Clinical trials: Malformation
Becker Muscular Dystrophy
Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)
(-)-Epicatechin will be evaluated for the treatment of progressive muscle loss and impaired skeletal muscle function in Becker Muscular Dystrophy (BMD) patients.
- Trial name
- NCT01856868
- Testing
- (-)-epicatechin
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Craig McDonald, MD
Leukemia, Lymphoma
Melphalan, Fludarabine, and Alemtuzumab Followed by Peripheral Stem Cell Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as melphalan and fludarabine, and a monoclonal antibody, such as alemtuzumab, before a donor bone marrow or peripheral blood stem cell transplant helps stop the growth of cancer…
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Trial name
- NCT00027560
- Testing
- alemtuzumab, cyclosporine, fludarabine phosphate
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Hemophilia B
Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B
Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.
- Trial name
- NCT03855280
- Testing
- APVO101
- Where
- 11 sites in Brazil, Georgia and 4 more
- Volunteers
- 21 people
- Sponsor
- Medexus Pharma, Inc.
Atopic Dermatitis
Phase 2a Study of the Safety, Tolerability, and Pharmacokinetics of Topically Administered PRN473 (SAR444727) in Patients With Mild to Moderate Atopic Dermatitis
This was a Ph2a study that consists of a double-blind, intra-patient placebo-controlled treatment period and an open-label uncontrolled treatment period with objective to evaluate the safety, tolerability, PK and preliminary…
- Trial name
- NCT04992546
- Testing
- PRN473 (SAR444727)
- Where
- 12 sites in United States, Canada
- Volunteers
- 39 people
- Sponsor
- Principia Biopharma, a Sanofi Company
Healthy
Controlled Randomized Study With Athena Base Plate Versus SenSura Base Plate on Perspiring Skin
Aim The aim of the Athena project is to develop an ostomy product that is softer and more flexible in order to improve the comfort for people with a stoma.
- Trial name
- NCT01245413
- Testing
- SenSura, Athena
- Where
- Denmark
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Coloplast A/S
Gaucher Disease Type 1
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher disease in patients who are unsuitable for enzyme replacement therapy (ERT), more data are required to establish the long term efficacy…
- Trial name
- NCT00319046
- Testing
- Miglustat
- Volunteers
- 42 people
- Sponsor
- Actelion
Autosomal Dominant Polycystic Kidney Disease
The ELiSA Study - Evaluation of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease
This is a Phase 2, open-label, parallel-group, multiple dose study designed to evaluate the pharmacokinetics, pharmacodynamics, safety and tolerability of multiple doses of lixivaptan in Autosomal Dominant Polycystic Kidney…
- Trial name
- NCT03487913
- Testing
- Lixivaptan
- Where
- 13 sites in United States
- Volunteers
- 31 people
- Sponsor
- Palladio Biosciences
Atopic Dermatitis
A Study of the Long-Term Safety of Crisaborole Ointment, 2% in Japanese Pediatric and Adult Participants With Mild to Moderate Atopic Dermatitis
This study is a Phase 3, multicenter, open-label, long-term safety extension study of Studies C3291032 and C3291031 in Japanese pediatric and adult participants with mild to moderate Atopic Dermatitis (AD).
- Trial name
- NCT04498403
- Testing
- Crisaborole 2%
- Where
- 11 sites in Japan
- Volunteers
- 40 people
- Sponsor
- Pfizer
Haemophilia A
Efficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10)
The study investigates how well the medicine called turoctocog alfa pegol (N8-GP) works in previously treated Chinese patients with severe haemophilia A. Participants will be treated with N8-GP.
- Trial name
- Pathfinder10 (NCT05082116)
- Testing
- turoctocog alfa pegol (N8-GP)
- Where
- 11 sites in China
- Volunteers
- 36 people
- Sponsor
- Novo Nordisk A/S
Hereditary Angioedema (HAE)
Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects
This trial is looking to gain information about the safety and tolerability of an investigational treatment (SHP623) in healthy adult volunteers.
- Trial name
- NCT02663687
- Testing
- Recombinant human C1 esterase inhibitor, SHP623
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Shire
Friedreich's Ataxia
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
The objectives of the study are: To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA).
- Trial name
- NCT01898884
- Testing
- VP 20629
- Where
- 5 sites in United States
- Volunteers
- 46 people
- Sponsor
- Shire
Muscular Dystrophy, Duchenne, Ambulatory Care
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD.
- Trial name
- DELOS (NCT01027884)
- Testing
- Idebenone
- Where
- 23 sites in United States, Austria and 8 more
- Volunteers
- 65 people
- Sponsor
- Santhera Pharmaceuticals
Heterozygous Familial Hypercholesterolemia, Coronary Artery Disease
Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease
The purpose of this study is to determine whether mipomersen safely and effectively lowers low-density lipoprotein cholesterol (LDL-C) in patients with Heterozygous Familial Hypercholesterolemia (HeFH) and coronary artery disease…
- Trial name
- RADICHOL II (NCT00706849)
- Testing
- mipomersen sodium
- Where
- 48 sites in United States, Canada
- Volunteers
- 124 people
- Sponsor
- Kastle Therapeutics, LLC
Nervous System Disorder, Genetic Syndrome
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX…
- Trial name
- IEDAT-01 (NCT01255358)
- Testing
- Dexamethasone
- Where
- 2 sites in Italy
- Volunteers
- 22 people
- Sponsor
- Quince Therapeutics S.p.A.
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.
Dexmedetomidine, Congenital Heart Disease
Pharmacokinetic Study of Intranasal Dexmedetomidine in Pediatric Patients With Congenital Heart Disease
The main objectives of the study are to determine peak plasma drug concentration levels and corresponding time of dexmedetomidine following intranasal administration in children age ≥1 mo to ≤ 6 yr with congenital heart disease…
- Trial name
- NCT03417999
- Testing
- Dexmedetomidine
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Children's Hospital of Philadelphia
Duchenne and Becker Muscular Dystrophy, Polycytemia Vera
Placebo-Corrected Effects of Therapeutic Dose (100 mg) and Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval
The study will evaluate the effect of a therapeutic dose and a supratherapeutic dose of ITF2357 on the QT/QTc interval.
- Trial name
- NCT04821063
- Testing
- ITF2357 10 mg/mL, Moxifloxacin Hydrochloride
- Where
- Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Italfarmaco
Atopic Dermatitis
Efficacy of Desonide (Desonatetm) Gel 0.05% in Younger and Older Subjects With Atopic Dermatitis
The purpose of this research study is to better understand how this study drug works when people use it to treat atopic dermatitis. Desonate has been approved by the US Food and Drug Administration (FDA) for atopic dermatitis.
- Trial name
- NCT00690833
- Testing
- topical desonide hydrogel 0.05%
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Wake Forest University
Primary Immune Deficiency Disorder
Follow-up Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)
The objective of this study is to assess the long-term safety, tolerability, and efficacy of IgPro20 in subjects with primary immunodeficiency (PID) as a follow-up to the pivotal study ZLB06_002CR (NCT01199705).
- Trial name
- NCT01458171
- Testing
- Immune globulin subcutaneous (Human)
- Where
- 9 sites in Japan
- Volunteers
- 23 people
- Sponsor
- CSL Behring
Prader-Willi Syndrome
Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS).
- Trial name
- NCT02844933
- Testing
- Cannabidiol
- Where
- 7 sites in United States
- Volunteers
- 7 people
- Sponsor
- Radius Pharmaceuticals, Inc.
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