Clinical trials: Deformity; Bone
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Fanconi Anemia
Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
The purpose of this research study is to: (1) determine if the combination of low dose total body irradiation, low dose cyclophosphamide and the addition of fludarabine, and a serum to suppress the immune system can allow…
- Trial name
- NCT00595127
- Testing
- Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
Craniosynostoses
Neurodevelopmental Outcomes in Craniosynostosis Repair
Context: Craniosynostosis is a common craniofacial abnormality which can be associated with various clinical syndromes. Though it has been established that children with craniosynostosis score lower on certain developmental…
- Trial name
- NCT04072783
- Testing
- MRI,DTI, functional MRI
- Where
- United States
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- University of Arkansas
Polydactyly Toe, Thumb Hypoplasia
Clonidine for Tourniquet-related Pain in Children
This study is designed as a randomized, placebo-controlled, double-blinded, clinical trial with two parallel groups. This study investigates the association between a single dose of the drug, Clonidine, and the total amount of…
- Conditions
- Polydactyly Toe, Thumb Hypoplasia, Trigger Finger, Polydactyly; Fingers
- Trial name
- CLOTCH (NCT04564430)
- Testing
- Catapresan, Saline
- Where
- Denmark
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Odense University Hospital
Short Stature
The Role of Igf-1 Generation Test in Diagnosis and Treatment of Short Stature
The purpose of the study is to evaluate the predictive value of IGF-1 generation test for growth velocity during GH treatment for 12 months.
- Trial name
- NCT01970800
- Testing
- Growth Hormone
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Maimonides Medical Center
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Myelodysplastic Syndromes, Beta-thalassemia
Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL.
- Trial name
- NCT04973280
- Where
- 4 sites in Austria, Belgium and 2 more
- Volunteers
- 187 people, healthy volunteers welcome
- Sponsor
- Celgene
Osteoarthritis, Traumatic Arthritis
Prospective Multicenter Post Approval Study of the LPS-Flex Mobile Bearing Knee
The purpose of the study is to assess the long-term performance of the NexGen LPS-Flex Mobile Bearing Knee in the treatment of patients with severe knee pain or degenerative knee disease.
- Conditions
- Osteoarthritis, Traumatic Arthritis, Avascular Necrosis of the Femoral Condyle, Moderate Varus
- Trial name
- NCT00854295
- Testing
- NexGen LPS-Flex Mobile Bearing Knee
- Where
- 3 sites in United States
- Volunteers
- 266 people, healthy volunteers welcome
- Sponsor
- Zimmer Biomet
Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism
Growing up With the Young Endocrine Support System (YESS!)
Transition from paediatric to adult endocrinology is a challenge for adolescents, families and doctors. Up to 25% of young adults with chronic endocrine disorders are lost to follow-up ('drop-out') once the young adult moves out…
- Conditions
- Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism, Growth Hormone Deficiency, Combined Pituitary Hormone Deficiency
- Trial name
- YESS (NCT04252001)
- Testing
- YESS! game, Snake '97, Transition-toolkit
- Where
- 7 sites in Belgium, Netherlands and 2 more
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Pectus Carinatum
Impact of Risser Stage on Pressure of Correction in Pectus Carinatum
Pectus carinatum (PC) is a deformity of the anterior chest wall which is a common pediatric condition, characterized by an idiopathic overgrowth of the costal cartilages resulting in protrusion of the sternum.
- Trial name
- NCT03548922
- Testing
- Tanner stage, Risser stage, Pressure of correction
- Where
- Turkey (Türkiye)
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Marmara University
Multiple Osteochondromas
Registry of Multiple Osteochondromas
REM is a retrospective and prospective registry, finalized for care and research purposes. It is articulated in main sections - strongly related and mutually dependent on each other - corresponding to different data domains…
- Trial name
- REM (NCT04133285)
- Where
- Italy
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Luca Sangiorgi
Pectus Excavatum
Quantification of Chest Wall Changes After Nuss Bar Removal Utilizing Three-dimensional Optical Surface Scans
Pectus excavatum is the most common anterior chest wall deformity that affects up to 1:400 of newborns. If an operative correction is indicated, patients are often operated via the Ravitsch or Nuss bar procedure. The latter (i.e.
- Trial name
- CHEST (NCT04052321)
- Testing
- 3D optical surface scan
- Where
- Netherlands
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Zuyderland Medisch Centrum
Cleft Lip and Palate
Methods of Providing Information Prior to an Alveolar Bone Graft
Patients born with a cleft of the lip and/or palate may have a gap in the bone in the roof of their mouth and in the area of bone where the teeth develop.
- Trial name
- NCT04381078
- Testing
- ACE - AudioVisual Guide
- Where
- United Kingdom
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Craniosynostoses
Craniosynostosis :Surgical Treatment Modalities and Outcome
Aim of study: 1. To evaluate outcome of cases( cosmoses and cognition) after Craniosynostosis surgery 2. - To ascertain intra- and postoperative complications
- Trial name
- NCT05511168
- Testing
- Craniostnostosis surgery
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Assiut University
Hypospadias
Molecular and Structural Comparison of Hypospadic and Normal Children Prepuces
The aim of this study is to compare prepuce tissue samples from healthy male children who applied for circumcision and children with isolated hypospadias.
- Trial name
- NCT03838458
- Where
- Turkey (Türkiye)
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Trakya University
Fitness
Fitness Level in Short Stature Children and After Growth Hormone Treatment
fitness and Physical activity will be evaluate in short stature children, using the Euro Fitness testing and the Wingate anaerobic test.
- Trial name
- NCT02977091
- Testing
- change in fitness level
- Where
- Israel
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Meir Medical Center
Hemifacial Microsomia
Computer Guided for Mandibular Distraction Osteogenesis
Objectives: Distraction Osteogenesis is an integral part in management of mandibular defects associated with Hemifacial Microsomia (HFM) and post-ankylotic mandibular defects.
- Trial name
- NCT03869021
- Testing
- 3D printed surgical guide constructed, free hand surgery
- Where
- Egypt
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Yasser Mohamed Nabil Ahmed Khirat El Hadidi
Sickle Cell Disease, Sickle Cell Anemia
Decision Aid for Therapeutic Options In Sickle Cell Disease
Sickle cell disease (SCD) is an inherited disorder with chronic multi-system manifestations affecting 100,000 individuals in the US, largely of minority origin and associated with substantial morbidity, premature mortality…
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SS, Hemoglobin SC
- Trial name
- NCT02326597
- Testing
- Decision Aid Tool, Standard Practice
- Where
- United States
- Volunteers
- 134 people, healthy volunteers welcome
- Sponsor
- Emory University
Asthma, Vitamin D Resistant Rickets
Assessment Of Vitamin D Role In The Pathogenesis Of Asthma In Vitamin D Resistent Patients
Research Title: Evaluation of airway reactivity, allergy and inflammatory mediators in exhaled breath condensate in vitamin D resistant rickets.
- Trial name
- NCT01578824
- Testing
- Methacholine Challenge Test
- Where
- Israel
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Rambam Health Care Campus
Congenital Hypothyroidism, Hearing Loss
The Prevalence of Hearing Loss Among Children With Congenital Hypothyroidism
Congenital hypothyroidism (CH) occurs in 1:4000 live births. Neurological disturbances like speech delay, motor delay and lower IQ were reported in children with CH.
- Trial name
- NCT01223638
- Testing
- Audiometry
- Where
- Israel
- Volunteers
- healthy volunteers welcome
- Sponsor
- HaEmek Medical Center, Israel