Clinical trials: Newborn
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment (refractory).
- Trial name
- NCT03190915
- Testing
- Bone Marrow Aspiration and Biopsy, Trametinib
- Where
- 57 sites in United States
- Volunteers
- 10 people
- Sponsor
- National Cancer Institute (NCI)
Atopic Dermatitis, Pruritus
Management of Pruritus With Xyzal in Atopic Dermatitis
It is historically well known that the management of pruritus in atopic dermatitis is very difficult. Most of the patients are not controlled with traditional antihistamines such as Clarinex, Claritin, and Allegra.
- Trial name
- NCT00884325
- Testing
- Levocetirizine dihydrochloride (Xyzal)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Derm Research, PLLC
Polycystic Ovary Syndrome
Treating Inflammation in Polycystic Ovary Syndrome to Ameliorate Ovarian Dysfunction
Polycystic Ovary Syndrome (PCOS) is characterized by hyperandrogenism, ovulatory dysfunction and polycystic ovaries. Insulin resistance (IR) is a common feature of PCOS, and the resultant hyperinsulinemia is theorized to promote…
- Trial name
- TIN-PCOS-AOD (NCT03229408)
- Testing
- Salsalate
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Illinois Chicago
Neurofibromatosis 1
Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1
Children with neurofibromatosis type 1 (NF1) commonly suffer from the effects of cognitive, behavioral, and motor impairments. At present there is no specific treatment for this NF1 complication.
- Trial name
- NF1NAC (NCT04481035)
- Testing
- N-acetylcysteine (NAC)
- Where
- United States
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Wilson Disease
The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment
This was a 24-month study to assess copper parameters in participants with Wilson disease (WD) treated with standard of care (SoC) medications.
- Trial name
- NCT02763215
- Testing
- Standard of Care Medications
- Where
- 10 sites in United States, Austria and 3 more
- Volunteers
- 64 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Gout
Phase 3, Febuxostat, Allopurinol and Placebo-Controlled Study in Gout Subjects.
The purpose of this study is to compare febuxostat, allopurinol and placebo, once daily (QD), in subjects with gout.
- Trial name
- APEX (NCT00174915)
- Testing
- Febuxostat, Allopurinol
- Volunteers
- 1,072 people
- Sponsor
- Takeda
Fuchs Endothelial Corneal Dystrophy
A Multicenter Study Assessing the Efficacy and Safety of STN1010904 Ophthalmic Suspension 0.03% and 0.1% Compared With Vehicle in Subjects With Fuchs Endothelial Corneal Dystrophy (FECD)
This is a Phase IIa study to assess efficacy and safety of STN1010904 ophthalmic suspension (0.03%, and 0.1 %), twice daily dosing when compared to Placebo in subjects diagnosed with Fuchs Endothelial Corneal Dystrophy (FECD).
- Trial name
- PHANTOM (NCT05376176)
- Testing
- STN1010904 ophthalmic suspension 0.03% BID, STN1010904 ophthalmic suspension 0.1% BID
- Where
- 15 sites in United States, France and 1 more
- Volunteers
- 86 people
- Sponsor
- Santen Inc.
Hereditary Angioedema, Angioneurotic Edema
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening.
- Trial name
- NCT00225147
- Testing
- Recombinant Human C1 Inhibitor
- Where
- Netherlands
- Volunteers
- 77 people
- Sponsor
- Pharming Technologies B.V.
Cancer
Aprepitant in Preventing Nausea and Vomiting in Patients Who Are Undergoing a Stem Cell Transplant
RATIONALE: Antiemetic drugs, such as aprepitant, ondansetron, and dexamethasone, may help lessen or prevent nausea and vomiting in patients undergoing a stem cell transplant.
- Trial name
- NCT00248547
- Testing
- aprepitant, dexamethasone, ondansetron
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- OHSU Knight Cancer Institute
Huntington's Disease
A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod
The primary objective of this study is to assess the efficacy of laquinimod as treatment in participants with HD after 52 weeks using the Unified Huntington's Disease Rating Scale Total Motor Score (UHDRS-TMS or TMS).
- Trial name
- LEGATO-HD (NCT02215616)
- Testing
- Laquinimod
- Where
- 53 sites in United States, Canada and 8 more
- Volunteers
- 352 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Leukemia, Lung Cancer
Vaccine Therapy and GM-CSF in Treating Patients With Acute Myeloid Leukemia, Myelodysplastic Syndromes, Non-Small Cell Lung Cancer, or Mesothelioma
RATIONALE: Vaccines made from peptides may help the body build an effective immune response to kill cancer cells. Biological therapies, such as GM-CSF, may stimulate the immune system in different ways and stop cancer cells from…
- Conditions
- Leukemia, Lung Cancer, Malignant Mesothelioma, Myelodysplastic Syndromes
- Trial name
- NCT00398138
- Testing
- WT-1 analog peptide vaccine, incomplete Freund's adjuvant, sargramostim
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Hemophilia A
A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A
The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A.
- Trial name
- Leopold II (NCT01233258)
- Testing
- rFVIII (BAY81-8973) on demand, rFVIII (BAY81-8973) prophylaxis low-dose, rFVIII (BAY81-8973) prophylaxis high-dose
- Where
- 47 sites in United States, Argentina and 16 more
- Volunteers
- 80 people
- Sponsor
- Bayer
Sickle Cell Disease, Renal Function Disorder
Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH)
The use of hydroxyurea in sickle cell disease patients with glomerular hyperfiltration and renal failure requires a specific monitoring and dose adjustment in order to remain within the therapeutic interval while limiting the…
- Trial name
- NCT02522104
- Testing
- Siklos
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Theravia
Cystic Fibrosis
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03447249
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 101 sites in United States, Australia and 9 more
- Volunteers
- 385 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Age-Related Macular Degeneration
Study of Safety and Efficacy of Brolucizumab 6 mg Dosed Every 4 Weeks Compared to Aflibercept 2 mg Dosed Every 4 Weeks in Patients With Retinal Fluid Despite Frequent Anti-VEGF Injections
This clinical study was designed to compare the safety and efficacy of brolucizumab 6 mg dosed every 4 weeks to aflibercept 2 mg dosed every 4 weeks in those neovascular age-related macular degeneration (nAMD) patients with…
- Trial name
- MERLIN (NCT03710564)
- Testing
- Brolucizumab, Aflibercept
- Where
- 61 sites in United States, Puerto Rico
- Volunteers
- 535 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Assessing the Safety of Buprenorphine in People With Sickle Cell Disease
This study will assess the safety of changing pain medications (opioids) adult sickle cell patients take to another type of medication therapy (buprenorphine). Patients will be asked questions about their quality of life.
- Trial name
- NCT03492099
- Testing
- buprenorphine
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- Johns Hopkins University
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Sirolimus, Tacrolimus, Thymoglobulin and Rituximab as Graft-versus-Host-Disease Prophylaxis in Patients Undergoing Haploidentical and HLA Partially Matched Donor Hematopoietic Cell Transplantation
This Phase II clinical trial was designed for patients with hematologic malignancies in need of donor peripheral blood stem cell transplant, and have no HLA matched donor.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT01116232
- Testing
- anti-thymocyte globulin, rituximab, sirolimus
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Graft Versus Host Disease, Leukemia
Clofarabine, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk and/or Advanced Hematologic Cancer or Other Disease
RATIONALE: Giving chemotherapy, such as clofarabine, melphalan, and thiotepa, before a donor stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00423514
- Testing
- filgrastim, clofarabine, melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease
Treatment of Sickle Cell Anemia With Stem Cell Transplant
This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor.
- Conditions
- Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease, Sickle Cell-β0-thalassemia
- Trial name
- NCT01350232
- Testing
- Fludarabine, Cytarabine, Cellular Infusions
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Transthyretin (TTR) Amyloid Cardiomyopathy
Vyndaqel Capsules Special Investigation (ATTR-CM)
Secondary Data Collection : To confirm the safety and effectiveness profiles under the actual medical practice of Vyndaqel in Japan.
- Trial name
- NCT04108091
- Testing
- Treatment for TTR amyloidosis
- Where
- Japan
- Volunteers
- 1,214 people
- Sponsor
- Pfizer
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