Personalized dosing of dichloroacetate for the treatment of rare and common diseases
二氯乙酸治疗罕见病和常见病的个性化剂量
基本信息
- 批准号:9517985
- 负责人:
- 金额:$ 69.51万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2017
- 资助国家:美国
- 起止时间:2017-07-01 至 2020-07-13
- 项目状态:已结题
- 来源:
- 关键词:AffectBiological AssayCLIA certifiedCessation of lifeChildChronicCollectionDataDichloroacetateDiseaseDoseDrug KineticsFailureFloridaGSTZ1 geneGeneticGenotypeGoalsGuidelinesHaplotypesIndividualInstructionInterventionKineticsLabelLaboratory ResearchLactic AcidosisLeadLife ExpectancyMitochondrial DiseasesNeuraxisNeurologicPatientsPhasePhase III Clinical TrialsPhysiciansPlasmaPreparationPrevalenceProceduresProcessProteinsPyruvate Dehydrogenase ComplexPyruvate Dehydrogenase Complex Deficiency DiseaseRare DiseasesRegimenRegulationResearchResearch DesignResidual stateRiskSafetySmall Business Technology Transfer ResearchTestingTissuesToxic effectUniversitiesVariantbasecommercializationcosteffective therapygenotyped patientsimprovedneuromuscularneurotoxicnovelopen labelprecision geneticsprospectiveresponsetargeted treatment
项目摘要
Pyruvate dehydrogenase complex (PDC) deficiency (PDCD) is a rare disease of mitochondrial energy failure in
which the life of expectancy of affected children is severely truncated. Treatment of PDCD remains a serious,
unmet, challenge. There has never been a controlled trial of any intervention for PDCD; thus, there is no
proven therapy for affected patients. Dichloroacetate (DCA) represents the first targeted therapy for PDCD by
stimulating residual PDC activity in all tissues, including the central nervous system (CNS). Based on both
controlled trials and open label studies of DCA in mitochondrial diseases, Medosome Biotec, LLC (MBT) and
its research partners at the University of Florida (UF) (Drs. Peter Stacpoole and Taimour Langaee, PD/PIs for
Phase I) determined the data were sufficiently compelling to justify a pivotal phase 3 trial of DCA in this
disease, for which Dr. Stacpoole at UF and Dr. J.L.P. Thompson at Columbia University are PD/PIs. In
response to these promising studies and extensive potential commercial market, Medosome Biotec, LLC
(MBT) and its research partners at UF have developed a central STTR hypothesis that GSTZ1 haplotypebased
dosing of DCA has the potential to provide safe and effective treatment for PDCD and other diseases for
which DCA may be beneficial. The team proposes a STTR Fast Track to test this. Phase I: Develop a
procedure for GSTZ1 haplotypes analysis (referred to as Halotype Identification Procedure or HIP) that can be
used by physicians for personalized dosing of DCA. Phase I Specific Aim: 1) Establish the accuracy and
validity of the GSTZ1 haplotype analysis by determining the plasma pharmacokinetics (PK) of DCA in subjects
identified in Specific Aim 2 who are predicted to be slow or fast DCA metabolizers, based on GSTZ1 haplotype
analysis. Phase II: Use the new procedure to accurately genotype and dose-stratify PDCD patients for the
Phase 3 trial. The team will test these hypotheses by accomplishing the following Phase II Specific Aims: 1)
Conduct a pivotal Phase 3 trial, consistent with FDA guidelines, that could lead to the agency’s approval of
DCA for PDCD, using the procedure developed during Phase I to provide personalized, genetics-based dosing
to improve the safety of chronic DCA treatment; and 2) Commercialize the kit and analytic procedures for use
in PDCD and other diseases amenable to DCA treatment.
丙酮酸脱氢酶复合物缺乏症(PDCD)是一种罕见的线粒体能量衰竭疾病
项目成果
期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
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Peter Wallace Stacpoole其他文献
Peter Wallace Stacpoole的其他文献
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{{ truncateString('Peter Wallace Stacpoole', 18)}}的其他基金
Personalized dosing of dichloroacetate for the treatment of rare and common diseases
二氯乙酸治疗罕见病和常见病的个性化剂量
- 批准号:
10471783 - 财政年份:2017
- 资助金额:
$ 69.51万 - 项目类别:
Personalized dosing of dichloroacetate for the treatment of rare and common diseases
二氯乙酸治疗罕见病和常见病的个性化剂量
- 批准号:
10216314 - 财政年份:2017
- 资助金额:
$ 69.51万 - 项目类别:
Personalized dosing of dichloroacetate for the treatment of rare and common diseases
二氯乙酸治疗罕见病和常见病的个性化剂量
- 批准号:
10010536 - 财政年份:2017
- 资助金额:
$ 69.51万 - 项目类别:
Phase 3 Trial of DCA in PDC Deficiency IND 028,625 (02/04/2015)
DCA 治疗 PDC 缺陷的 3 期试验 IND 028,625 (02/04/2015)
- 批准号:
10436136 - 财政年份:2016
- 资助金额:
$ 69.51万 - 项目类别:
Phase 3 Trial of DCA in PDC Deficiency IND 028,625 (02/04/2015)
DCA 治疗 PDC 缺陷的 3 期试验 IND 028,625 (02/04/2015)
- 批准号:
9625517 - 财政年份:2016
- 资助金额:
$ 69.51万 - 项目类别:
Planning Grant for Phase 3 Trial of Dichloroacetate in PDH Deficiency
二氯乙酸治疗 PDH 缺乏症第三阶段试验的规划拨款
- 批准号:
7976507 - 财政年份:2010
- 资助金额:
$ 69.51万 - 项目类别:
Pharmacotoxicology of Trichloroethylene Metabolites
三氯乙烯代谢物的药理学
- 批准号:
7812724 - 财政年份:2009
- 资助金额:
$ 69.51万 - 项目类别:
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