Clinical trials: Defect, Congenital
Sickle Cell Disease
Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease
The aim of this study is to evaluate the overall safety and feasibility of using haploidentical or one antigen mismatch unrelated hematopoietic stem cell transplant (HSCT) for adult patients with severe sickle cell disease (SCD)…
- Trial name
- NCT02678143
- Testing
- Alemtuzumab, Cyclophosphamide, Mycophenolate mofetil
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Washington University School of Medicine
Cystic Fibrosis
Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation
The purpose of this study is to investigate the safety, tolerability and pharmacokinetics of N6022, and to obtain descriptive information on the effect of N6022 on biomarkers of CFTR function and inflammation in adult cystic…
- Trial name
- SNO-1 (NCT01746784)
- Testing
- N6022, Normal saline
- Where
- 17 sites in United States
- Volunteers
- 66 people
- Sponsor
- Nivalis Therapeutics, Inc.
Gout, Arthritis, Gouty
Phase 2 to Assess Efficacy and Safety in AR882 Alone or in Combination With Allopurinol in Patients With Tophaceous Gout
This study will assess the serum urate lowering effect, tophi reduction, and safety of AR882 alone and in combination with allopurinol in patients with tophaceous gout at two doses compared to allopurinol over 24 weeks.
- Conditions
- Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
- Trial name
- NCT05253833
- Testing
- AR882 Dose 1, AR882 Dose 2, Allopurinol Tablet
- Where
- 12 sites in United States, New Zealand and 1 more
- Volunteers
- 42 people
- Sponsor
- Arthrosi Therapeutics
Alzheimer's Disease, Multiple Sclerosis
A Biospecimen and Clinical Data Study on Patients for Drug & Biomarker Discovery
The purpose of this research project is to collect and store blood samples and clinical data. Researchers can then use the stored samples in future studies.
- Conditions
- Alzheimer's Disease, Multiple Sclerosis, Parkinson's Disease, Huntington's Disease
- Trial name
- NCT01592552
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Arteriovenous Fistula, Hemodialysis
Effectiveness of a Structured Intervention on the Development of Self-Care Behaviors With AVF in HD Patients
End stage renal disease patients (ESRD) should be educated to take care of their arteriovenous fistula (AVF). Educational programs should have clear objectives and the interventions should be well defined.
- Trial name
- SISC-AVF (NCT03830658)
- Testing
- Structured Intervention on Self-care with AVF (SISC-AVF)
- Where
- Portugal
- Volunteers
- 89 people
- Sponsor
- Instituto Portugues de Oncologia, Francisco Gentil, Porto
Charcot-Marie-Tooth Disease Type 1A
Clinical Trial Evaluating the Impact of an Intensive Rehabilitation Program Combined With Tendon Vibratory Stimulation on Functional Balance in Individuals With Charcot-Marie-Tooth Disease Type 1A
Charcot-Marie-Tooth (CMT) disease is caused by a genetic abnormality involving the PMP22 gene, resulting in demyelination of the peripheral nerves.
- Trial name
- EQUIVIB-CMT (NCT07726043)
- Testing
- Multidisciplinary rehabilitation program
- Where
- France
- Volunteers
- 30 people
- Sponsor
- UGECAM PACA et Corse
Craniosynostosis
Osteogenic Profiling of Tissue From Children With Craniosynostosis
The primary objectives of this study are to procure human temporalis muscle, subcutaneous adipose (fat), and bone tissue samples from children with craniosynostosis, to grow cells from these tissues in vitro, to evaluate the…
- Trial name
- NCT00773643
- Testing
- biopsy
- Where
- 2 sites in United States
- Volunteers
- 29 people
- Sponsor
- University of Pittsburgh
Neurofibromatoses, Neurofibromatosis 1
Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing
The aims of this study are to compare the effect and durability of two stress and symptom management programs tailored for patients with neurofibromatosis on quality of life and psychosocial functioning.
- Conditions
- Neurofibromatoses, Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis
- Trial name
- NCT03406208
- Testing
- Stress and Symptom Management Program 1, Stress and Symptom Management Program 2
- Where
- United States
- Volunteers
- 228 people
- Sponsor
- Massachusetts General Hospital
Venous Malformation, Low Flow, Venous Malformations
Acetylsalicylic Acid Versus Placebo as an add-on Treatment to Local Non-steroidal Anti-inflammatory Drug for the Management of Thrombotic Episodes in Superficial Venous Malformations in Children Aged 6 to 17 Years.
Superficial venous malformations (SVMs) are rare congenital anomalies that present as bluish masses. These masses may be focal, with limited skin involvement, or segmental, with more extensive involvement.
- Trial name
- ASPIRIN (NCT07663825)
- Testing
- AAS at anti-inflammatory doses from 3 days to 14 days., Application of Diclofenac gel 1% twice a day
- Where
- 6 sites in France
- Volunteers
- 34 people
- Sponsor
- University Hospital, Tours
Lactose Intolerance
Incidence of Lactose Intolerance Among Self-reported Lactose Intolerant People
This is a 3-sites, double-blinded, randomized, 2X2 cross-over study aiming to compare effects of milk containing only A2 type beta casein versus milk containing both A1 and A2 beta casein proteins on the gastrointestinal symptoms…
- Trial name
- NCT02878876
- Testing
- Oral consumption of milk with sequence A1-A2, Oral consumption of milk with sequence A2-A1
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- a2 Milk Company Ltd.
Hemophilia
Imaging of Arthropathy in Boys With Hemophilia in China
Hemophilia is a genetic condition characterized by marked phenotypic heterogeneity. Bleeding into a joint is the single most important risk factor for the development of hemophilic arthropathy (HA).
- Trial name
- NCT03914716
- Testing
- Study group
- Where
- China
- Volunteers
- 49 people
- Sponsor
- Andrea Doria
Tourette's Syndrome
Hypersensitivity in Tourette Syndrome
This study will investigate how the sensitivity to touch and smell in patients with Tourette syndrome (TS) may differ from that of people without TS.
- Trial name
- NCT00368433
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Fragile X Syndrome, Behavior
An Investigation of Psilocybin on Behavioural and Cognitive Symptoms of Adults With Fragile X Syndrome
Diverse symptomatology makes Fragile X Syndrome (FXS) difficult to treat, and currently there are no approved prevention or treatment methods for FXS.
- Conditions
- Fragile X Syndrome, Behavior, Cognitive Dysfunction
- Trial name
- NCT05832255
- Testing
- Psilocybin, 1.5 mg
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- Nova Mentis Life Science Corp
Phenylketonurias
Effects of Synergy on Nutrient Intake and Acceptability in Phenylketonuria (PKU)
Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine…
- Trial name
- ESSENTIAL (NCT03777826)
- Testing
- PKU Synergy
- Where
- 3 sites in Germany, Netherlands
- Volunteers
- 13 people
- Sponsor
- Nutricia Research
Infantile Colic
The Effect of Chiropractic Treatment of Infantile Colic
Infantile colic is condition that affects more than 10% of babies and their families. The reason, and hence proper treatment, for this condition is unknown and many causes have been suggested.
- Trial name
- NCT02595515
- Testing
- Chiropractic treatment, Visit without active treatment
- Where
- Denmark
- Volunteers
- 186 people
- Sponsor
- Nordic Institute of Chiropractic and Clinical Biomechanics
Atopic Dermatitis
Study of Equivalence of Generic Pimecrolimus Cream 1% and Elidel® 1% in Subjects With Mild to Moderate Atopic Dermatitis
The purpose of this study is to compare the safety and efficacy profiles of Watson Laboratories, Inc.'s Pimecrolimus Cream, 1% to those of Valeant Pharmaceuticals North America LLC's Elidel® (pimecrolimus) Cream, 1%, and to…
- Trial name
- NCT02791308
- Testing
- Pimecrolimus Cream 1% (Valeant), Vehicle cream, Pimecrolimus Cream, 1%
- Where
- 53 sites in United States
- Volunteers
- 587 people, healthy volunteers welcome
- Sponsor
- Actavis Inc.
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Ixabepilone in Treating Young Patients With Solid Tumors or Leukemia That Haven't Responded to Therapy
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: This phase I trial is studying the side effects and best dose of ixabepilone in treating young…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00030108
- Testing
- ixabepilone
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Cardiac Valve Disease
Prothrombin Complex Concentrate vs Fresh Frozen Plasma in Goal-directed Bleeding Management in Cardiac Surgery
The primary endpoint of this study is the completion time of hemostasis treatment when administered Fresh frozen plasma (FFP) and frozen powder coagulation factor concentrate (PCC) in goal-directed bleeding management for cardiac…
- Trial name
- NCT06144125
- Testing
- fresh frozen plasma, prothrombin complex concentrate
- Where
- South Korea
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Konkuk University Medical Center
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Donor T Cells After Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This pilot phase II trial studies how well giving donor T cells after donor stem cell transplant works in treating patients with hematologic malignancies.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT01839916
- Testing
- therapeutic allogeneic lymphocytes, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- University of Chicago
Huntington Disease
Multi-Modal Digital Monitoring of Disease Symptoms Huntington's Disease
The objective of the study is to validate the use of wearable sensors and digital health technologies for monitoring disease activity in Huntington's Disease (HD).
- Trial name
- BioDigit HD-02 (NCT07315984)
- Where
- 2 sites in United States
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- BioSensics