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Mucopolysaccharidosis I clinical trials

View clinical trials related to Mucopolysaccharidosis I.

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NCT ID: NCT04284254 Withdrawn - Clinical trials for Mucopolysaccharidosis Type IH

MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT

Start date: December 2022
Phase: Phase 1/Phase 2
Study type: Interventional

This is a single center, Phase 1/2 study in which patients with Hurler syndrome who have previously undergone allogeneic hematopoietic stem cell transplantation are treated with autologous plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system.

NCT ID: NCT02298712 Withdrawn - Clinical trials for Metabolism, Inborn Errors

Biomarker for Hurler Disease (BioHurler)

BioHurler
Start date: August 20, 2018
Phase:
Study type: Observational

Development of a new MS-based biomarker for the early and sensitive diagnosis of Hurler disease from plasma. Testing for clinical robustness, specificity and long-term stability of the biomarker.

NCT ID: NCT00286689 Withdrawn - HIV Infections Clinical Trials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.