Clinical trials: Newborn
Muscular Dystrophies, Muscular Dystrophy, Duchenne
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne Muscular Dystrophy (DMD).
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- HOPE-2 (NCT03406780)
- Testing
- CAP-1002
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Capricor Inc.
Neuroendocrine Tumors, Von Hippel-Lindau Syndrome
Evaluation of 68Gallium-DOTATATE PET/CT for Detecting Neuroendocrine Tumors
Background: Neuroendocrine tumors (NETs) are rare but have been more common over the past decade. The only treatment for NETs is surgery, but most are found when they are too advanced for surgery.
- Conditions
- Neuroendocrine Tumors, Von Hippel-Lindau Syndrome, Hippel-Lindau Disease
- Trial name
- NCT01967537
- Testing
- 68Gallium DOTATATE, Radio-guided surgery
- Where
- United States
- Volunteers
- 341 people
- Sponsor
- National Cancer Institute (NCI)
Chronic Myeloproliferative Disorders, Leukemia
Busulfan, Fludarabine, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Stem Cell Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and busulfan, before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- Bu Flu TBI (NCT00245037)
- Testing
- therapeutic allogeneic lymphocytes, busulfan, cyclosporine
- Where
- United States
- Volunteers
- 147 people
- Sponsor
- OHSU Knight Cancer Institute
Multiple Myeloma
WT1 Vaccine Treatment of Patients With Multiple Myeloma After Autologous Stem Cell Transplantation
The purpose of this trial is to assess the immune response after vaccination with a peptide vaccine called galinpepimut-S (or GPS) in a type of blood cancer called multiple myeloma.
- Trial name
- NCT01827137
- Testing
- Galinpepimut-S, GM-CSF, Montanide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Sellas Life Sciences Group
Stillbirth, Neonatal Death
Intermittent Auscultation Using Handheld Doppler: a Randomized Controlled Trial Comparing Perinatal Outcomes in Uganda
A novel handheld Doppler has been developed which is rugged and is powered only by a hand crank. This study aims to test the use of this Doppler versus the Pinard horn to monitor the fetal heart rate in labour in a hospital…
- Trial name
- NCT02273037
- Testing
- Fetal heart rate Doppler, Partograph
- Where
- 2 sites in Canada, Uganda
- Volunteers
- 1,987 people, healthy volunteers welcome
- Sponsor
- The Hospital for Sick Children
Epilepsy, Dravet Syndrome (DS)
A Study of Soticlestat in Adults and Children With Rare Epilepsies
The main aim is to assess the long-term safety and tolerability of soticlestat when used along with other anti-seizure treatment. Participants will receive soticlestat twice a day.
- Conditions
- Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
- Trial name
- Endymion 1 (NCT03635073)
- Testing
- Soticlestat
- Where
- 59 sites in United States, Australia and 6 more
- Volunteers
- 156 people
- Sponsor
- Takeda
Gout
Long-Term Safety of Febuxostat in Subjects With Gout.
The purpose of this study is to evaluate the long-term safety of febuxostat, once daily (QD), in maintaining serum urate levels within clinically acceptable levels in subjects with gout.
- Trial name
- FOCUS (NCT00174941)
- Testing
- Febuxostat
- Volunteers
- 116 people
- Sponsor
- Takeda
Sickle Cell Disease
Simvastatin (Zocor) Therapy in Sickle Cell Disease
Recent clinical and experimental data indicate that statins have effects beyond cholesterol lowering that may be beneficial in sickle cell disease by protecting the vascular endothelium.
- Trial name
- NCT00508027
- Testing
- Simvastatin
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Cystic Fibrosis
A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the…
- Trial name
- NCT03447262
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 100 sites in United States, Australia and 9 more
- Volunteers
- 484 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cerebral Cavernous Malformation
The Symptomatic Cerebral Cavernous Malformation Trial of REC-994
This is a two-part, multi-center, randomized, double-blind, placebo-controlled study to investigate the safety, efficacy and pharmacokinetics of REC-994 (200 mg and 400 mg) compared to placebo in participants with symptomatic…
- Trial name
- SYCAMORE (NCT05085561)
- Testing
- REC-994
- Where
- 15 sites in United States
- Volunteers
- 62 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Gout
RDEA3170 and Allopurinol Combination Study in Gout Subjects
This is a Phase 1b, randomized, open-label, drug-drug interaction study in adult male subjects with gout. It is designed to assess the pharmacokinetics (PK) and pharmacodynamics (PD) of RDEA3170 or allopurinol alone and in…
- Trial name
- NCT02279641
- Testing
- RDEA3170 10 mg, allopurinol 300 mg
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Ardea Biosciences, Inc.
Hemoglobin SC Disease
Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes. Hemoglobin SCD (HbSC) is a form of SCD that is characterized by dense red blood cells.
- Trial name
- NCT00532883
- Testing
- Hydroxyurea, Magnesium Pidolate
- Where
- 19 sites in United States
- Volunteers
- 44 people
- Sponsor
- St. Jude Children's Research Hospital
Becker Muscular Dystrophy
(-)- Epicatechin Becker Muscular Dystrophy
This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial.
- Trial name
- NCT03236662
- Testing
- (-)-Epicatechin
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Craig McDonald, MD
Huntington Chorea
Efficacy and Safety on SOM3355 in Huntington's Disease Chorea
Phase IIb, randomized, double-blind, placebo-controlled study in parallel groups assessing the efficacy and safety of two doses of SOM3355 in patients suffering from Huntington's Disease with choreic movements.
- Trial name
- NCT05475483
- Testing
- SOM3355 200 mg capsules, SOM3355 300 mg capsules
- Where
- 23 sites in France, Germany and 5 more
- Volunteers
- 139 people
- Sponsor
- SOM Innovation Biotech SA
Epidermolysis Bullosa
Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB). Funding Source: FDA Office of Orphan Products Development
- Trial name
- NCT02090283
- Testing
- SD-101 dermal cream (6%)
- Where
- 7 sites in United States
- Volunteers
- 42 people
- Sponsor
- Scioderm, Inc.
Atopic Dermatitis
Efficacy and Safety of MEDI3506 in Adult Subjects With Atopic Dermatitis
This is a research study to determine the efficacy and safety of investigational drug MEDI3506 for the treatment of adult subjects with Atopic Dermatitis.
- Trial name
- NCT04212169
- Testing
- MEDI3506
- Where
- 38 sites in United States, Australia and 4 more
- Volunteers
- 148 people
- Sponsor
- AstraZeneca
Hemophilia A With Inhibitor, Hemophilia A
INdividualized ITI Based on Fviii(ATE) Protection by VWF
The primary goal of the INITIATE trial is to compare the clinical outcome of individualized lot selection to random lot selection utilizing one plasma-derived von Willebrand factor (VWF)/coagulation factor (FVIII) complex…
- Trial name
- INITIATE (NCT03204539)
- Testing
- Wilate
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- University of California, Davis
Prader-Willi Syndrome
Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
This Phase 3 study is designed to test the effectiveness of intranasal carbetocin (LV-101) in participants with Prader-Willi syndrome (PWS). Carbetocin is an oxytocin analog (a man-made chemical that is like oxytocin).
- Trial name
- CARE-PWS (NCT03649477)
- Testing
- 3.2 mg intranasal carbetocin, 9.6 mg intranasal carbetocin
- Where
- 24 sites in United States, Australia and 1 more
- Volunteers
- 130 people
- Sponsor
- Levo Therapeutics, Inc.
Eczema, Dermatitis
Efficacy and Safety Study of Desloratadine (MK-4117) in Japanese Participants With Eczema/Dermatitis and Dermal Pruritus (MK-4117-202)
This is an efficacy and safety study of up to 12 weeks of desloratadine in Japanese participants with eczema/dermatitis and dermal pruritus.
- Conditions
- Eczema, Dermatitis, Dermal Pruritus
- Trial name
- NCT01916980
- Testing
- Desloratadine 5 mg
- Volunteers
- 94 people
- Sponsor
- Organon and Co
Sickle Cell Anemia
Prasugrel Versus Placebo in Adult Sickle Cell Disease
The purpose of this trial is to assess the safety of Prasugrel in adult patients with sickle cell disease (SCD) by monitoring the rate and severity of hemorrhagic events requiring medical intervention compared to placebo for 30…
- Trial name
- NCT01167023
- Testing
- Prasugrel
- Where
- 14 sites in United States
- Volunteers
- 62 people
- Sponsor
- Eli Lilly and Company
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