Clinical trials: Musculoskeletal Complication
- Condition Musculoskeletal Complication
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
McCune-Albright Syndrome
Arimidex in McCune Albright Syndrome
The primary objective of this study is to evaluate the safety and efficacy of anastrozole 1 mg given once daily in subjects with McCune-Albright Syndrome.
- Trial name
- NCT00055302
- Testing
- Arimidex 1 mg
- Where
- 7 sites in France, Germany and 2 more
- Volunteers
- 40 people
- Sponsor
- AstraZeneca
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Observational Prospective Study on Patients Treated With Norditropin®
This observational study is conducted globally. The aim of the study is to investigate the effectiveness and safety of real-life treatment with Norditropin®.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Adult Growth Hormone Deficiency, Genetic Disorder
- Trial name
- NCT00960128
- Testing
- somatropin
- Where
- 23 sites in Argentina, Belgium and 21 more
- Volunteers
- 21,249 people
- Sponsor
- Novo Nordisk A/S
X-linked Hypophosphatemia
Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
The objectives of the study are to: Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants Establish the safety profile of burosumab for the treatment of…
- Trial name
- NCT02163577
- Testing
- burosumab
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 52 people
- Sponsor
- Kyowa Kirin, Inc.
Turner's Syndrome
A Clinical Study to Assess the Efficacy and Safety of DA-3002
A study demonstrates the non-inferiority of DA-3002 when compared with Genotropin®.
- Trial name
- NCT01813630
- Testing
- DA-3002, Genotropin®
- Where
- South Korea
- Volunteers
- 58 people
- Sponsor
- Dong-A ST Co., Ltd.
Septic Shock, Acute Respiratory Distress Syndrome
CytOSorb TreatMent Of Critically Ill PatientS Registry
Registry intended to provide a data repository and reporting infrastructure for the surveillance of CytoSorb device use in real-world critical care settings, and to serve as an objective, comprehensive, and scientifically-based…
- Conditions
- Septic Shock, Acute Respiratory Distress Syndrome, Trauma, Rhabdomyolysis
- Trial name
- COSMOS (NCT05146336)
- Testing
- CytoSorb
- Where
- 28 sites in Austria, Germany and 4 more
- Volunteers
- 3,000 people
- Sponsor
- CytoSorbents, Inc
Turner Syndrome, Renal Insufficiency, Chronic
Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq
The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.
- Conditions
- Turner Syndrome, Renal Insufficiency, Chronic, Pituitary Diseases, Dwarfism
- Trial name
- OPTIMA (NCT00234533)
- Testing
- Somatropin (rDNA origin)
- Where
- 45 sites in Belgium, Czechia and 12 more
- Volunteers
- 251 people
- Sponsor
- Ipsen
X-linked Hypophosphatemia, Tumor-Induced Osteomalacia
Expanded Access to Burosumab
Individual patient expanded access requests may be considered for patients who have no other treatment options
- Trial name
- NCT03775187
- Testing
- Burosumab
- Sponsor
- Kyowa Kirin Co., Ltd.
Alopecia Areata, Depressive Episode
The Epidemiology, Management, and the Associated Burden of Related Conditions in Alopecia Areata
This study series consists of four related studies and aims to explore and describe many important elements of alopecia areata over three key areas: (1) the current epidemiology of alopecia areata, (2) the prevalence and…
- Conditions
- Alopecia Areata, Depressive Episode, Recurrent Depressive Disorder, Anxiety Disorders
- Trial name
- NCT04239521
- Testing
- Exposure of interest (studies 2 & 3).
- Where
- United Kingdom
- Volunteers
- 51,955 people
- Sponsor
- Momentum Data
X-Linked Hypophosphatemia
Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)
The primary objectives of this study are to: Assess the long-term safety of KRN23 subcutaneous (SC) administration in adult subjects with XLH Assess the proportion of subjects achieving serum phosphorus levels in the normal range…
- Trial name
- NCT02312687
- Testing
- KRN23
- Where
- 5 sites in United States
- Volunteers
- 20 people
- Sponsor
- Kyowa Kirin, Inc.
Autosomal Dominant Hypocalcemia
ADH1 and ADH2 Disease Monitoring Study (DMS)
A global, multi-center, Disease Monitoring Study (DMS) in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1) or Autosomal Dominant Hypocalcemia Type 2 (ADH2) designed to characterize ADH1 and ADH2 disease…
- Trial name
- CLARIFY (NCT05227287)
- Where
- 27 sites in United States, Australia and 11 more
- Volunteers
- 95 people
- Sponsor
- Calcilytix Therapeutics, Inc., a BridgeBio company
Rheumatoid Arthritis (RA), Systemic Lupus Erthematosus (SLE)
BAFF CAR-T Cells (LMY-922) for Treatment of Refractory Autoimmune Disease
Therapy with chimeric antigen receptor T (CAR-T) cells has demonstrated activity against refractory autoimmune disease, however not all disease responds or remains in response to CD19 targeted CAR-T cells.
- Conditions
- Rheumatoid Arthritis (RA), Systemic Lupus Erthematosus (SLE), Dermatomyositis (DM), Systemic Sclerosis (SSc)
- Trial name
- NCT07729995
- Testing
- LMY-922
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Luminary Therapeutics
Psychological Stress, Cardiopulmonary Bypass Surgery
CARINAE for Stress Relief in Perioperative Care
Preventing pre-surgical stress can help patients achieve positive outcomes on health and well-being. However, very few patients receive adequate stress relief support prior to a surgical procedure.
- Conditions
- Psychological Stress, Cardiopulmonary Bypass Surgery, Coronary Artery Bypass Surgery, Cardiac Valve Replacement
- Trial name
- CARINAE (NCT05184725)
- Testing
- SaMD CARINAE
- Where
- 5 sites in Italy, Netherlands and 1 more
- Volunteers
- 50 people
- Sponsor
- Adhera Health, Inc.
X-Linked Hypophosphataemia
A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH
This study aims to provide Health-related Quality of Life (HRQoL) data from children and adolescents with growing skeletons in the United Kingdom (UK), including those treated with burosumab or alternative XLH treatment, as part…
- Trial name
- NCT04819490
- Where
- 6 sites in United Kingdom
- Volunteers
- 32 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age
A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Paediatric Patients from Birth to Less than 1 Year of Age with X-linked Hypophosphatemia…
- Trial name
- NCT04188964
- Testing
- Burosumab
- Where
- 9 sites in Austria, France and 4 more
- Volunteers
- 16 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Antineutrophilic Cytoplasmic Antibody (ANCA)- Associated Vasculitis (AAV), Idiopathic Inflammatory Myositis (IIM)
A Phase 1 Study of FT819 in B-cell Mediated Autoimmune Disease
This is a phase 1 study designed to evaluate the safety, pharmacokinetics (PK), and anti-B-cell activity of FT819 following treatment with or without auxiliary medicinal product (AMP) in participants with moderate-to-severe…
- Conditions
- Antineutrophilic Cytoplasmic Antibody (ANCA)- Associated Vasculitis (AAV), Idiopathic Inflammatory Myositis (IIM), Systemic Sclerosis (SSc), Systemic Lupus Erythematosus (SLE)
- Trial name
- NCT06308978
- Testing
- FT819, Fludarabine, Cyclophosphamide
- Where
- 21 sites in United States, France and 2 more
- Volunteers
- 244 people
- Sponsor
- Fate Therapeutics
Growth Hormone Deficiency, Turner Syndrome
Evaluation of Convenience and Compliance of the Easypod™ Electronic Self-injector
This is an international, multicenter study involving children treated with Saizen®, a growth hormone, who will be trained to use easypod, a new electronic injector and will complete a questionnaire after 12 week of use.
- Conditions
- Growth Hormone Deficiency, Turner Syndrome, Chronic Renal Failure, Small for Gestational Age
- Trial name
- NCT00738205
- Volunteers
- 834 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Dwarfism, Pituitary, Turner Syndrome
Bone Mineral Density (BMD) in Adolescents With Growth Hormone Deficiency (GHD)
This study is a multicenter, open-label, postmarketing surveillance study. The substudy will collect information on BMD in adolescents and young adults with GHD or Turner syndrome who are completing GH treatment for statural…
- Trial name
- NCT00097526
- Volunteers
- 125 people
- Sponsor
- Genentech, Inc.
Turner Syndrome, Short Stature Homeobox Gene Mutation
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates.
- Conditions
- Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
- Trial name
- NCT07221851
- Testing
- Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
- Where
- 51 sites in United States, France and 5 more
- Volunteers
- 186 people
- Sponsor
- Ascendis Pharma A/S
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
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.