Clinical trials: Skeletal Dysplasia
Exostoses, Multiple Hereditary
Establishing the Genetic Profile of Multiple Hereditary Exostoses (HME) in Families of BC
The purpose of this study is to establish the genetic profile of families in British Columbia with HME.
- Trial name
- NCT00473850
- Where
- Canada
- Volunteers
- 2,000 people
- Sponsor
- University of British Columbia
Osteogenesis Imperfecta
Effects of Bisphosphonates on OI-Related Hearing Loss
Osteogenesis Imperfecta-related hearing loss usually occurs in individuals with mild (type I) OI and is much earlier in onset than age-related hearing loss, with the majority of individuals experiencing some minor hearing loss in…
- Trial name
- NCT04152551
- Testing
- Risedronate Oral Tablet
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Hospital for Special Surgery, New York
Rheumatoid Arthritis, Osteoarthritis
A Study to Assess the Long-term Performance of SmartSet® HV and SmartSet® GHV Bone Cements in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of artificial hip joints implanted with two different bone cements, SmartSet® HV and SmartSet® GHV, in the treatment of patients with hip joint disease requiring a total hip…
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00872066
- Testing
- SmartSet® HV bone cement, SmartSet® GHV bone cement
- Where
- Slovakia
- Volunteers
- 243 people
- Sponsor
- DePuy International
Osteogenesis Imperfecta
A First-in-Human Study Evaluating AGA2115 in Adult Healthy Volunteers
To understand if AGA2115 is safe and well tolerated in healthy adult volunteers.
- Trial name
- NCT06086613
- Testing
- AGA2115
- Where
- United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Angitia Incorporated Limited
Osteogenesis Imperfecta
An Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta
The investigators have currently finished conducting an international multi-center trial that compares the efficacy and safety of pamidronate and zoledronate in the treatment of moderate to severe forms of Osteogenesis Imperfecta…
- Trial name
- INFOI (NCT00982124)
- Testing
- Zoledronic Acid
- Where
- Canada
- Volunteers
- 14 people
- Sponsor
- Shriners Hospitals for Children
Osteogenesis Imperfecta
Dental Malocclusion and Craniofacial Development in OI
Osteogenesis imperfecta (OI) is a rare inherited disorder that causes bones to break easily. Individuals with osteogenesis imperfecta break bones often and may have other problems, including hearing loss and pain and difficulty…
- Trial name
- NCT02934451
- Where
- 3 sites in United States, Canada
- Volunteers
- 75 people
- Sponsor
- Baylor College of Medicine
Multiple Osteochondroma, Enchondromatosis
Survey on Multiple Osteochondromas and Enchondromatoses During the Transition From Paediatric Care to Adult Care
This prospective observational study aims to understand the transition experiences and unmet needs of patients living with multiple osteochondromas or enchondromatoses during the paediatric to adult care, in order to produce a…
- Trial name
- PJMOE (NCT07839637)
- Where
- Italy
- Volunteers
- 91 people, healthy volunteers welcome
- Sponsor
- Istituto Ortopedico Rizzoli
Multiple Osteochondroma, Ollier Disease
Ready to Sail: Evaluating Sailing's Feasibility as Ergotherapy
Background Individuals with rare skeletal disorders frequently experience psychological distress, social isolation, unmet needs, and significant challenges due to limited treatment options.
- Conditions
- Multiple Osteochondroma, Ollier Disease, Osteogenesis Imperfecta
- Trial name
- PaS (NCT06397443)
- Testing
- 5-day sailing activity
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Istituto Ortopedico Rizzoli
Skeletal Dysplasia, Reverse Overjet
Assessment of Artificial Intelligence for Treatment Decision Recommendation of Adult Skeletal Class III Patients
The study titled "Reliability Of Artificial Intelligence for Treatment Decision Recommendation of Adult Skeletal Class III Patients" aims to assess the accuracy and dependability of artificial intelligence (AI) in providing…
- Trial name
- NCT06002373
- Where
- Egypt
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Cairo University
Coronary Disease
Platelet Activation Markers in Pediatric Cardiac Surgery
In this study the focus will be on correlating the levels of platelet activation markers (proteins that are released when blood cells are activated)to the duration of cardiopulmonary bypass, temperature during cardiopulmonary…
- Trial name
- NCT00450151
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Weill Medical College of Cornell University
Osteoarthritis (OA) of the Hip, Avascular Necrosis of Bone of Hip
MDR Vivacit-E Elevated Study
The main objective of this study is to confirm long-term safety, performance, and clinical benefits of the Vivacit-E Vitamin E HXLPE Elevated Liners when used in primary total hip arthroplasty.
- Conditions
- Osteoarthritis (OA) of the Hip, Avascular Necrosis of Bone of Hip, Protrusio Acetabuli, Traumatic Arthropathy-Hip
- Trial name
- NCT07150221
- Testing
- Vivacit-E Elevated Liners
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Zimmer Biomet
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
BACKGROUND/OBJECTIVE: Quantitative urine screening for mucopolysaccharides (MPS) has been the primary method for detecting mucopolysaccharidoses in children.
- Trial name
- NCT01707433
- Testing
- Enzyme testing
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Children's Hospitals and Clinics of Minnesota
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Achondroplasia
Radiological Analysis on Patients With Achondroplasia Disorder
Achondroplasia is a genetic disorder characterized by disproportionate short stature. It affects about 1 in 2500 live births in the world.
- Trial name
- Achondroplasia (NCT04184817)
- Testing
- Collection of clinical data registered in Easily software
- Where
- France
- Volunteers
- 60 people
- Sponsor
- Hospices Civils de Lyon
Food Allergy, Loeys-Dietz Syndrome
Natural History and Genetics of Food Allergy and Related Conditions
Background: About 15 million Americans have a food allergy. Because there are no cures or effective prevention or treatment for food allergies, researchers want to learn more about them.
- Conditions
- Food Allergy, Loeys-Dietz Syndrome, Atopic Dermatitis, Eosinophilic Esophagitis
- Trial name
- NCT02504853
- Where
- United States
- Volunteers
- 1,800 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Achondroplasia
A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia
This is a multicenter, non-randomized, open-label, phase I/II study in children with ACH. This study will start with a dose escalation of ABSK061 in children with ACH to evaluate the safety, tolerability, PK, and efficacy.
- Trial name
- NCT07297875
- Testing
- ABSK061
- Where
- 7 sites in China
- Volunteers
- 110 people
- Sponsor
- Abbisko Therapeutics Co, Ltd
Syringomyelia, Type I Arnold Chiari Malformation
Genetic Analysis of the Chiari I Malformation
The purpose of this study is to better understand the genetic factors related to the Chiari I malformation. In people with this abnormality, the lower part of the skull is smaller than normal.
- Trial name
- NCT00004738
- Where
- 2 sites in United States, Russia
- Volunteers
- 152 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Slipped Capital Femoral Epiphyses
"Gait and Balance in Patients With SCFE"
Slipped Capital Femoral Epiphysis (SCFE) treated with one screw in situ fixation results in an altered gait. It is thought that the protraction of the pelvis at the affected side, seen in patients with unilateral stable SCFE, is…
- Trial name
- NCT03326531
- Testing
- Not applicable for this study
- Where
- Netherlands
- Volunteers
- 25 people
- Sponsor
- Maastricht University Medical Center
Fibrous Dysplasia of Bone
Effect of Risedronate on Bone Morbidity in Fibrous Dysplasia of Bone
This trial is intended to test the efficacy of an oral bisphosphonate (risedronate) to decrease bone pain and improve radiological aspect in fibrous dysplasia of bone.
- Trial name
- PROFIDYS (NCT00445575)
- Testing
- risedronate
- Where
- 8 sites in Belgium, France and 2 more
- Volunteers
- 80 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Achondroplasia
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with…
- Trial name
- ACH (NCT02055157)
- Testing
- BMN 111
- Where
- 9 sites in United States, Australia and 2 more
- Volunteers
- 35 people
- Sponsor
- BioMarin Pharmaceutical