Study of NTBC for Tyrosinemia I
| Status: | Recruiting |
|---|---|
| Healthy: | No |
| Age Range: | Any - 17 |
| Updated: | 3/16/2015 |
| Start Date: | October 1998 |
OBJECTIVES: I. Assess the safety and efficacy of NTBC in children with tyrosinemia I.
II. Evaluate the effects of NTBC on survival, rate of neurologic crises, improvement in
renal tubular damage, reduction in the need for liver transplantation, and reduction in the
development of hepatocarcinoma in these patients.
II. Evaluate the effects of NTBC on survival, rate of neurologic crises, improvement in
renal tubular damage, reduction in the need for liver transplantation, and reduction in the
development of hepatocarcinoma in these patients.
PROTOCOL OUTLINE: Patients are stratified according to age at onset of symptoms (0-2 months
vs 2-6 months vs greater than 6 months).
Patients receive oral NTBC twice a day. Treatment continues in the absence of unacceptable
toxicity.
Patients are followed once a month for 6 months, then every 3 months thereafter.
vs 2-6 months vs greater than 6 months).
Patients receive oral NTBC twice a day. Treatment continues in the absence of unacceptable
toxicity.
Patients are followed once a month for 6 months, then every 3 months thereafter.
- Confirmed diagnosis of tyrosinemia Detected through newborn screening before the
onset of symptoms OR Diagnosed on the basis of symptoms (liver disease, neurological
crises, growth failure) and succinylacetone in urine or blood and/or
fumarylacetoacetate dehydratase deficiency in cultured fibroblasts or liver biopsy
We found this trial at
2
sites
3181 S.W. Sam Jackson Park Rd.
Portland, Oregon 97201
Portland, Oregon 97201
503 494-8311
Oregon Health Sciences University In 1887, the inaugural class of the University of Oregon Medical...
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