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Main
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Note: This record shows only the 20 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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17 October 2012 |
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Main ID: |
NCT00107783 |
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Date of registration:
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07/04/2005 |
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Primary sponsor: |
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Public title:
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Long-Term Study of Nitisinone to Treat Alkaptonuria
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Scientific title:
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Long-Term Clinical Trial of Nitisinone in Alkaptonuria |
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Date of first enrolment:
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January 2005 |
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Target sample size:
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40 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00107783 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Outcomes Assessor), Primary Purpose: Treatment
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Countries of recruitment
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United States
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Key inclusion & exclusion criteria
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- INCLUSION CRITERIA:
- Age 30-80 years, either gender
- Diagnosis of alkaptonuria based upon urinary HGA excretion greater than 0.4 g/24h
- At least one hip joint remaining
- Some evidence of hip involvement, e.g., pain or decreased range of motion
- Ability to travel to the NIH Clinical Research Center for admissions
- Ability to consent
- Availability of local medical follow-up
EXCLUSION CRITERIA:
- Age less than 30 or greater than 80
- Non-alkaptonuria causes of ochronosis
- Bilateral hip joint replacement
- Keratopathy
- Contact lenses
- Uncontrolled glaucoma
- History of myocardial infarction
- History of emphysema or pulmonary insufficiency (Forced vital capacity less than 70%)
- Psychiatric illness or neurological disease that interferes with compliance or
communication with health care personnel
- Current malignancy
- Open skin lesions
- Dietary habits or use of homeopathic therapies that interfere with tyrosine
catabolism. The diet must be reasonably balanced, as determined by a dietician.
- Uncontrolled hypertension (blood pressure greater than 180 systolic or greater than
95 diastolic)
- History of extreme alcohol abuse or sever liver disease
- Liver greater than 3 cm below the right costal margin
- Electrocardiogram changes indicative of myocardial infarction, arrhythmia,
tachycardia, bradycardia, left bundle branch block
- Chest radiographic abnormalities, including an infiltrate, mass, congestive heart
failure, embolism, atelectasis
- Serum postassium less than 3. 0 mEq/L
- Serum creatinine greater than 2.0 mg/dL
- SGPT greater than 41 U/L or SGOT greater than 34 U/L
- CK greater than 500 U/L
- Hemoglobin less than 10.0 g/dL
- Platelets less than 100 k/mm(3)
- WBC less than 3.0 k/microL
- T4 greater than 15 microg/dL
- T4 less than 4 microg/dL
- ESR greater than 100 mm/h
- Plasma tyrosine greater than 150 microM
Age minimum:
30 Years
Age maximum:
80 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Alkaptonuria
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Intervention(s)
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Drug: Nitisinone (NTBC)
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Primary Outcome(s)
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Change in Total ROM Worse Hip.
[Time Frame: Measured at baseline and at 36 months]
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Secondary Outcome(s)
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Change in 6 Minute Walk Test (6MWT)
[Time Frame: Measured at baseline and at 36 months]
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Change in Functional Reach Assessment
[Time Frame: Measured at baseline and at 36 months]
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Change in Schober's Test
[Time Frame: Measured at baseline and at 36 months]
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Change in Timed Get up and go
[Time Frame: Measured at baseline and at 36 months]
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Secondary ID(s)
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05-HG-0076
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050076
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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