WO2015033224A2 - Méthodes de traitement du syndrome de l'x fragile et de troubles associés - Google Patents
Méthodes de traitement du syndrome de l'x fragile et de troubles associés Download PDFInfo
- Publication number
- WO2015033224A2 WO2015033224A2 PCT/IB2014/002398 IB2014002398W WO2015033224A2 WO 2015033224 A2 WO2015033224 A2 WO 2015033224A2 IB 2014002398 W IB2014002398 W IB 2014002398W WO 2015033224 A2 WO2015033224 A2 WO 2015033224A2
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- metadoxine
- mice
- treatment
- fmrl knockout
- administration
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/4415—Pyridoxine, i.e. Vitamin B6
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
- A61K31/4015—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil having oxo groups directly attached to the heterocyclic ring, e.g. piracetam, ethosuximide
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis
Definitions
- the metadoxme or acceptable derivative thereof may be formulated for immediate release upon administration to the subject.
- the metadoxme or acceptable derivative thereof may be formulated for sustained and/or controlled release, and may optionally be formulated to have both immediate release and sustained and/or controlled release
- an embodiment of the method of the present invention is to administer the therapeutic compound described herein in a sustained release form.
- Any controlled or sustained release method known to those of ordinary skill in the art may be used with the compositions and methods of the invention such as those described in Langer, Science 249(4976): 1527-33 ( 1990).
- Such method comprises administering a sustained-release composition, a suppository, or a coated implantable medical device so that a therapeutically effective dose of the composition of the invention is continuously delivered to a subject of such a method.
- the metadoxine or metadoxine derivative in compositions used by the invention may be formulated for sustained or controlled release over a period of between about 0.5 or 1 or 2 or 3 or 4 hours and about 5, 6, 7, 8, 9, 10, 1 1 or 12 hours. In certain embodiments, the metadoxine or metadoxine derivative in compositions used by the invention may be formulated for sustained or controlled release over a period of between about 5 or 6 or 7 or 8 hours and about 9, 10, 11 or 12 hours.
- the metadoxine or metadoxine derivative in compositions used by the invention may be formulated to release up to 5, 10, 15, 20, 25, 30, 35, 40, 45, 50, 55, 60, 65, 70, 75, 80, 85, 90, 95, 99, 99.5 or 100% of the total metadoxine or metadoxine derivative in about 0.5, 1, 2, 3, 4, 5, 6, 7 or 8 hours.
- metadoxine or metadoxine derivatives formulations of the present invention may be administered in a sufficient amount to produce a reasonable benefit/risk ratio applicable to a selected treatment, as may be determined by the skilled artisan.
- salt adducf is meant to encompass a salt product of a direct addition of two or more distinct ions, wherein the overall charge of the salt adduct is zero.
- the salt adduct comprises one positively charged moiety having a single positive charge functional group (i.e., the positively charged moiety is charged with H- 1 net charge) and one negatively charged moiety having a single negative charge functional group (i.e., the negatively charged moiety is charged with -1 net charge).
- the salt adduct comprises a positively charged moiety charged with +n net charge (originating from one or more positively charged functional groups, which may be the same or different), and a negatively charge moiety having -n (originating from one or more negatively charged functional groups, which may be the same or different) net charge, wherein n is an integer which may be equal to 1 , 2, 3, 4, 5 or 6.
- the successive alleys test effectively measured anxiety (latency to enter the Alley 1 ) and hyperactivity (Alleys 2 to 4). Progression from Alley 1 through the successive Alleys 2, 3, and 4 was associated with exposure to an increasingly brightly colored environment with increasingly lower wails and narrower, more exposed open arms. Time spent on, and entries into, the open arms indicated anxiety; conversely, increasing time spent in more open arms reflected hyperactivity. These factors allowed for a sensitive test bracketing a range of anxiety-like behaviors together with hyperactivity.
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Urology & Nephrology (AREA)
- Immunology (AREA)
- Analytical Chemistry (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- General Physics & Mathematics (AREA)
- Pathology (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (AREA)
- Cell Biology (AREA)
- Food Science & Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Plant Substances (AREA)
- Steroid Compounds (AREA)
Abstract
Priority Applications (11)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CA2922901A CA2922901A1 (fr) | 2013-09-09 | 2014-09-09 | Methodes de traitement du syndrome de l'x fragile et de troubles associes |
AU2014316779A AU2014316779A1 (en) | 2013-09-09 | 2014-09-09 | Methods of treating fragile X Syndrome and related disorders |
KR1020167009040A KR20160078956A (ko) | 2013-09-09 | 2014-09-09 | 취약 x 증후군 및 관련 장애의 치료 방법 |
CN201480049671.5A CN105517546A (zh) | 2013-09-09 | 2014-09-09 | 治疗脆性x综合征及相关疾病的方法 |
EA201690557A EA201690557A1 (ru) | 2013-09-09 | 2014-09-09 | Способы лечения синдрома ломкой х-хромосомы и связанных расстройств |
EP14830858.8A EP3043792A2 (fr) | 2013-09-09 | 2014-09-09 | Méthodes de traitement du syndrome de l'x fragile et de troubles associés |
SG11201601605YA SG11201601605YA (en) | 2013-09-09 | 2014-09-09 | Methods of treating fragile x syndrome and related disorders |
JP2016539645A JP2016530291A (ja) | 2013-09-09 | 2014-09-09 | 脆弱x症候群および関連障害の処置方法 |
MX2016003006A MX2016003006A (es) | 2013-09-09 | 2014-09-09 | Metodos para tratar el sindrome de x fragil y trastornos relacionados. |
US14/917,169 US9851354B2 (en) | 2013-09-09 | 2014-09-09 | Methods of treating fragile X syndrome and related disorders |
IL244343A IL244343A0 (en) | 2013-09-09 | 2016-02-29 | Methods for the treatment of fragile x syndrome and related disorders |
Applications Claiming Priority (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361875384P | 2013-09-09 | 2013-09-09 | |
US61/875,384 | 2013-09-09 | ||
US14/038,258 | 2013-09-26 | ||
US14/038,258 US20150073023A1 (en) | 2013-09-09 | 2013-09-26 | Method Of Treating Fragile X Syndrome And Related Disorders |
US201461991351P | 2014-05-09 | 2014-05-09 | |
US61/991,351 | 2014-05-09 |
Publications (2)
Publication Number | Publication Date |
---|---|
WO2015033224A2 true WO2015033224A2 (fr) | 2015-03-12 |
WO2015033224A3 WO2015033224A3 (fr) | 2015-07-02 |
Family
ID=52629036
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/IB2014/002398 WO2015033224A2 (fr) | 2013-09-09 | 2014-09-09 | Méthodes de traitement du syndrome de l'x fragile et de troubles associés |
PCT/US2014/054816 WO2015035402A1 (fr) | 2013-09-09 | 2014-09-09 | Procédés de détermination d'une réponse à un traitement |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2014/054816 WO2015035402A1 (fr) | 2013-09-09 | 2014-09-09 | Procédés de détermination d'une réponse à un traitement |
Country Status (12)
Country | Link |
---|---|
EP (2) | EP3043792A2 (fr) |
JP (2) | JP2016530536A (fr) |
KR (2) | KR20160078956A (fr) |
CN (2) | CN105917225A (fr) |
AU (2) | AU2014316779A1 (fr) |
CA (2) | CA2923421A1 (fr) |
EA (2) | EA201690557A1 (fr) |
IL (2) | IL244343A0 (fr) |
MX (2) | MX2016003002A (fr) |
SG (2) | SG11201601830PA (fr) |
TW (2) | TW201605443A (fr) |
WO (2) | WO2015033224A2 (fr) |
Cited By (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9851354B2 (en) | 2013-09-09 | 2017-12-26 | Alcobra Ltd. | Methods of treating fragile X syndrome and related disorders |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3239161A1 (fr) * | 2013-07-31 | 2017-11-01 | UDC Ireland Limited | Complexes de carbène-métal diazabenzimidazole luminescent |
CN108538365A (zh) * | 2017-03-24 | 2018-09-14 | 华东师范大学 | 一种基于数据分析技术的自闭症社交能力评估系统 |
CN112055587A (zh) * | 2018-04-13 | 2020-12-08 | Healx有限公司 | 脆性x综合征的治疗 |
KR20190121569A (ko) * | 2018-04-18 | 2019-10-28 | 건국대학교 글로컬산학협력단 | 아그마틴 및 이의 약학적으로 허용 가능한 염을 포함하는 취약 x 증후군 예방 또는 치료용 약학조성물 |
AU2019280980A1 (en) * | 2018-06-07 | 2021-01-07 | Ovid Therapeutics Inc. | Use of (S)-3-amino-4-(difluoromethylenyl) cyclopent-1-ene-1-carboxylic acid and related compounds, (1S,3S)-3-amino-4-(difluoromethylidene) cyclopentane-1-carboxylic acid and vigabatrin in the treatment of developmental disorders |
WO2021141426A1 (fr) * | 2020-01-08 | 2021-07-15 | 건국대학교 글로컬산학협력단 | Composition de traitement du syndrome de l'x fragile ou des troubles du développement associés, comprenant un composé de lisuride en tant que principe actif |
CN115397414A (zh) * | 2020-02-07 | 2022-11-25 | 株式会社纽若梵提 | 包含利美尼定的用于治疗脆性x染色体综合征的组合物 |
Citations (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4313952A (en) | 1980-06-30 | 1982-02-02 | Massimo Baldacci | Method of treating acute alcoholic intoxication with pyridoxine P.C.A. |
WO2009004629A2 (fr) | 2007-07-03 | 2009-01-08 | Alcobra Ltd. | Procédé pour réduire les symptômes de consommation d'alcool |
Family Cites Families (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20020155170A1 (en) * | 2000-11-30 | 2002-10-24 | Walsh William John | Nutrient supplements and methods for treating autism and for preventing the onset of autism |
EP2445498A1 (fr) * | 2009-06-25 | 2012-05-02 | Alcobra Ltd. | Procédé pour le traitement, l'atténuation de symptômes et le soulagement, l'amélioration et la prévention d'une maladie, d'un trouble ou d'un état cognitif |
-
2014
- 2014-09-05 TW TW103130856A patent/TW201605443A/zh unknown
- 2014-09-05 TW TW103130859A patent/TW201606304A/zh unknown
- 2014-09-09 KR KR1020167009040A patent/KR20160078956A/ko not_active Application Discontinuation
- 2014-09-09 EA EA201690557A patent/EA201690557A1/ru unknown
- 2014-09-09 CN CN201480060722.4A patent/CN105917225A/zh active Pending
- 2014-09-09 JP JP2016540930A patent/JP2016530536A/ja active Pending
- 2014-09-09 MX MX2016003002A patent/MX2016003002A/es unknown
- 2014-09-09 KR KR1020167009042A patent/KR20160086818A/ko not_active Application Discontinuation
- 2014-09-09 EA EA201690559A patent/EA201690559A1/ru unknown
- 2014-09-09 AU AU2014316779A patent/AU2014316779A1/en not_active Abandoned
- 2014-09-09 WO PCT/IB2014/002398 patent/WO2015033224A2/fr active Application Filing
- 2014-09-09 MX MX2016003006A patent/MX2016003006A/es unknown
- 2014-09-09 CN CN201480049671.5A patent/CN105517546A/zh active Pending
- 2014-09-09 SG SG11201601830PA patent/SG11201601830PA/en unknown
- 2014-09-09 CA CA2923421A patent/CA2923421A1/fr not_active Abandoned
- 2014-09-09 EP EP14830858.8A patent/EP3043792A2/fr not_active Withdrawn
- 2014-09-09 JP JP2016539645A patent/JP2016530291A/ja active Pending
- 2014-09-09 CA CA2922901A patent/CA2922901A1/fr not_active Abandoned
- 2014-09-09 SG SG11201601605YA patent/SG11201601605YA/en unknown
- 2014-09-09 AU AU2014315026A patent/AU2014315026A1/en not_active Abandoned
- 2014-09-09 EP EP14776939.2A patent/EP3044589A1/fr not_active Withdrawn
- 2014-09-09 WO PCT/US2014/054816 patent/WO2015035402A1/fr active Application Filing
-
2016
- 2016-02-29 IL IL244343A patent/IL244343A0/en unknown
- 2016-03-06 IL IL244453A patent/IL244453A0/en unknown
Patent Citations (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4313952A (en) | 1980-06-30 | 1982-02-02 | Massimo Baldacci | Method of treating acute alcoholic intoxication with pyridoxine P.C.A. |
WO2009004629A2 (fr) | 2007-07-03 | 2009-01-08 | Alcobra Ltd. | Procédé pour réduire les symptômes de consommation d'alcool |
Non-Patent Citations (5)
Title |
---|
"Remington's Pharmaceutical Sciences", 1990, MACK PUB. |
LANGER, SCIENCE, vol. 249, no. 4976, 1990, pages 1527 - 33 |
LU YUAN ET AL., CHIN. MED. 1, vol. 120, no. 2, 2007, pages 160 - 168 |
LU YUAN ET AL., CHIN. MED. J, vol. 120, no. 2, 2007, pages 160 - 168 |
MCCRACKEN, J. T. ET AL., N. ENGL. J. MED., vol. 347, 2002, pages 314 - 321 |
Cited By (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9851354B2 (en) | 2013-09-09 | 2017-12-26 | Alcobra Ltd. | Methods of treating fragile X syndrome and related disorders |
US9851355B2 (en) | 2013-09-09 | 2017-12-26 | Alcobra Ltd. | Methods of determining response to therapy |
Also Published As
Publication number | Publication date |
---|---|
IL244343A0 (en) | 2016-04-21 |
CA2922901A1 (fr) | 2015-03-12 |
WO2015033224A3 (fr) | 2015-07-02 |
CA2923421A1 (fr) | 2015-03-12 |
TW201605443A (zh) | 2016-02-16 |
EA201690559A1 (ru) | 2016-08-31 |
JP2016530291A (ja) | 2016-09-29 |
CN105917225A (zh) | 2016-08-31 |
MX2016003006A (es) | 2016-06-10 |
EP3044589A1 (fr) | 2016-07-20 |
SG11201601605YA (en) | 2016-04-28 |
MX2016003002A (es) | 2016-09-08 |
AU2014315026A1 (en) | 2016-03-24 |
EP3043792A2 (fr) | 2016-07-20 |
IL244453A0 (en) | 2016-04-21 |
WO2015035402A1 (fr) | 2015-03-12 |
TW201606304A (zh) | 2016-02-16 |
KR20160078956A (ko) | 2016-07-05 |
SG11201601830PA (en) | 2016-04-28 |
CN105517546A (zh) | 2016-04-20 |
AU2014316779A1 (en) | 2016-03-17 |
EA201690557A1 (ru) | 2016-07-29 |
KR20160086818A (ko) | 2016-07-20 |
JP2016530536A (ja) | 2016-09-29 |
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