Clinical trials: Abnormalities
Fabry Disease
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study.
- Trial name
- NCT02194985
- Testing
- migalastat HCl 150 mg
- Where
- 28 sites in United States, Argentina and 13 more
- Volunteers
- 84 people
- Sponsor
- Amicus Therapeutics
Phenylketonurias
Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
This is a 2 arm, randomised, controlled, cross-over study in 16 children with PKU. Subjects who are currently taking a Phe free/low Phe protein substitute will be recruited for a 31-day trial.
- Trial name
- NCT05487378
- Testing
- PKU GOLIKE
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- APR Applied Pharma Research s.a.
Atopic Dermatitis
Evaluation of Upadacitinib in Adolescent and Adult Patients With Moderate to Severe Atopic Dermatitis (Eczema)
The objective of this study is to assess the efficacy and safety of upadacitinib for the treatment of adolescent and adult participants with moderate to severe atopic dermatitis (AD) who are candidates for systemic therapy.
- Trial name
- Measure Up 1 (NCT03569293)
- Testing
- Upadacitinib
- Where
- 180 sites in United States, Argentina and 23 more
- Volunteers
- 912 people
- Sponsor
- AbbVie
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
Hemophilia A
A Study of TAK-660 in Surgical Procedures for People With Hemophilia A.
This study is about a factor VIII medicine called Adynovate (TAK-660) used during surgery for people with hemophilia A who have low blood levels of factor VIII.
- Trial name
- NCT04941898
- Testing
- PEGylated Recombinant Factor VIII
- Where
- Japan
- Volunteers
- 16 people
- Sponsor
- Takeda
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase.
- Trial name
- NCT02279498
- Testing
- Liprotamase, porcine (pig) PERT
- Where
- 54 sites in United States, Canada and 5 more
- Volunteers
- 128 people
- Sponsor
- Anthera Pharmaceuticals
Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia
Fludarabine and Total-Body Irradiation Followed By Donor Stem Cell Transplant and Cyclosporine and Mycophenolate Mofetil in Treating HIV-Positive Patients With or Without Cancer
This clinical trial studies the side effects and best dose of giving fludarabine and total-body irradiation (TBI) together followed by a donor stem cell transplant and cyclosporine and mycophenolate mofetil in treating human…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00112593
- Testing
- fludarabine phosphate, total-body irradiation, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Fred Hutchinson Cancer Center
Anemia, Sickle Cell
Effectiveness of Arginine as a Treatment for Sickle Cell Anemia
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited genetic disease that can cause intense pain episodes.
- Trial name
- Arginine (NCT00513617)
- Testing
- Arginine
- Where
- 17 sites in United States
- Volunteers
- 128 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Atopic Dermatitis
Study to Assess the Safety of Dupilumab (REGN668/SAR231893) Administered Concomitantly With Topical Corticosteroids (TCS) in Patients With Moderate-to-severe Atopic Dermatitis (AD)
The purpose of this study was to assess the safety of Dupilumab administered concomitantly with topical corticosteroids (TCS) in patients with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT01639040
- Testing
- Dupilumab, Topical Corticosteroid (TCS)
- Where
- 13 sites in Germany, Hungary and 1 more
- Volunteers
- 31 people
- Sponsor
- Regeneron Pharmaceuticals
Congenital Heart Disease
Endothelin Blockade in Patients With Single Ventricle Physiology
Recent studies have shown beneficial effects of sildenafil in patients with single ventricle congenital heart disease. The purpose of this study is to determine whether Bosentan, a drug with similar effects, will have similar…
- Trial name
- NCT00989911
- Testing
- Bosentan
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of California, Los Angeles
Hereditary Angioedema (HAE)
Open-label, Single-arm Study to Assess the Pharmacokinetics, Safety, and Tolerability of a Single Subcutaneous Dose of Icatibant in Healthy Japanese Volunteers
This is a single-dose study to evaluate the pharmacokinetics, safety, and tolerability of icatibant administered to adult Japanese subjects.
- Trial name
- NCT02045264
- Testing
- Icatibant (30 mg)
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Shire
Congenital Heart Defect
Erythropoietin and Pediatric Cardiac Surgery
Lack of bloodflow to the heart and brain when the heart is stopped during heart surgery can cause damage to those organs. We hypothesize that a single dose of erythropoietin prior to the heart bypass portion of surgery may…
- Trial name
- EPO (NCT00451698)
- Testing
- acyanotic erythropoietin
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Medical College of Wisconsin
Autosomal Dominant Polycystic Kidney Disease
A Study to Evaluate the Effects of GLPG2737 in Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
This is an exploratory, randomized, double-blind, placebo-controlled, parallel group, multicenter, proof of concept study (Phase 2a), evaluating orally administered GLPG2737 for a double-blind (DB) treatment period of 52 weeks…
- Trial name
- NCT04578548
- Testing
- GLPG2737
- Where
- 20 sites in Belgium, Czechia and 5 more
- Volunteers
- 66 people
- Sponsor
- Lakefront Biotherapeutics NV
Cleft Palates
Preventing Complications in Cleft Palate Repair With Antibiotics
The primary objective of this study is to determine the efficacy of administering a single dose of preoperative antibiotics to prevent complications in patients undergoing primary closure of a cleft secondary palate.
- Trial name
- NCT00760006
- Testing
- Unasyn, saline solution
- Where
- United States
- Volunteers
- 224 people
- Sponsor
- Joseph Losee
Rheumatoid Arthritis
Comorbidities and Outcomes in Early Rheumatoid Arthritis
Identify the burden of comorbidity at the time of diagnosis of rheumatoid arthritis. Identify the impact of comorbidity present at diagnosis on cardiovascular disease, death, rheumatoid arthritis disease progression and…
- Trial name
- NCT03675516
- Testing
- No specific intervention
- Where
- United Kingdom
- Volunteers
- 13,182 people
- Sponsor
- Momentum Data
Congenital Diaphragmatic Hernia
Delayed Cord Clamping for Congenital Diaphragmatic Hernia
Congenital diaphragmatic hernia (CDH) is a congenital anomaly associated with a high risk of mortality and need for life-saving interventions such as extracorporeal membrane oxygenation (ECMO), nitric oxide, and vasopressor…
- Trial name
- DING (NCT03314233)
- Testing
- DING
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Children's Hospital of Philadelphia
Down Syndrome, Physical Inactivity
Decreasing Alzheimer's Risk Through oNline Choreographed Exercise - Down Syndrome
The investigators are assessing acceptability, appropriateness, and feasibility of an remote choreographed exercise intervention using validated scales alongside qualitative data among young adults with Down Syndrome.
- Trial name
- DANCE-DS (NCT06537648)
- Testing
- Decreasing Alzheimer's Risk Through oNline Choreographed Exercise - Down Syndrome Program
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Kansas Medical Center
Diabetes Prevention in Women After GDM Who Are at High-risk
Dapagliflozin and Metformin,Alone and in Combination, in Overweight/Obese Prior GDM Women
Women with a history of gestational diabetes (GDM) are at substantially increased risk of type 2 diabetes mellitus (T2DM). Compared with the general population, these women are more likely to be overweight or obese.
- Trial name
- DAPA-GDM (NCT02338193)
- Testing
- DAPA/MET XR, DAPA, MET XR
- Where
- United States
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- Woman's
Atopic Dermatitis
A Study to Assess the Efficacy and Safety of Dupilumab in Participants With Severe Atopic Dermatitis (AD) That Are Not Controlled With Oral Cyclosporine A (CSA) or for Those Who Cannot Take Oral CSA Because it is Not Medically Advisable
The main objective of the trial is to evaluate the efficacy of 2 dose regimens of dupilumab compared to placebo, administered with concomitant topical corticosteroids (TCS), in adult patients with severe AD who are not adequately…
- Trial name
- NCT02755649
- Testing
- Dupilumab
- Where
- 73 sites in Austria, Belgium and 8 more
- Volunteers
- 325 people
- Sponsor
- Regeneron Pharmaceuticals
Hypophosphatasia (HPP)
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.
- Trial name
- NCT01419028
- Where
- 12 sites in United States, Australia and 5 more
- Volunteers
- 48 people
- Sponsor
- Alexion Pharmaceuticals, Inc.