Clinical trials: Syndrome X, open to volunteers
Fragile X Syndrome, Autism Spectrum Disorder
Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders
Fragile X Syndrome (FXS) is a complex neurodevelopmental disorder caused by a mutation on the X chromosome. Scientists have investigated FXS extensively in both humans and animals.
- Conditions
- Fragile X Syndrome, Autism Spectrum Disorder, Autistic Disorder, Asperger Syndrome
- Trial name
- ENTRAIN (NCT06227780)
- Testing
- Alpha Auditory Entrainment
- Where
- United States
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Neonatal Respiratory Distress Syndrome, PreTerm Neonate
MINImising Total Radiation EXposure in Preterm Infants
Being born too early (preterm birth) is the leading cause of death in children world-wide. In Australia, 97% of very preterm babies who are admitted to Neonatal Intensive Care Units need breathing support after birth to survive.
- Trial name
- MINI T-Rex (NCT06975189)
- Testing
- Lung ultrasound group, Standard Care
- Where
- Australia
- Volunteers
- 180 people
- Sponsor
- Western Health, Australia
Fragile X Syndrome (FXS), Autism Spectrum Disorder
Group CBT in Adolescents With Fragile X Syndrome and in Adolescents With Autism Spectrum Disorder
Fragile X Syndrome (FXS) is a rare genetic syndrome, caused by a mutation in the FMR1 gene located on the X chromosome. It is considered the leading hereditary cause of intellectual disability (ID) and the primary cause of Autism…
- Trial name
- NCT06677866
- Testing
- Cognitive behavioral therapy, Waiting List
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Bambino Gesù Hospital and Research Institute
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)
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
Rett Syndrome
Lamivudine as a Novel Clinical Effort for Rett Syndrome
Rett syndrome (RTT) is a rare genetic neurodevelopmental disorder caused primarily by mutations in the MECP2 gene, leading to progressive impairments in motor function, communication, and behavior following an initial period of…
- Trial name
- LANCE (NCT07640503)
- Testing
- Lamivudine
- Where
- Brazil
- Volunteers
- 10 people
- Sponsor
- Maria Denise Fernandes Carvalho de Andrade
Rett Syndrome
A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).
- Trial name
- NCT07503444
- Testing
- fenfluramine hydrochloride
- Where
- 40 sites in United States, Belgium and 10 more
- Volunteers
- 200 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Rett Syndrome
A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome
This study will examine the potential efficacy and safety of Rett-T for core motor deficits of Rett syndrome, and will explore biological markers of safety and treatment response.
- Trial name
- NCT04041713
- Testing
- Rett-T
- Where
- Canada
- Volunteers
- 35 people
- Sponsor
- Holland Bloorview Kids Rehabilitation Hospital
Diabetic Microvascular Angina
Compound Danshen Dripping Pill for Diabetic Microvascular Angina
To investigate the efficacy and safety of Compound Danshen Dripping Pill in patients with diabetic microvascular angina pectoris.
- Trial name
- MVA (NCT06727890)
- Testing
- Danshen Dripping Pill
- Where
- 3 sites in China
- Volunteers
- 162 people
- Sponsor
- Qilu Hospital of Shandong University
Severe Combined Immunodeficiency (SCID), Leaky SCID
Natural History Study of SCID Disorders
This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions.
- Conditions
- Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis
- Trial name
- NCT01186913
- Where
- 44 sites in United States, Canada
- Volunteers
- 690 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Fragile X Syndrome (FXS), Creatine Transporter Deficiency
Optical Imaging in X-linked Disorders.
Fragile X syndrome (FXS, OMIM #300624) and Creatine Transporter Deficiency (CTD, #300352) are the two most common causes of X-linked intellectual disability.
- Trial name
- IMAGINER (NCT06868979)
- Testing
- Clinical assessment, Parental questionnaires, Cognitive assessment
- Where
- France
- Volunteers
- 118 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Tuberculosis (TB), Tuberculosis Infection, Latent
Screening With Portable X-rays for Rapid Recognition of TB
SPOT-TB (Screening with Portable X-rays for rapid recOgniTion of TB) is a prospective study evaluating a community-based tuberculosis (TB) screening strategy for migrant populations in New York City.
- Conditions
- Tuberculosis (TB), Tuberculosis Infection, Latent, HIV (Human Immunodeficiency Virus)
- Trial name
- SPOT-TB (NCT07737275)
- Testing
- Mobile chest radiography for tuberculosis screening
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Neonatal Pneumonia, Respiratory Distress Syndrome
Chest X-Ray Versus Lung Ultrasound in Neonatal Pneumonia
This prospective observational study aims to compare the diagnostic accuracy of chest X-ray versus lung ultrasound in the diagnosis of neonatal pneumonia among neonates admitted to the Neonatal Intensive Care Unit of Assiut…
- Conditions
- Neonatal Pneumonia, Respiratory Distress Syndrome, Pneumonia Neonatal
- Trial name
- NCT07807514
- Volunteers
- 100 people
- Sponsor
- Assiut University
Chronic Granulomatous Disease, Infection
NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
Background: CGD is caused by a gene mutation. For people with CGD, their cells cannot kill germs well, so they can get frequent or life-threatening infections.
- Trial name
- NCT05189925
- Testing
- gp91 Grans
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Urea Cycle Disorder, Organic Acidemia
Systemic Biomarkers of Brain Injury From Hyperammonemia
Ammonia is a waste product of protein and amino acid catabolism and is also a potent neurotoxin. High blood ammonia levels on the brain can manifest as cytotoxic brain edema and vascular compromise leading to intellectual and…
- Conditions
- Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I
- Trial name
- NCT04602325
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Children's National Research Institute
Angina Pectoris, Microvascular Angina
Efficacy of Targeted Medical Therapy in Angina and Nonobstructive Coronary Arteries
The goal of this clinical trial is to learn if targeted medical therapy will improve symptoms and quality of life in patients with angina and non-obstructive coronary arteries compared to placebo, after the underlying cause of…
- Conditions
- Angina Pectoris, Microvascular Angina, Vasospastic Angina, Myocardial Bridge of Coronary Artery
- Trial name
- MVP-ANOCA (NCT06424834)
- Testing
- Amlodipine, Nebivolol
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Stanford University
Relapsed/Refractory Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS)
CLN-049 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
CLN-049-001 is a Phase 1/2, open-label, multicenter, first-in-human trial of CLN-049 in patients with Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
- Conditions
- Relapsed/Refractory Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), TP53-mutated MDS and AML
- Trial name
- NCT05143996
- Testing
- CLN-049
- Where
- 17 sites in United States
- Volunteers
- 275 people
- Sponsor
- Cullinan Therapeutics Inc.
Brain Disorders, Aicardi Syndrome
Brain Development Research Program
Dr. Elliott Sherr and his collaborators at University of California, San Francisco (UCSF) are studying the genetic causes of disorders of cognition and epilepsy, in particular disorders of brain development that affect the corpus…
- Trial name
- NCT00305305
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- University of California, San Francisco
Dyskeratosis Congenita, Telomere Disease
Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD)
The purpose of this study is to see if a vitamin-like substance called quercetin is safe for people who have a rare condition called Dyskeratosis congenita (DC) or telomere biology disorders (TBD).
- Trial name
- NCT07628972
- Testing
- Quercetin
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Rett Syndrome, Family Caregivers
Adapting a Psychosocial Support Intervention for the Rett Syndrome Community
This study seeks to adapt Caregiver Speaks, a psychosocial supportive intervention delivered through Facebook, for parent caregivers of children living with Rett Syndrome.
- Conditions
- Rett Syndrome, Family Caregivers, Social Support, Psychological Support
- Trial name
- NCT07730112
- Testing
- Those who have patients prescribed Daybue, Caregiver Speaks + Daybue
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Colorado State University