Clinical trials: Multiple Congenital Anomaly
Cancer
Fentanyl Sublingual Spray in Treating Patients With Breakthrough Cancer Pain
This is a phase III, randomized, double-blind, placebo-controlled, multicenter study of the clinical response to fentanyl sublingual spray as a treatment for breakthrough cancer pain.
- Trial name
- NCT00538850
- Testing
- Fentanyl sublingual spray
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- INSYS Therapeutics Inc
Dermatitis, Atopic
A Study of Bermekimab for the Treatment of Adult Participants With Moderate-to-Severe Atopic Dermatitis
The purpose of this study is to evaluate the efficacy of Bermekimab, compared with placebo, in participants with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT04990440
- Testing
- Bermekimab
- Where
- 11 sites in United States, Argentina
- Volunteers
- 6 people
- Sponsor
- Janssen Research & Development, LLC
Dyslipidemias, Hypercholesterolemia
Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies
The study is a placebo-controlled, double-blind, randomized, phase 3 study in participants with heterozygous familial hypercholesterolemia (HeFH) and/or atherosclerotic cardiovascular disease (ASCVD) or multiple ASCVD risk…
- Conditions
- Dyslipidemias, Hypercholesterolemia, Familial Hypercholesterolemia, ASCVD
- Trial name
- TANDEM (NCT06005597)
- Testing
- Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe
- Where
- 45 sites in United States
- Volunteers
- 407 people
- Sponsor
- NewAmsterdam Pharma
Cystic Fibrosis
Safety, Tolerability, Pharmacokinetics, and Preliminary Pharmacodynamics of QBW251 in Healthy Subjects and Cystic Fibrosis Patients
This study is designed to assess the safety, tolerability, pharmacokinetics and preliminary pharmacodynamics (proof of concept) of QBW251 in healthy subjects and cystic fibrosis patients following single and multiple doses.
- Trial name
- NCT02190604
- Testing
- QBW251
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 153 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Alpha 1-Antitrypsin Deficiency
Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)
The goal of this trial was to explore the utility of evaluating emphysema progression through CT scans measuring lung density during a 2 year period of weekly infusions of either placebo or human alpha-1-antitrypsin (AAT…
- Trial name
- NCT00263887
- Testing
- Alpha1-Proteinase Inhibitor (Human), Albumin (Human) 20%, United States Pharmacopeia (USP)
- Where
- 3 sites in Denmark, Sweden and 1 more
- Volunteers
- 77 people
- Sponsor
- Grifols Therapeutics LLC
Gout
Review of Safety Using Rilonacept in Preventing Gout Exacerbations (RE-SURGE)
The purpose of this clinical research study is to determine the safety and effectiveness of an experimental drug called rilonacept in subjects with gout who are on urate-lowering therapy.
- Trial name
- NCT00856206
- Testing
- Rilonacept
- Where
- 124 sites in United States, Germany and 4 more
- Volunteers
- 1,315 people
- Sponsor
- Regeneron Pharmaceuticals
RET-altered Non Small Cell Lung Cancer, Medullary Thyroid Cancer
Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Participants With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors
This is a Phase 1/2, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic activity of pralsetinib (BLU-667)…
- Conditions
- RET-altered Non Small Cell Lung Cancer, Medullary Thyroid Cancer, RET-altered Papillary Thyroid Cancer, RET-altered Colon Cancer
- Trial name
- ARROW (NCT03037385)
- Testing
- pralsetinib (BLU-667)
- Where
- 71 sites in United States, Belgium and 11 more
- Volunteers
- 590 people
- Sponsor
- Hoffmann-La Roche
Dermatitis, Atopic
Study to Evaluate Efficacy and Safety of PF-04965842 in Subjects Aged 12 Years And Older With Moderate to Severe Atopic Dermatitis
B7451012 is a Phase 3 study to evaluate PF-04965842 in patients aged 12 years and older with a minimum body weight of 40 kg who have moderate to severe atopic dermatitis.
- Trial name
- JADE Mono-1 (NCT03349060)
- Testing
- PF-04965842 100 mg, PF-04965842 200 mg
- Where
- 88 sites in United States, Australia and 6 more
- Volunteers
- 387 people
- Sponsor
- Pfizer
Tuberous Sclerosis Complex, Epilepsy
Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
This phase I/II clinical trial is an open-label clinical trial design to verify safety and dosing for TAVT-18 (sirolimus) powder for oral solution in TSC infants (N=5).
- Trial name
- STOP2 (NCT04595513)
- Testing
- TAVT-18 (sirolimus)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Carney Complex, Chondrosarcoma
Linsitinib in Treating Patients With Gastrointestinal Stromal Tumors
This phase II trial studies how well linsitinib works in treating younger and adult patients with gastrointestinal stromal tumors.
- Conditions
- Carney Complex, Chondrosarcoma, Gastrointestinal Stromal Tumor, Paraganglioma
- Trial name
- NCT01560260
- Testing
- Laboratory Biomarker Analysis, Linsitinib, Pharmacological Study
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme.
- Trial name
- NCT00074984
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 26 sites in United States, Canada and 4 more
- Volunteers
- 82 people
- Sponsor
- Genzyme, a Sanofi Company
Atopic Dermatitis
A Phase 2 Study of CIM331 for Atopic Dermatitis Patients
To assess the safety, tolerability and efficacy of CIM331, compared to placebo, in atopic dermatitis patients who are inadequately controlled by or intolerant to topical therapy
- Trial name
- NCT01986933
- Testing
- nemolizumab (CIM331)
- Where
- 18 sites in United States
- Volunteers
- 264 people
- Sponsor
- Chugai Pharmaceutical
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Palifermin in Preventing Chronic Graft-Versus-Host Disease in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer
RATIONALE: Growth factors, such as palifermin, may prevent chronic graft-versus-host disease caused by donor stem cell transplant.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01233921
- Testing
- palifermin, flow cytometry, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Martin, Paul
Autosomal Dominant Polycystic Kidney Disease
Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy
Currently the only approved use for rapamycin (sirolimus) is for immunosuppression after renal transplantation. This trial is designed to determine whether rapamycin is safe and effective treatment for patients with polycystic…
- Trial name
- NCT00920309
- Testing
- Rapamycin
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Yale University
Hemophilia A With Inhibitor
The Hemophilia Inhibitor Eradication Trial
This is a multi-center randomized phase III clinical trial, the Inhibitor Eradication Trial, in which Eloctate ITI plus Emicizumab will be compared with Eloctate ITI alone to eradicate inhibitors in severe hemophilia A.
- Trial name
- NCT04303572
- Testing
- Eloctate ITI, Emicizumab
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Margaret Ragni
Measles, Rubella
Measles-Rubella Vaccine Immunogenicity at 6 and 9 Months of Age
This is an open-label, randomized, 2-arm clinical trial in healthy infants in Bangladesh. The primary purpose of the study is to assess the immunogenicity of measles-rubella (MR) vaccine when delivered at 6 months.
- Conditions
- Measles, Rubella, Rubella Syndrome, Congenital
- Trial name
- NCT03071575
- Testing
- Measles-rubella (MR) vaccine at 6 months of age, Measles-rubella (MR) vaccine at 9 months of age
- Where
- Bangladesh
- Volunteers
- 637 people, healthy volunteers welcome
- Sponsor
- Centers for Disease Control and Prevention
Acute Gout
Targeted Dose Finding of Canakinumab (ACZ885) for Management of Acute Flare in Refractory or Contraindicated Gout Patients
This 8-week study is designed to determine the target dose of canakinumab (ACZ885) for the management of acute flare in gout patients who are contraindicated to Non-Steroidal anti-inflammatory drugs and/or colchicine.
- Trial name
- NCT00798369
- Testing
- Canakinumab, Triamcinolone acetonide
- Where
- 75 sites in United States, Argentina and 9 more
- Volunteers
- 200 people
- Sponsor
- Novartis
Sickle Cell Disease
Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD
Study participants will undergo up to four periods of voxelotor administered orally at progressively higher dose levels from 1500 mg until either a maximum tolerated dose (MTD) or 3000 mg/day dose is reached, whichever occurs…
- Trial name
- NCT04247594
- Testing
- Voxelotor
- Where
- 6 sites in United Kingdom
- Volunteers
- 6 people
- Sponsor
- Pfizer
Hemophilia B
Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients
This study is a phase 1, open-label, multi-center, dose-escalation study to investigate the safety, pharmacokinetics and pharmacodynamics of ISU304/CB2679d in previously treated hemophilia B patients.
- Trial name
- NCT03186677
- Testing
- ISU304/CB2679d/Dalcinonacog alfa 75~150 IU/kg, BeneFIX
- Where
- 3 sites in South Korea
- Volunteers
- 11 people
- Sponsor
- ISU Abxis Co., Ltd.
Cardiomyopathy, Hypertrophic, Cardiomyopathy, Hypertrophic, Familial
Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure
The purpose of this study is to evaluate the effect of perhexiline on exercise performance (efficacy) and safety in patients with hypertrophic cardiomyopathy and moderate-to-severe heart failure following dosing for 16 weeks.
- Trial name
- NCT02862600
- Testing
- Perhexiline, Use of bioanalytical assay to monitor plasma levels of perhexiline
- Where
- 11 sites in United States
- Volunteers
- 35 people
- Sponsor
- Heart Metabolics Limited