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| ID | Type | Description | Link |
|---|---|---|---|
| BWH-FDR000996-EF | |||
| SLRH-CU-FDR000996-EF |
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| Name | Class |
|---|---|
| St. Luke's-Roosevelt Hospital Center | OTHER |
OBJECTIVES:
I. Determine the efficacy of cysteine hydrochloride in preventing or decreasing photosensitivity in patients with erythropoietic protoporphyria.
PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, crossover study.
Patients are randomly assigned to 1 of 2 groups to receive cysteine hydrochloride orally twice daily, 2 capsules with breakfast and 2 with lunch. Group 1 receives cysteine hydrochloride in drug ingestion period 1 followed by placebo in period 2. Group 2 receives placebo in period 1 followed by cysteine hydrochloride in period 2. Both groups ingest placebo for 1 week between the periods. Each drug ingestion period lasts 8 weeks.
Follow up phone calls are made at the end of months 1 and 3. All patients schedule follow up visits at the end of each drug ingestion period.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| cysteine hydrochloride | Drug |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics-- Documented erythropoietic protoporphyria Determination of elevated protoporphyrin within the past year Experiencing photosensitivity --Prior/Concurrent Therapy-- No concurrent use of betacarotene --Patient Characteristics-- Other: Not pregnant or nursing Fertile female patients must use effective contraception while on study and for 3 weeks thereafter
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| Name | Affiliation | Role |
|---|---|---|
| Micheline M Mathews-Roth | St. Luke's-Roosevelt Hospital Center | Study Chair |
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| ID | Term |
|---|---|
| D046351 | Protoporphyria, Erythropoietic |
| D008661 | Metabolism, Inborn Errors |
| D011164 | Porphyrias |
| D035583 | Rare Diseases |
| ID | Term |
|---|---|
| D017094 | Porphyrias, Hepatic |
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D012873 | Skin Diseases, Genetic |
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| ID | Term |
|---|---|
| D003545 | Cysteine |
| ID | Term |
|---|---|
| D000603 | Amino Acids, Sulfur |
| D013457 | Sulfur Compounds |
| D009930 | Organic Chemicals |
| D013438 | Sulfhydryl Compounds |
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| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D021542 |
| Amino Acids, Neutral |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |