Clinic-type studies

Clinic-type studies of children with high lead levels... 

These studies evaluated samples of non-overtly lead-intoxicated children selected to be representative of general paediatric populations. They focus mainly on asymptomatic children with lower lead body burdens than those of high-risk children in clinic-type studies. 

The IND. Before submitting an NDA to FDA, the sponsor of a drug must conduct, or arrange to be conducted, various types of non-clinical (in vitro and animal) tests and clinical (human) studies designed to demonstrate that the drug is safe and effective for its intended use. 2... 

Cellular therapy is the replacement of lost or dysfunctional tissues with new ones. Various cell types have been evaluated and considered for therapy. In the CNS, fetal neuronal tissue has been particularly evaluated for its merit in treating neurological diseases and injuries [1]. While numerous experimental and clinical transplantation studies showed that fetal neuronal transplants improve functional deficits in models of CNS diseases [2-5], others reported less positive outcomes [6, 7]. In addition, the rate of survival of fetal neuronal cells transplanted into the adult brain is relatively low, requiring large quantities of tissue, generally from several fetuses, for therapy. Researchers are looking at other opportunities for cellular therapy, particularly in the CNS. 

Lebovitz HE, Dole JF, Patwardhan R, Rappaport EB, Freed MIRosiglitazone Clinical Trials Study Group. Rosiglitazone monotherapy is effective in patients with type 2 diabetes. J Clin Endocrinol Metab 2001 86(l) 280-8. 

Raskin P, Rendell M, Riddle MC, Dole JF, Freed MI, Rosenstock JRosiglitazone Clinical Trials Study Group. A randomized trial of rosiglitazone therapy in patients with inadequately controlled insulin-treated type 2 diabetes. Diabetes Care 2001 24(7) 1226-32. 

Similarly, synthetic modification of castanospermine showed that the lipophilic 6-0-acyl derivatives were more potent inhibitors of HIV than the natural product. In particular, the 6-0-butanoyl derivative (MDL 28,574) was approximately twenty times more active than castanospermine and fifty times more active than A-butyl-DNJ. However, once inside cells, 6-0-butanoylcastanospermine appeared to be hydrolysed to release castanospermine and hence this compound can also be considered to be a pro-dmg. " Recently, this compound was reported to be tolerated well by patients during a phase II clinical trial. ° Studies in vitro have shown synergistic activity against HIV type 1 and 2 replication when castanospermine (and its 6-C>-butanoyI derivative) are combined with AZT and other similar dideoxynucleoside drags. ... 

B15. Blyth, H., and Pugh, R. J., Muscular dystrophy in childhood. The genetic aspect. A field study in the Leeds region of clinical types and their inheritance. Ann. Human Genet. 23, 127 (1959). 

Laundgren, B. Johannisson, E. Xing, S. Aedo, A. Diczfalusy, E. A clinical pharmacological study of a new type of vaginal delivey system for levonorgestrel. Contraception 1985, 26, 581-601. 

Not all clinical trials, especially large, multisite, multinational phase III studies and phase IV postmarketing surveillance, pharmacoeconomic, and quality-of-life studies, can be conducted at a CSO facility. These types of studies, and many phase II efficacy studies, are conducted in research- or university-based hospitals or other investigational sites where a sufficient patient population with the disease or disorder to be tested is available. A number of CSOs offer services to support clinical trial studies that are implemented at one or more clinical trial sites. These services can be broken down into relatively broad categories, which are summarized later. 

Apohpoprotein E2 occurs in about 1% of the population in North America, and homozygotic subjects for this isoform exhibit P-VLDL. However, overt type III hyperlipoproteinemia occurs in only one to two persons per thousand in the general population (LRC prevalence study), indicating that the occurrence of the defective alleles is necessary but not sufficient to produce clinical type III hyperlipoproteinemia. The development of overt hyperlipoproteinemia in these patients is modulated by genetic, hormonal, or environmental factors—such as hypothyroidism, glucose intolerance, decreased estrogen levels after menopause, obesity, and diet— that may lead to decreased LDL receptor activity, increased VLDL production, or increased plasma cholesterol... 

Case reports of conditions, which today would be denoted as muscular dystrophies, have appeared for over a hundred years, but only comparatively recently has a satisfactory classification of the different varieties been achieved, based on both clinical and genetic criteria. The basic classification of Walton and Nattrass (W6) with some subsequent modifications is widely accepted. However, occasional cases occur that do not fit exactly into the existing scheme, therefore minor modifications may still be required [see Walton and Gardner-Medwin (W5) ]. It hardly requires emphasizing that in biochemical investigations on muscular dystrophy the clinical type of the case under study should be known and specified in the reported findings, since there may be important biochemical differences between the varieties. Unfortunately, this has not been the case in a few reports in the literature. 

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