Clinical trials: Hereditary Sensory and Autonomic Neuropathy 3
Familial Dysautonomia
Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
The purpose of this placebo controlled interventional study is to collect preliminary data on administering dexmedetomidine in patients with Familial Dysautonomia (FD) during a rapid cessation of autonomic crisis.
- Trial name
- NCT06148311
- Testing
- Dexmedetomidine
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Dysautonomia, Familial, Baroreflex Failure Syndrome
Carbidopa for the Treatment of Excessive Blood Pressure Variability
The overall study objectives are to determine whether carbidopa (Lodosyn®) is safe and well tolerated and to assess whether it can inhibit catecholamine-induced paroxysmal hypertension and normalize or reduce the exaggerated…
- Trial name
- CarbiFD (NCT02553265)
- Testing
- Carbidopa Low-Dose, Carbidopa High-Dose
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- NYU Langone Health
Pancreatic Adenocarcinoma
9-ING-41 Combined With Retifanlimab, Plus Modified FOLFIRINOX for Patients With Advanced Pancreatic Adenocarcinoma (RiLEY)
This is a study of the combination of 9 ING-41 (elraglusib) and retifanlimab plus mFOLFIRINOX in patients with pancreatic cancer without prior systemic therapy for advanced disease.
- Trial name
- RiLEY (NCT06896188)
- Testing
- Retifanlimab, Chemotherapy, 9-ING-41
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Anwaar Saeed
Lymphoma
Study Evaluating Duvelisib in Japanese Subjects With Relapsed or Refractory Lymphoma
This study seeks to evaluate the safety and pharmacokinetics of duvelisib in Japanese participants with relapsed or refractory lymphoma.
- Trial name
- NCT02598570
- Testing
- duvelisib
- Where
- 3 sites in Japan
- Volunteers
- 7 people
- Sponsor
- AbbVie
Asthma
A Study to Evaluate the Safety, Tolerability and Absorption to the Blood After Administration of Single and Multiple Doses of AZD8154 in Healthy Participants.
This study is a Phase 1, first in human (FiH) study, consisting of 3 parts (Part 1, Part 2, and Part 3) in healthy male and female participants of non-childbearing potential, performed at a single study center.
- Trial name
- NCT03436316
- Testing
- AZD8154
- Where
- Germany
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Colorectal Cancer Metastatic
Influence of BRAF and PIK3K Status on the Efficacy of 5-Fluorouracil/Leucovorin/Oxaliplatin (FOLFIRI) Plus Bevacizumab or Cetuximab in Patients With RAS Wild-type Metastatic Colorectal Carcinoma and < 3 Circulating Tumor Cells (CTC)
The purpose of the study is to explore the influence of BRAF and PIK3K status on the efficacy of FOLFIRI plus Bevacizumab or Cetuximab, as first line therapy of patients with RAS wild-type metastatic colorectal carcinoma and < 3…
- Trial name
- VISNU-2 (NCT01640444)
- Testing
- FOLFIRI + bevacizumab, FOLFIRI + cetuximab
- Where
- Spain
- Volunteers
- 240 people
- Sponsor
- Spanish Cooperative Group for the Treatment of Digestive Tumours (TTD)
Arthritis, Osteoarthritis
Tanezumab in Osteoarthritis of the Knee
Test the efficacy and safety of 3 doses in Osteoarthritis of the knee in patients.
- Trial name
- NCT00733902
- Testing
- tanezumab
- Where
- 82 sites in United States
- Volunteers
- 697 people
- Sponsor
- Pfizer
Osteoarthritis
Tanezumab In Osteoarthritis Of The Hip Or Knee
The purpose of the study is to test the efficacy and safety of 2 doses of tanezumab compared to oxycodone CR and placebo in patients with osteoarthritis
- Trial name
- NCT00985621
- Testing
- tanezumab 10 mg, tanezumab 5 mg, oxycodone
- Where
- 112 sites in United States, Austria and 5 more
- Volunteers
- 614 people
- Sponsor
- Pfizer
Familial Dysautonomia
Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
This is a pilot open-label study to evaluate the feasibility of conducting a clinical trial using sublingual dexmedetomidine sublingual film to treat hyperadrenergic autonomic crises in patients with Familial Dysautonomia at…
- Trial name
- NCT06128356
- Testing
- Dexmedetomidine Sublingual
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- NYU Langone Health
Autoimmune Hemolytic Anemia
A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
The purpose of this study is to evaluate the safety and efficacy of parsaclisib administered orally to participants with autoimmune hemolytic anemia (AIHA) who have decreased hemoglobin and evidence of ongoing hemolysis that…
- Trial name
- NCT03538041
- Testing
- Parsaclisib
- Where
- 12 sites in United States, Austria and 2 more
- Volunteers
- 25 people
- Sponsor
- Incyte Corporation
Colorectal Cancer Metastatic, Advanced Non-Small Cell Lung Carcinoma
The Oncopanel Pilot (TOP) Study
The BCCA Oncopanel is a clinical assay being developed to determine genotype status of a prospectively defined set of genes. The purpose of this pilot study is to assess the feasibility and effect on clinical-decision-making of…
- Conditions
- Colorectal Cancer Metastatic, Advanced Non-Small Cell Lung Carcinoma, Advanced Melanoma, Gastrointestinal Stromal Tumors
- Trial name
- NCT02171286
- Where
- 5 sites in Canada
- Volunteers
- 432 people
- Sponsor
- British Columbia Cancer Agency
Osteoarthritis, Arthritis
Long-Term Analgesic Efficacy And Safety Of Tanezumab Alone Or In Combination With Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) Versus NSAIDs Alone In Patients With Osteoarthritis Of The Knee Or Hip
The purpose of this study is to investigate the long-term analgesic efficacy and safety of tanezumab for patients with osteoarthritis (OA) of the knee or hip currently experiencing partial benefit from, and are tolerating…
- Trial name
- NCT00809354
- Testing
- NSAID, tanezumab
- Where
- 333 sites in United States, Canada and 10 more
- Volunteers
- 2,720 people
- Sponsor
- Pfizer
Familial Dysautonomia
The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia
Familial dysautonomia (FD) is a devastating hereditary disease in which the development of selective neuronal populations is impaired because of a deficiency of the protein IKAP (Slaugenhaupt, 2002). There is no known cure.
- Trial name
- NCT02276716
- Testing
- Phosphatidylserine
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- NYU Langone Health
MPN (Myeloproliferative Neoplasms)
A Study of INCB050465 in Combination With Ruxolitinib in Subjects With Myelofibrosis
The purpose of this study is to evaluate the safety, tolerability, and efficacy of the combination of parsaclisib and ruxolitinib in subjects with myelofibrosis.
- Trial name
- NCT02718300
- Testing
- Parsaclisib, Ruxolitinib
- Where
- 39 sites in United States
- Volunteers
- 74 people
- Sponsor
- Incyte Corporation
Familial Dysautonomia (Riley-Day Syndrome), Hereditary Sensory and Autonomic Neuropathies
The Natural History of Familial Dysautonomia
The study will collect clinical information from patients with FD and allow them to give blood to help develop biological markers of the disease to aid diagnosis and treatment.
- Conditions
- Familial Dysautonomia (Riley-Day Syndrome), Hereditary Sensory and Autonomic Neuropathies, Hereditary Sensory and Autonomic Neuropathy 3
- Trial name
- NCT03920774
- Where
- 2 sites in United States, Israel
- Volunteers
- 400 people
- Sponsor
- NYU Langone Health
Familial Dysautonomia
The Safety and Tolerability of Kinetin, in Patients With Familial Dysautonomia
This is a study of kinetin, a nutritional supplement that corrects the mRNA splicing defect in patients with familial dysautonomia (FD, also known as Riley Day syndrome or hereditary sensory and autonomic neuropathy type III).
- Trial name
- NCT02274051
- Testing
- Kinetin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Neurodegenerative Disorders
Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases
Human fibroblasts and possibly other human somatic cells may be reprogrammed into induced pluripotent stem (iPS) cells by the forced expression of transcription factors (1-5).
- Trial name
- NCT00874783
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Hadassah Medical Organization
Overactive Bladder Syndrome
Comparisons of 3-month Versus 6-month Solifenacin Treatment for Female OAB Patients
To investigate whether prolonged period of treatment (6 months) can have a better therapeutic outcome than conventional period (3 months) of antimuscarinics.
- Trial name
- NCT01876186
- Testing
- Solifenacin for 12 weeks, Solifenacin for 24 weeks
- Where
- Taiwan
- Volunteers
- 200 people
- Sponsor
- National Taiwan University Hospital
Atunomic Neuropathy
Proprioception and Sensorimotor Control in Hereditary Sensory and Autonomic Neuropathy
This project comprises three sets of physiological studies - testing eight specific hypotheses - that will contribute new knowledge on proprioception and motor control in a genetic disorder that affects specific components of the…
- Trial name
- NCT02876939
- Testing
- kinesiology tape around the hip, knee and ankle joints, No kinesiology tape around the hip, knee and ankle joints
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- NYU Langone Health
Follicular Lymphoma
Study of Duvelisib in Combination With Rituximab vs Rituximab in Subjects With Previously Treated Follicular Lymphoma
A study to evaluate the safety and efficacy of duvelisib administered in combination with rituximab vs placebo in combination with rituximab in patients with previously treated CD20-positive follicular lymphoma who are not…
- Trial name
- DYNAMO + R (NCT02204982)
- Testing
- Duvelisib, Rituximab
- Where
- 5 sites in Australia, France and 2 more
- Volunteers
- 13 people
- Sponsor
- SecuraBio