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Hurler Syndrome clinical trials

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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NCT ID: NCT00286689 Withdrawn - HIV Infections Clinical Trials

Effects of Growth Hormone in Chronically Ill Children

Start date: February 3, 2006
Phase: N/A
Study type: Interventional

The specific aims for this study are - 1. To determine the effect of GH on height, height velocity, body weight and lean body mass. This specific aim tests the hypothesis that GH significantly improves height, height velocity, weight, weight velocity and lean body mass in chronically ill children who have grown poorly despite adequate nutritional rehabilitation. 2. To determine the effect of GH on whole body protein turnover (WBPT), IGF-1 levels and on cytokines. This specific aim tests the hypothesis that chronically ill children have increased catabolism, caused by high levels of circulating cytokines and low levels of IGF-1, and that these abnormalities improve with GH treatment. 3. Evaluation of bone mineral density and bone turnover. This specific aim tests the hypothesis that bone density is low in chronically ill children secondary to increased osteoclast activity correlating with elevated cytokine levels. We hypothesize that the anabolic effects of growth hormone (GH) will improve the height and weight of chronically ill children who have failed to grow despite receiving adequate nutrition via gastrostomy tube or oral supplementation.