Clinical trials: Deformity; Bone
Adrenal Hyperplasia, Congenital, Congenital Hypothyroidism
Clinical Evaluation of NeoPlex4 Assay and NeoPlex System
The purpose of this study is to assess the agreement of clinical performance between the proposed NeoPlex 4 assay and NeoPlex System and the comparator devices in clinical use in newborn screening programs for detection of T4…
- Conditions
- Adrenal Hyperplasia, Congenital, Congenital Hypothyroidism, Cystic Fibrosis
- Trial name
- NCT01488721
- Where
- 3 sites in United States
- Volunteers
- 7,462 people, healthy volunteers welcome
- Sponsor
- Luminex Corporation
Knee Pain Chronic, Osteoarthritis, Knee
Persona OsseoTi Keel Compatibility Study (Total Knee Arthroplasty)
The main objective of the study is to evaluate the safety, performance and clinical benefits of the Persona implant and its instrumentation in primary total knee arthroplasty
- Conditions
- Knee Pain Chronic, Osteoarthritis, Knee, Rheumatoid Arthritis, Traumatic Arthritis
- Trial name
- NCT05787821
- Testing
- Total Knee Arthroplasty
- Where
- 16 sites in United States
- Volunteers
- 760 people
- Sponsor
- Zimmer Biomet
X-Linked Hypophosphatemia
Study of Longitudinal Observation for Patient With X-linked Hypophosphatemic Rickets/Osteomalacia in Collaboration With Asian Partners
Through observation of patients with X-linked hypophosphatemic rickets/osteomalacia (XLH) for up to 10 years, the study intends to collect data that allow achievement of the following objectives: 1.
- Trial name
- SUNFLOWER (NCT03745521)
- Testing
- no intervention
- Where
- Japan
- Volunteers
- 226 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Osteogenesis Imperfecta
Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta
The primary objective of this study is to evaluate the effect of romosozumab treatment for 12-months compared with bisphosphonate(s) on the number of clinical fractures at 12-months; the number of any fractures at 12-months and…
- Trial name
- NCT05972551
- Testing
- Romosozumab, Bisphosphonate
- Where
- 65 sites in United States, Australia and 16 more
- Volunteers
- 111 people
- Sponsor
- Amgen
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum
Noonan Syndrome
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
The purpose of this study in children with Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment.
- Trial name
- NCT06668805
- Testing
- Vosoritide Injection
- Where
- 38 sites in United States, Australia and 5 more
- Volunteers
- 30 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
A Study to Learn About Recifercept in Patients With Achondroplasia
The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs.
- Trial name
- NCT05659719
- Testing
- Recifercept
- Where
- United States
- Volunteers
- 248 people
- Sponsor
- Pfizer
Advanced Cancer
A Study of LY3200882 and Pembrolizumab in Participants With Advanced Cancer
The main purpose of this study is to see if the drug LY3200882 which is an inhibitor of transforming growth factor-β (TGFβ) receptor 1 in combination with pembrolizumab is safe and effective in participants with cancer that has…
- Trial name
- NCT04158700
- Testing
- LY3200882, Pembrolizumab
- Where
- 6 sites in United States, France and 1 more
- Sponsor
- Eli Lilly and Company
Achondroplasia
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial.
- Trial name
- AttaCH (NCT05929807)
- Testing
- TransCon CNP
- Where
- 19 sites in United States, Australia and 10 more
- Volunteers
- 140 people
- Sponsor
- Ascendis Pharma Growth Disorders A/S
Acute-graft-versus-host Disease, Steroid Refractory Acute Graft Versus Host Disease
Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)
An Early Access Program for patients with steroid refractory acute GvHD after hematopoietic stem cell transplantation. This Program is available for female and male who are recipients of allogenic HSCT and who have been newly…
- Conditions
- Acute-graft-versus-host Disease, Steroid Refractory Acute Graft Versus Host Disease, Graft-versus-host-disease, Graft Vs Host Disease
- Trial name
- NCT03172455
- Testing
- Glassia
- Sponsor
- Impatients N.V. trading as myTomorrows
Exostoses, Multiple Hereditary
An Efficacy and Safety Study of Palovarotene for the Treatment of MO
This is a randomized, double-blind, placebo-controlled study comparing the safety and efficacy of 2 dosage regimens of palovarotene versus placebo in preventing disease progression in pediatric subjects with multiple…
- Trial name
- MO-Ped (NCT03442985)
- Testing
- Palovarotene 2.5 mg, Palovarotene 5.0 mg
- Where
- 31 sites in United States, Australia and 10 more
- Volunteers
- 193 people
- Sponsor
- Clementia Pharmaceuticals Inc.
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03301168
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 10 sites in United States
- Volunteers
- 120 people
- Sponsor
- Bellicum Pharmaceuticals
Osteogenesis Imperfecta
Post-market Observational Study on JTIN Telescopic Nail in Osteogenesis Imperfecta Pediatric Patients
This study has been planned as part of the Orthofix Srl post-market active surveillance plan for the collection of data on both the clinical performance and the safety profile of the JTIN.
- Trial name
- JTIN (NCT05612139)
- Testing
- JTIN implantation surgery
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Orthofix s.r.l.
Primary Osteoarthritis, Secondary Osteoarthritis
Prospective Study for the Evaluation of the Cementless Anatomic Femoral Stem Minimax -Launay
This is a post-marketing surveillance on MiniMAX Stem
- Conditions
- Primary Osteoarthritis, Secondary Osteoarthritis, Traumatic Arthritis, Rheumatoid Arthritis
- Trial name
- NCT06012656
- Testing
- MiniMAX
- Where
- France
- Volunteers
- 100 people
- Sponsor
- Medacta International SA
Plagiocephaly, Positional, Plagiocephaly, Nonsynostotic
Effectiveness of Ottobock Helmet Therapy in Infants With Plagiocephaly, Brachycephaly or Combination of Both
The objective of the research is to measure the effectiveness of the Ottobock helmet, an innovative 3D-printed cranial orthosis used to correct infant cranial positional deformation: plagiocephaly, brachycephaly or combination of…
- Conditions
- Plagiocephaly, Positional, Plagiocephaly, Nonsynostotic, Brachycephaly
- Trial name
- NCT05154175
- Testing
- Ottobock helmet
- Where
- 12 sites in France
- Volunteers
- 452 people
- Sponsor
- Otto Bock France SNC
X-linked Hypophosphatemia
A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
A first-in-human study of KK8123 in adults with X-linked hypophosphatemia.
- Trial name
- NCT06525636
- Testing
- KK8123
- Where
- 10 sites in United States, Canada and 3 more
- Volunteers
- 39 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Atopic Dermatitis
A Study of Crisaborole Ointment 2%; Crisaborole Vehicle; TCS and TCI in Subjects Aged ≥ 2 Years, With Mild-moderate AD
This 4-week study will evaluate the safety and efficacy of crisaborole ointment 2%; crisaborole vehicle; topical corticosteroid and topical calcineurin inhibitor, applied twice daily (BID) in subjects who are at least 2 years of…
- Trial name
- NCT03539601
- Testing
- Crisaborole ointment, 2%, Hydrocortisone butyrate cream, 0.1%, Pimecrolimus cream, 1%
- Where
- 32 sites in United States, Germany and 5 more
- Volunteers
- 237 people
- Sponsor
- Pfizer
X-linked Hypophosphatemia (XLH)
Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia
The purpose of this study is to better understand how X-linked hypophosphatemia (XLH) affects the body and daily life. Phosphate levels are critical in managing XLH, we aim to study how these levels change in patients taking…
- Trial name
- IBIS XLH (NCT07607600)
- Testing
- No treatment given
- Where
- 23 sites in United States, Australia and 6 more
- Sponsor
- Novo Nordisk A/S
Joint Disease
Post Market Clinical Follow-Up Study Protocol for PROFEMUR® TL Modular Femoral Stems
Sponsor is conducting this post market clinical follow-up (PMCF) study to evaluate the safety and efficacy of its total hip arthroplasty (THA) marketed in the European Union (EU) .
- Trial name
- NCT02239783
- Testing
- total hip arthroplasty implant
- Where
- Canada
- Volunteers
- 143 people
- Sponsor
- MicroPort Orthopedics Inc.
Cancer
Fentanyl Sublingual Spray in Treating Patients With Breakthrough Cancer Pain
This is a phase III, randomized, double-blind, placebo-controlled, multicenter study of the clinical response to fentanyl sublingual spray as a treatment for breakthrough cancer pain.
- Trial name
- NCT00538850
- Testing
- Fentanyl sublingual spray
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- INSYS Therapeutics Inc