Clinical trials: Fetal Developmental Abnormality
Prader-Willi Syndrome
A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome
The objective of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in participants with Prader-Willi Syndrome.
- Trial name
- NCT03458416
- Testing
- Cannabidiol Oral Solution
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Myeloid Leukemia in Remission
Decitabine and Total-Body Irradiation Followed By Donor Bone Marrow Transplant and Cyclophosphamide in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
This phase II trial studies how well decitabine and total-body irradiation followed by donor bone marrow transplant and cyclophosphamide works in treating patients with relapsed or refractory acute myeloid leukemia.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT01707004
- Testing
- decitabine, fludarabine phosphate, busulfan
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Wisconsin, Madison
Huntington Disease
Pilot Study of Minocycline in Huntington's Disease
This study is being conducted to assess the impact of minocycline on the progression of symptoms of HD. The study will also assess whether it is reasonable to continue with further study of minocycline in HD.
- Trial name
- NCT00277355
- Testing
- minocycline
- Where
- 12 sites in United States, Canada
- Volunteers
- 114 people
- Sponsor
- Merit Cudkowicz
Cystic Fibrosis
Aztreonam Lysine for Pseudomonas Infection Eradication Study
This is an open-label, multi-center study in pediatric patients age 3 months to less than 18 years with cystic fibrosis (CF) and newly detected Pseudomonas aeruginosa (PA) pulmonary colonization/infection.
- Trial name
- ALPINE (NCT01375049)
- Testing
- Aztreonam for Inhalation Solution (AZLI)
- Where
- 58 sites in United States, Austria and 7 more
- Volunteers
- 105 people
- Sponsor
- Gilead Sciences
Intracranial Arteriovenous Malformations (AVM)
Protocol for Staged Stereotactic Radiosurgery for Large Arteriovenous Malformations
Stereotactic radiosurgery is a well established treatment option for arteriovenous malformations (AVMs). The potential complications related to radiosurgery are well documented and are predominately related to radiation effects…
- Trial name
- NCT02576535
- Testing
- Fractionated stereotactic radiosurgery, Leskell gamma unit
- Volunteers
- 18 people
- Sponsor
- NYU Langone Health
X-linked Hypophosphatemia
Examining the Effect of Burosumab on Muscle Function
Patients with X-linked hypophosphatemia (XLH) often report symptoms of fatigue and weakness particularly after exertion, in addition to their skeletal complaints.
- Trial name
- NCT04146935
- Testing
- Burosumab Injection [Crysvita]
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Yale University
Cirrhosis, Congenital Heart Disease
Role of Sildenafil for Fontan Associated Liver Disease (SiFALD) Study
The purpose of this study is to determine if the medication, sildenafil (also known as Revatio), can slow or stop the progression of liver disease in patients who previously had a Fontan operation.
- Trial name
- SiFALD (NCT03154476)
- Testing
- Sildenafil
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mayo Clinic
Retinitis Pigmentosa
A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II \& III, and Choroideremia.
- Trial name
- NCT00447993
- Testing
- Low Dose NT-501, High Dose NT-501
- Where
- 11 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurotech Pharmaceuticals
Cystic Fibrosis
Supplementation of Oral Reduced Glutathione in Pediatric Cystic Fibrosis Patients
Many individuals with cystic fibrosis experience growth failure. The reasons are not clear, but inflammation of the gut in these patients seems to be one important reason.
- Trial name
- NCT02029521
- Testing
- Oral reduced l-glutathione
- Where
- Italy
- Volunteers
- 47 people
- Sponsor
- Clark Bishop
Alpha 1-Antitrypsin Deficiency
Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)
The purpose of this study is to evaluate the effects of weekly augmentation therapy with ARALAST Fraction IV-1 (Fr IV-1) on epithelial lining fluid (ELF) alpha 1-proteinase inhibitor levels and other ELF analytes and to assess…
- Trial name
- NCT00396006
- Testing
- Alpha1-Proteinase Inhibitor
- Where
- 5 sites in Australia, New Zealand
- Volunteers
- 21 people
- Sponsor
- Baxalta now part of Shire
Aniridia, Iris; Deformity
Clinical Evaluation of Morcher Artificial Iris Diaphragms
The purpose of this study is to determine the safety and efficacy of Morcher iris diaphragms in the treatment of congenital and acquired aniridia.
- Trial name
- NCT00812708
- Testing
- Morcher iris diaphragm implantation
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- University of California, Los Angeles
Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Simplex Kobner
Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
Epidermolysis bullosa (EB) simplex is a rare orphan disease caused by a mutation in DNA leading to abnormal dominant keratins in the skin.
- Conditions
- Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Simplex Kobner, Weber-Cockayne Syndrome
- Trial name
- NCT02960997
- Testing
- Sirolimus, 2%, Vehicle
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Stanford University
Hyperlipoproteinemia Type II, Hypercholesterolemia, Familial
Study to Assess the Tolerability and Efficacy of Anacetrapib (MK-0859) Co-Administered With Statin in Participants With Heterozygous Familial Hypercholesterolemia (MK-0859-020)
The objective of this study is to evaluate the efficacy and tolerability of adding anacetrapib to ongoing statin therapy in participants with heterozygous familial hypercholesterolemia (HeFH).
- Trial name
- REALIZE (NCT01524289)
- Testing
- Anacetrapib
- Volunteers
- 306 people
- Sponsor
- Merck Sharp & Dohme LLC
Congenital Hypothyroidism, Hypothyroidism
Generic vs. Name-Brand Levothyroxine
This study compares two different brands of thyroxine (thyroid hormone). Currently, pharmacists may be substituting generic formulations of thyroid hormone without your doctor knowing about this.
- Trial name
- NCT00403390
- Testing
- Brand Name Levothyroxine (Synthroid), Generic formulation of Levothyroxine
- Where
- United States
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Huntington Disease
Effect of PBT2 in Patients With Early to Mid Stage Huntington Disease
Huntington disease (HD) is an inherited neurodegenerative disease which affects over 30,000 people in both the United States and Australia.
- Trial name
- Reach2HD (NCT01590888)
- Testing
- PBT2
- Where
- 19 sites in United States, Australia
- Volunteers
- 109 people
- Sponsor
- Prana Biotechnology Limited
Cystic Fibrosis, Mucociliary Clearance Defect
Researching the Effects of Airway Clearance Therapies in Cystic Fibrosis
This is a pilot study investigating the effectiveness and clinical efficacy of airway clearance therapy (ACT) in cystic fibrosis (CF).
- Trial name
- REACT-CF (NCT03078127)
- Testing
- Baseline (Huff-Cough alone), Oscillatory Positive Expiatory Pressure Device, Whole-Body Vibration Platform
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Anemia
A Phase II Trial of Regadenoson in Sickle Cell Anemia
This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called Regadenoson (or Lexiscan) to learn whether the drug works in treating a specific disease, in this case…
- Trial name
- NCT01788631
- Testing
- Regadenoson
- Where
- 12 sites in United States
- Volunteers
- 100 people
- Sponsor
- Dana-Farber Cancer Institute
Pulmonary Disease, Transcatheter Pulmonary Valve Replacement (TPVR)
ALTERRA: SAPIEN 3 THV With the Alterra Adaptive Prestent
To demonstrate the safety and effectiveness of the Edwards Alterra Adaptive Prestent in conjunction with the Edwards SAPIEN 3 Transcatheter Heart Valve (THV) System in subjects with a dysfunctional right ventricular outflow…
- Conditions
- Pulmonary Disease, Transcatheter Pulmonary Valve Replacement (TPVR), Tetralogy of Fallot, Congenital Heart Disease
- Trial name
- NCT03130777
- Testing
- Edwards Alterra Adaptive Prestent with SAPIEN 3 THV
- Where
- 14 sites in United States
- Volunteers
- 86 people
- Sponsor
- Edwards Lifesciences
Hemophilia A
Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)
To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To…
- Trial name
- NCT01736475
- Testing
- Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, PEGylated Recombinant Factor VIII
- Where
- 72 sites in United States, Australia and 18 more
- Volunteers
- 159 people
- Sponsor
- Baxalta now part of Shire
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
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