Clinical Trials Logo

Clinical Trial Summary

This is an open-label, single-arm prospective pilot study to study the effects of a single dose regimen of daily growth hormone medication (Norditropin) on pre-pubertal children with Aggrecan deficiency. The growth response will be tracked over a 12 month period. A protocol extension has been approved to continue subjects on treatment for an additional 2 years.


Clinical Trial Description

This is a single center study. All participants will be recruited from the USA. The study intends to recruit pre-pubertal children for inclusion in the 1 year growth hormone treatment trial. The study also plans to collect the following detailed phenotypic data at the first study visit: height, weight, musculoskeletal evaluation, photographs, radiographs (knees, left hand), MRI of the knees. The participants will be required to return to Cincinnati Childrens Hospital Medical Center (CCHMC) for 3 study visits over the 12 month period. At the follow up visits participants will receive a routine physical/pubertal exam, joint exam, have vitals sign checked, height/weight taken, and labs drawn. A funduscopic exam will be performed to look for signs of intracranial pressure. Study participants are also required to complete phone check ins at scheduled intervals, and a locally drawn blood sample to check insulin-like growth factor (IGF1) levels approximately 3 months after treatment start date. Concomitant medications, adverse events will be recorded and updated at all study visits and phone check ins. Medication will be resupplied and shipped to the participant as needed at every point of follow up contact. If the subjects grow well enough in the first 12 months per study protocol, they are eligible to continue treatment through the study for an additional 2 years. They will continue to come to CCHMC every 6 months for follow up study visits. Study procedures will remain the same as the initial 1 year trial with the following additions: (1) local labs may need to be obtained for dose adjustment purposes, (2) repeat knee MRI may be completed for participants that are found to have early signs of joint disease on their baseline knee MRI scan. The study will enroll interested and affected relatives of the participant in the joint phenotyping protocol as well. These participants will come to CCHMC only one time to complete the phenotyping visit. This is not an outcome of the study but the information gathered from the imaging will provide insight into the effects of ACAN mutation on the joint cartilage. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03288103
Study type Interventional
Source Children's Hospital Medical Center, Cincinnati
Contact
Status Completed
Phase Phase 1/Phase 2
Start date February 1, 2018
Completion date August 31, 2023

See also
  Status Clinical Trial Phase
Completed NCT02826902 - Effect of Anesthesia on Quality of Recovery in Patients Undergoing Correctional Tibial Osteotomy - A Randomized Controlled Trial N/A
Completed NCT00235599 - The IGFBP-3 Stimulation Test: A New Tool for the Diagnosis of Growth Hormone Deficiency in Children. N/A
Completed NCT01911260 - Weekly Zinc Chelate Supplementation on Children's Growth Phase 2/Phase 3
Completed NCT02137538 - Aromatase Inhibitor Growth Study: Letrozole vs. Anastrozole Phase 4
Recruiting NCT01934270 - Growth Hormone Secretion Following the Anaerobic Exercise N/A
Completed NCT00443144 - D3-GHR Polymorphism and Turner Syndrome N/A
Recruiting NCT06295341 - Short Stature and Psychological Well-being
Recruiting NCT05849389 - Vosoritide for Short Stature in Turner Syndrome Phase 2
Recruiting NCT05829252 - Testing the Feasibility of a Novel Growth Monitoring Smartphone App
Withdrawn NCT03323177 - Long Term Effects of Nutritional Supplementation on Final Height N/A
Completed NCT02389803 - Evaluating the Effect of Nutritional Supplementation on Growth of Short and Lean Adolescents Boys N/A
Recruiting NCT06294860 - Biological Age in Children With GH Deficiency Undergoing Hormone Replacement Therapy
Recruiting NCT03123003 - Bone Age Assessment in Children Using Ultrasound Compared to Wrist X-ray N/A
Completed NCT03575221 - Natural History of the Collagen-Related Disorder Osteogenesis Imperfecta and Genotype Phenotype Correlation
Terminated NCT01237041 - Free Fatty Acids, Body Weight, and Growth Hormones Secretion in Children Phase 1/Phase 2
Completed NCT04244123 - Web-based Adherence Information Integrated Nurse-led Monitoring Clinic
Recruiting NCT01901666 - Assessment Of Gh-Igf-1 Axis In Children With Chronic Myelogenous Leukemia (CML) In Remission Phase 4
Withdrawn NCT01970800 - The Role of Igf-1 Generation Test in Diagnosis and Treatment of Short Stature N/A
Completed NCT00830141 - Study of the Molecular Basis in the Pathophysiology of Food Intake and Growth in Children N/A
Completed NCT00562705 - Effects of Growth Hormone (GH) Treatment on Eating Regulation N/A