169. Menkes disease Clinical trials / Disease details

Clinical trials : 7 / Drugs : 7 - (DrugBank : 4) / Drug target genes : 9 - Drug target pathways : 14

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04977388 (ClinicalTrials.gov)	July 12, 2021	9/7/2021	NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome	Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Adults With Occipital Horn Syndrome: Double-blind Placebo-controlled Randomized Crossover Clinical Trial	Menkes Disease;Occipital Horn Syndrome	Drug: Droxidopa;Other: Placebo	Stephen G. Kaler, MD	NULL	Recruiting	18 Years	50 Years	All	6	Phase 1/Phase 2	United States
2	NCT04337684 (ClinicalTrials.gov)	December 1, 2019	1/4/2020	Long Term Follow-up on Menkes Disease Patients	Long Term Follow-Up and Collection of Historical Control Data on Menkes Disease Patients	Menkes Disease	Drug: Long Term Follow-Up	Cyprium Therapeutics, Inc.	NULL	Enrolling by invitation	N/A	N/A	All	50		United States
3	NCT04074512 (ClinicalTrials.gov)	October 15, 2019	27/8/2019	Copper Histidinate Treatment for Menkes Disease	Copper Histidinate Treatment for Menkes Disease	Menkes Disease	Drug: Copper Histidinate	Cyprium Therapeutics, Inc.	NULL	Available	N/A	6 Years	All			NULL
4	JPRN-UMIN000030626	2014/11/28	28/12/2017	Investigation on the effectiveness of histidine copper treatment of Menkes disease patients		Menkes disease	Weekly,subcutaneous injection of histidine copper (750microgram once) is performed three times per week.It is continued every week until the end of the research during the research period.	Hamamatsu University School of Medicine	NULL	Complete: follow-up complete	Not applicable	Not applicable	Male and Female	2	Not selected	Japan
5	JPRN-UMIN000005259	2011/03/03	18/03/2011	Clinical trial of histidine-copper and diethyldithiocarbamate combination therapy for patients with Menkes disease		Menkes disease	give a dose of copper-histidine and diethyldithiocarbamate	Kobe Unniversitu Graduate School of Medicine	NULL	Complete: follow-up complete	Not applicable	Not applicable	Male and Female	2	Not selected	Japan
6	NCT00811785 (ClinicalTrials.gov)	February 27, 2009	18/12/2008	Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency	Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency	Menkes Disease;Occipital Horn Syndrome;Unexplained Copper Deficiency	Drug: Copper Histidine	Cyprium Therapeutics, Inc.	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);National Center for Complementary and Integrative Health (NCCIH)	Completed	N/A	80 Years	All	93	Phase 3	United States
7	NCT00001262 (ClinicalTrials.gov)	June 1990	3/11/1999	Copper Histidine Therapy for Menkes Diseases	Early Copper Histidine Therapy in Menkes Disease	Kinky Hair Syndrome	Drug: Copper Histidine	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	NULL	Completed	N/A	N/A	All	60	Phase 1/Phase 2	United States