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Sponsors and Collaborators: |
FDA Office of Orphan Products Development Bezoloven, Inc. |
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Information provided by: | FDA Office of Orphan Products Development |
ClinicalTrials.gov Identifier: | NCT00552630 |
Childhood Lead Poisoning is a widespread disease that has few effective treatments. The specific aims of this proposed clinical trial are threefold:
Condition | Intervention | Phase |
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Lead Poisoning Vitamin D Deficiency |
Device: d-penicillamine Drug: placebo |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | A Phase 2/3 Trial of d-Penicillamine Chelation in Lead-Poisoned Children |
Enrollment: | 50 |
Study Start Date: | September 2007 |
Estimated Study Completion Date: | August 2010 |
Estimated Primary Completion Date: | August 2010 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental
This group will receive d-penicillamine for 6 weeks
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Device: d-penicillamine
d-penicillamine twice daily, 15 mg/kg/day, for 6 weeks
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2: Placebo Comparator
This group will receive placebo for 6 weeks
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Drug: placebo
placebo with same characteristics as drug
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Approximately 300,000 children in the US have elevated blood lead levels (10 mcg/dl or greater). Lead poisoning in children is unequivocally harmful, producing the neurodevelopmental consequences of cognitive losses, attentional difficulties and behavioral disturbances, including antisocial or delinquent tendencies. Non-neurodevelopmental consequences of lead poisoning include impairment of heme synthesis, reduction in 1- hydroxylation of 25(OH) - cholecalciferol (the Vitamin D precursor) and renal injury that results in microproteniuria, an increased risk of hypertension and a greater likelihood of renal failure in adulthood. Despite these well-defined toxicities, treatments for childhood lead poisoning have been inadequate. Currently, chelation therapy is uniformly recommended only for children with severe lead poisoning (blood lead > 45 mcg/dl). Approved chelating agents for severe plumbism are CaNa2EDTA and succimer. For children with blood lead levels less than 45 mcg/dl treatment is fraught with difficulties including inconsistent recommendations by clinical experts, lack of proven benefit of chelation and the absence of a chelating agent approved for use in this range. d-Penicillamine is a lead chelator that has been used off-label for almost 4 decades. Several studies have suggested that d-penicillamine is both safe and effective in the treatment of low-level lead poisoning. We propose to evaluate, in a Phase II/III randomized, placebo-controlled clinical trial, the effectiveness of d-penicillamine in 50 children aged 6 months to 16 years with blood lead levels 15-25 mcg/dl. The d-penicillamine product will be a newly developed, IND-approved liquid formulation. The study will be performed in the Pediatric Environmental Health Center of Children's Hospital Boston. The primary outcome measure will be the ability of a 6-week course of d-penicillamine to produce sustained reductions in blood lead level. Secondary outcome measures will be normalization of non-neurodevelopmental physiologic aberrations known to occur with lead poisoning, specifically abnormalities in heme and Vitamin D synthesis. If this clinical trial demonstrates safety and efficacy, d-penicillamine will potentially provide another option among the limited treatment choices for lead-poisoned children. This trial will also provide a basis for examining the drug's efficacy in improving neurodevelopment outcome in children exposed to harmful amounts of lead.
Ages Eligible for Study: | 6 Months to 16 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Massachusetts | |
Children's Hospital Boston | |
Boston, Massachusetts, United States, 02115 |
Study Director: | Michael W Shannon, MD | Children's Hospital Boston |
Responsible Party: | Children's Hospital Boston ( Michael Shannon, MD ) |
Study ID Numbers: | 3361, 1 R01 FD003361-01A1 |
Study First Received: | November 1, 2007 |
Last Updated: | December 26, 2007 |
ClinicalTrials.gov Identifier: | NCT00552630 |
Health Authority: | United States: Food and Drug Administration |
lead poisoning chelation |
Vitamin D Deficiency Malnutrition Avitaminosis Penicillamine Lead Poisoning |
Poisoning Nutrition Disorders Disorders of Environmental Origin Deficiency Diseases |
Molecular Mechanisms of Pharmacological Action Therapeutic Uses Physiological Effects of Drugs Chelating Agents |
Antirheumatic Agents Protective Agents Pharmacologic Actions Antidotes |