at 2 weeks and 4 weeks to check on side effects, if any. At 6 months, they will undergo a repeat eye examination. Patients (or their parents) will keep a daily diary recording the condition of each eye. Effectiveness Study Children and adults from Ann Arbor, Michigan, LaJolla, California, and the NEI clinic may be enrolled in this study. Participants will receive medication as described above for the safety trial. They will be observed daily for the first week and will have repeat eye examinations, including photographs, at months 3, 6, 9 and 12 to see if the crystals have decreased. Patients will keep a daily diary of the condition of both eyes. |
| New Formulation | NCT00010426 ↗ | Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis | Completed | Leadiant Biosciences, Inc. | N/A | 1999-12-01 | OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation. II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen. |
| New Formulation | NCT00010426 ↗ | Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis | Completed | Sigma Tau Pharmaceuticals, Inc. | N/A | 1999-12-01 | OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation. II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen. |
| New Formulation | NCT00010426 ↗ | Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis | Completed | FDA Office of Orphan Products Development | N/A | 1999-12-01 | OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation. II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen. |
| >Trial Type | >Trial ID | >Title | >Status | >Sponsor | >Phase | >Start Date | >Summary |
All Clinical Trials for CYSTEAMINE HYDROCHLORIDE
| Trial ID | Title | Status | Sponsor | Phase | Start Date | Summary |
|---|---|---|---|---|---|---|
| NCT00001213 ↗ | Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis | Completed | National Eye Institute (NEI) | Phase 2 | 1986-04-01 | Cystinosis is an inherited disease that results in poor growth and kidney disease, among other things. The damage to the kidneys and other organs is thought to be due to accumulation of cystine inside the cells of various body tissues. This chemical also accumulates in the cornea-the covering of the eye over the pupil and iris. After 10 to 20 years, the corneas of some patients become so packed with crystals that the surfaces may become irregular, occasionally causing small, painful breaks. Patients enrolled in a NIH study on cystinosis are receiving the drug cysteamine. Taken by mouth, this drug reduces cystine in some tissues, but not in the cornea. This study began in 1986 to test whether cysteamine eye drops could prevent or reduce corneal cystine crystals in these patients. The drops have been very effective in removing crystals and reducing pain in patients who take the medication as directed. Patients who do not take the medication as prescribed do not benefit. After the effectiveness of the drops was proven, the main purpose was modified to continue to evaluate the long-term safety and effectiveness of cysteamine eye drops for treating cystine crystals in the corneas of patients with cystinosis until the drops are approved by the Food and Drug Administration (FDA). When the New Drug Application (NDA) for the Sigma-Tau standard formulation is granted, this protocol will be terminated. |
| NCT00001736 ↗ | New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis | Completed | National Eye Institute (NEI) | Phase 1 | 1998-05-01 | This study will evaluate the safety and effectiveness of a new formulation of eye drops used to treat cystine crystals that form in the corneas of patients with cystinosis. Cystinosis is an inherited disease caused by a defective enzyme, in which excessive amounts of the amino acid cystine accumulate in the body. Among others, symptoms include poor growth and development of kidney failure. In addition, after 10 to 20 years, the cornea-the outside covering of the eye over the iris and pupils-becomes so packed with cystine crystals that small, painful breaks may develop. This corneal condition is treated with cysteamine eye drops. This study is designed to provide additional information about this medication that the Food and Drug Administration requires before approving it for marketing. The study will examine, in two separate but simultaneous investigations, the safety and effectiveness of a new cysteamine formulation. In both studies, before treatment begins, patients will have a complete eye examination, and photographs of the eye will be taken using a bright flash. Safety Study Children and adults currently enrolled in a cystinosis study at the National Institutes of Health may participate in this trial. They will receive the current cysteamine formulation in one eye and the new preparation in the other eye. The drops will be given every hour during waking hours. Patients will be observed daily for the first week of treatment and will be called at 2 weeks and 4 weeks to check on side effects, if any. At 6 months, they will undergo a repeat eye examination. Patients (or their parents) will keep a daily diary recording the condition of each eye. Effectiveness Study Children and adults from Ann Arbor, Michigan, LaJolla, California, and the NEI clinic may be enrolled in this study. Participants will receive medication as described above for the safety trial. They will be observed daily for the first week and will have repeat eye examinations, including photographs, at months 3, 6, 9 and 12 to see if the crystals have decreased. Patients will keep a daily diary of the condition of both eyes. |
| NCT00002110 ↗ | A Phase II/III Study of Cysteamine (Mercaptoethylamine) and Zidovudine for the Treatment of HIV Disease | Completed | Mylan Laboratories | Phase 2 | 1969-12-31 | To determine the safety and tolerance of low-dose versus high-dose cysteamine administered concurrently with zidovudine (AZT). To determine the pharmacokinetics and effects on immune function and viral load in patients receiving these drug regimens. |
| NCT00010426 ↗ | Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis | Completed | Leadiant Biosciences, Inc. | N/A | 1999-12-01 | OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation. II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen. |
| NCT00010426 ↗ | Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis | Completed | Sigma Tau Pharmaceuticals, Inc. | N/A | 1999-12-01 | OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation. II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen. |
| NCT00010426 ↗ | Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis | Completed | FDA Office of Orphan Products Development | N/A | 1999-12-01 | OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation. II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen. |
| 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. |
| >Trial ID | >Title | >Status | >Sponsor | >Phase | >Start Date | >Summary |
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