Clinical trials: Development Disorders
Achondroplasia
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.
- Trial name
- NCT04085523
- Testing
- TransCon CNP
- Where
- 15 sites in United States, Australia and 6 more
- Volunteers
- 57 people
- Sponsor
- Ascendis Pharma A/S
Basal Cell Nevus Syndrome, Gorlin's Syndrome
Trial Comparing the Effects of Intermittent Vismodegib vs. PDT in Patients With Multiple Basal Cell Carcinomas
The purpose of this study is to evaluate and compare the safety and efficacy of intermittent vismodegib and of Photodynamic Therapy (PDT).
- Trial name
- Vismodegib (NCT01556009)
- Testing
- Vismodegib, Aminolevulinic acid %20 topical solution
- Where
- 3 sites in United States
- Volunteers
- 24 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Covid19, Intellectual Disability
Supporting the Health and Well-being of Children With Intellectual and Developmental Disability During COVID-19 Pandemic
The primary goal of this project is to identify the best messaging and implementation strategies to maximize SARS-CoV-2 testing for children with intellectual and developmental disabilities (IDD) and their teachers to help ensure…
- Conditions
- Covid19, Intellectual Disability, Developmental Disability, Child Development Disorder
- Trial name
- NCT04565509
- Testing
- Standard Messaging, Enhanced Messaging, COVID-19 Testing
- Where
- 3 sites in United States
- Volunteers
- 1,143 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Adolescent Behavior, Intellectual Disability
Promotion of Physical Activity in Adolescents With Intellectual and Developmental Disabilities
The objective of this study is to compare the effect of two strategies to increase MVPA in adolescents with intellectual and developmental disabilities (IDD): a single level intervention delivered to the adolescent only, and a…
- Conditions
- Adolescent Behavior, Intellectual Disability, Down Syndrome, Physical Activity
- Trial name
- NCT03684512
- Testing
- Group Exercise Sessions, Individual Support Sessions, Facebook Group
- Where
- United States
- Volunteers
- 116 people
- Sponsor
- University of Kansas
Asthma
Role of the Isomerase Pin-1 in the Development and Treatment of Asthma
Pin1 is activated in asthmatic airways, increasing cytokine mRNA stability and eosinophil survival. This study is designed to test whether the Pin1 enzyme regulates TLR/IL-1R signal pathways in multiple cells in asthma.
- Trial name
- Pin1 (NCT01691612)
- Testing
- installation of D. pteronyssinus allergens
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Brigham and Women's Hospital
Autism Spectrum Disorder
Safety, Tolerability and Pharmacokinetics Study of STP1 in a Subgroup of Patients With Autism Spectrum Disorder (ASD)
The main purpose of this study is to evaluate safety and tolerability in a subgroup of patients with Autism Spectrum Disorder (ASD).
- Trial name
- NCT04644003
- Testing
- STP1
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Stalicla SA
Obesity, Physical Activity
Positive Psychology for Physical Activity Promotion
This study is for physically inactive adults. Participants will be enrolled in the Positive Psychology and Physical Activity intervention designed to help them overcome barriers to physical activity, based on the principals of…
- Trial name
- PPPA (NCT03826173)
- Testing
- PPPA, PA
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- Brown University
Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
Bortezomib and Combination Chemotherapy in Treating Younger Patients With Recurrent, Refractory, or Secondary Acute Myeloid Leukemia
This phase II trial is studying the side effects and best dose of bortezomib and to see how well it works when given together with combination chemotherapy in treating younger patients with recurrent, refractory, or secondary…
- Conditions
- Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1)
- Trial name
- NCT00666588
- Testing
- idarubicin, cytarabine, bortezomib
- Where
- 82 sites in United States, Australia and 1 more
- Volunteers
- 52 people
- Sponsor
- National Cancer Institute (NCI)
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
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Vorinostat, Azacitidine, and Gemtuzumab Ozogamicin for Older Patients With Relapsed or Refractory AML
The purpose of this study is to test the safety of vorinostat (Zolinza) and azacitidine (Vidaza) when combined with gemtuzumab ozogamicin (GO) at different dose levels.
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT00895934
- Testing
- vorinostat, gemtuzumab ozogamicin, azacitidine
- Where
- 4 sites in United States
- Volunteers
- 52 people
- Sponsor
- National Cancer Institute (NCI)
Aggression, Attention Deficit-Hyperactivity
Metabolic Effects of Antipsychotics in Children
The project aims to describe and compare the outcome of 12 weeks of prospective, randomized treatment with olanzapine, risperidone or aripiprazole on insulin action in skeletal muscle, liver and adipose tissue, abdominal fat…
- Conditions
- Aggression, Attention Deficit-Hyperactivity, Oppositional Defiant Disorder, Pervasive Development Disorders
- Trial name
- MEAC (NCT00205699)
- Testing
- risperidone, olanzapine, aripiprazole
- Where
- United States
- Volunteers
- 144 people
- Sponsor
- Washington University School of Medicine
Autism Spectrum Disorder
Providing Accessible Diagnostic Evaluations and Psychoeducation for Autism Spectrum Disorder in Rural Southwest Virginia
This study will be the pilot/feasibility phase of a future clinical trial. The proposed feasibility and pilot study aims to bridge the barriers to diagnosis by providing easy-to-access assessment for free, through a mobile unit…
- Trial name
- NCT05822921
- Testing
- In-person Psychoeducation (IP), Telehealth Psychoeducation (TH), Psychoeducation as Usual (PAU)
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- Virginia Polytechnic Institute and State University
Desmoplastic/Nodular Medulloblastoma, Medulloblastoma
Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed, Non-metastatic Desmoplastic Medulloblastoma
This phase II trial studies how well combination chemotherapy works in treating younger patients with newly diagnosed, non-metastatic desmoplastic medulloblastoma.
- Conditions
- Desmoplastic/Nodular Medulloblastoma, Medulloblastoma, Medulloblastoma With Extensive Nodularity, Nevoid Basal Cell Carcinoma Syndrome
- Trial name
- NCT02017964
- Testing
- Carboplatin, Cognitive Assessment, Cyclophosphamide
- Where
- 173 sites in United States, Australia and 2 more
- Volunteers
- 26 people
- Sponsor
- Children's Oncology Group
Non-small Cell Lung Cancer
Erlotinib Therapy and Subsequent Development of Mechanisms of Secondary Resistance in Patients With NSCLC
Erlotinib is a drug which targets non small cell lung cancer with a genetic change (mutation) in the epidermal growth factor receptor (EGFR).
- Trial name
- NCT00997334
- Testing
- Erlotinib
- Where
- 2 sites in United States
- Volunteers
- 60 people
- Sponsor
- David M. Jackman, MD
Brain Ischemia
Strategy for Adequate Blood Pressure Lowering in the Patients With Intracranial Atherosclerosis
To develop adequate blood pressure (BP) lowering strategy after subacute ischemic stroke patients with symptomatic severe intracranial atherosclerosis.
- Trial name
- STABLE-ICAS (NCT01104311)
- Testing
- Aggressive BP lowering, modest blood pressure lowering
- Where
- 16 sites in South Korea
- Volunteers
- 132 people
- Sponsor
- Asan Medical Center
Stroke
New Tool to Enhance Post-stroke Upper Extremity Disability
Individuals with low socioeconomic status (SES) are more likely to have a stroke, more disabled at 3 months, and less likely to be independently ambulatory.
- Trial name
- NCT05277389
- Testing
- START - Startle Adjuvant Rehabilitation Therapy
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Arizona State University
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.
Opioid-use Disorder
Digital Therapeutic Development of Virtual Cognitive-Affective Training for Opioid Use Disorder
This is a Phase 1 trial that aims to establish the safety of MORE-VR, as well as to collect feasibility, usability, and engagement data, for patients receiving medications for opioid use disorder (mOUD).
- Trial name
- NCT05034276
- Testing
- MORE-VR
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- University of Utah
Cancer, Inflammatory Disease
Translational Development of Photon-counting CT Imaging
Using an investigational CT scanner which uses a new type of detector to capture X-rays, (a photon counting CT detector), the goal of this experimental study is to compare conventional CT images to CT images using the photon…
- Conditions
- Cancer, Inflammatory Disease, Vascular Diseases, Metabolic Bone Disease
- Trial name
- NCT03878134
- Testing
- PHOTON COUNTING CT Scanner
- Where
- United States
- Volunteers
- 67 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Myeloma-Multiple, Myeloma, Plasma-Cell
T Cells Expressing a Novel Fully-Human Anti-BCMA CAR for Treating Multiple Myeloma
Background: Multiple myeloma is a cancer of the blood plasma cells. It usually becomes resistant to standard treatments. Researchers have developed a procedure called gene therapy.
- Trial name
- NCT03602612
- Testing
- Cyclophosphamide, Fludarabine, Anti-B Cell Maturation Antigen (BCMA) chimeric antigen receptors (CARs) T cells
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Cancer Institute (NCI)
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