187. Kabuki syndrome Clinical trials / Disease details
Clinical trials : 4 / Drugs : 9 - (DrugBank : 2) / Drug target genes : 2 - Drug target pathways : 11
| 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 | NCT04722315 (ClinicalTrials.gov) | April 1, 2021 | 24/11/2020 | Study of Modified Atkins Diet in Kabuki Syndrome | Pilot Clinical Trial of Modified Atkins Diet for Kabuki Syndrome | Kabuki Syndrome | Dietary Supplement: Modified Atkins diet | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | NULL | Recruiting | 18 Years | N/A | All | 15 | Early Phase 1 | United States |
| 2 | NCT03855631 (ClinicalTrials.gov) | September 28, 2020 | 22/2/2019 | Exploiting Epigenome Editing in Kabuki Syndrome: a New Route Towards Gene Therapy for Rare Genetic Disorders | Exploiting Epigenome Editing in Kabuki Syndrome: a New Route Towards Gene Therapy for Rare Genetic Disorders | Kabuki Syndrome 1 | Genetic: Intervention on primary cultured cells | University Hospital, Montpellier | Association Française contre les Myopathies Telethon | Completed | 6 Years | N/A | All | 8 | France | |
| 3 | EUCTR2012-000432-26-NL (EUCTR) | 26/09/2012 | 27/03/2012 | Prospective study on the metabolic and linear growth effects of growh hormone treatment in children with Kabuki Syndrome | Prospective study on the metabolic and linear growth effects of growh hormone treatment in children with Kabuki Syndrome - Metabolic effects of Growth Hormone | Kabuki Syndrome (KS, OMIM 147920) with a mutation in MLL2 gene. ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Trade Name: Genotropin Product Name: Genotropin INN or Proposed INN: SOMATROPIN Other descriptive name: recombinant DNA-derived human growth hormone | Maastricht University Medical Centre | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Netherlands | ||||
| 4 | NCT00723580 (ClinicalTrials.gov) | May 2008 | 24/7/2008 | Actigraphic Analysis of Treatment Response | Actigraphic Analysis of Treatment Response in a Six Year Old Girl With Kabuki Syndrome | Sleep Disorders, Circadian Rhythm;Insomnia;Psychomotor Agitation | Drug: risperidone | Child Psychopharmacology Institute | NULL | Completed | 2 Years | 10 Years | Female | 1 | N/A | United States |