Use of cxcr4 antagonists

An antagonist, methylene technology, applied in the field of use of CXCR4 antagonists

Inactive Publication Date: 2014-02-19
GENZYME CORP +1
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  • Summary
  • Abstract
  • Description
  • Claims
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Problems solved by technology

[0013] None of the aforementioned documents specifically mentions the use of the CXCR4 antagonists disclosed therein for the treatment or prevention of WHIM syndrome or congenital myeloid agranulocytosis or the diseases or conditions disclosed herein

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  • Use of cxcr4 antagonists
  • Use of cxcr4 antagonists
  • Use of cxcr4 antagonists

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example 1

[0389] Example 1: Clinical Trial Results - Plerixafor is a potential therapy for congenital myeloid agranulocytosis, WHIM syndrome.

[0390] Research design: This is an open-label, single-center, phase I study to examine the hematological effects, pharmacokinetics, and safety of plerixafor in patients with congenital myelogranulocytosis due to CXCR4 mutations. The study used serial, escalating doses of plerixafor administered on days 1, 3, 5, 8, and 10. Five escalating dose levels, 20mcg / kg, 40mcg / kg, 80mcg / kg and 240mcg / kg, were examined daily in patients. Subjects were patients at the University of Washington Comprehensive Clinical Research Center for up to 10 days; the study required subject participation for up to 14 days. Patients were monitored for the hematological effects of plerixafor and observed for adverse reactions. Plerixafor will be discontinued if a normal blood neutrophil count is achieved and maintained for at least 24 hours prior to the highest dose.

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Abstract

Presently disclosed are methods and compositions for treating or preventing WHIM syndrome and certain other disorders or conditions with a certain CXCR4 antagonist.

Description

[0001] related application [0002] This application claims the benefit of US Provisional Application No. 61 / 486,632, filed May 16, 2011. The entire teachings of the aforementioned applications are incorporated herein by reference. [0003] The disclosed invention is directed to the use of certain CXCR4 antagonists for the treatment or prevention of WHIM syndrome and certain other diseases or conditions, and the use of corresponding pharmaceutical compositions comprising these CXCR4 antagonists. Background of the invention [0004] WHIM (warts, hypogammaglobulinemia, immunodeficiency, congenital myelogranulocytosis) syndrome is a rare disorder characterized by warts, hypogammaglobulinemia, recurrent bacterial infections, and leukocytes Decrease and neutropenia. Neutropenia is thought to be due to congenital myelogranulocytosis, a form of neutropenia associated with abnormal retention of mature neutrophils in the bone marrow and hypercellularity of the bone marrow. Balabania...

Claims

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Application Information

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IPC IPC(8): C07D255/02
CPCA61K31/395A61K38/193A61K45/06A61K31/4178A61K31/4427A61K31/52A61K2300/00A61P7/06A61K9/0019A61K2121/00C07D255/02
Inventor D.C.戴尔G.J.布里杰F.J.许
Owner GENZYME CORP
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