Clinical trials: Genetic Disorder
Familial Amyloid Polyneuropathy, ATTR-PN
An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
This study is designed to determine the long-term safety and tolerability of Fx-1006A as well as the effects of Fx-1006A on clinical outcomes in patients with ATTR-PN.
- Trial name
- NCT00791492
- Testing
- Fx-1006A
- Where
- 11 sites in Argentina, Brazil and 4 more
- Volunteers
- 86 people
- Sponsor
- Pfizer
Congenital Adrenal Hyperplasia, Hyperplasia
Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia
This is an open-label, non-randomized, single arm proof-of concept feasibility trial comparing standard oral hydrocortisone (HC) treatment with a six-week intervention of interval bolus delivery (pulsatile) of subcutaneous (SQ)…
- Conditions
- Congenital Adrenal Hyperplasia, Hyperplasia, Adrenal Hyperplasia, Congenital Disorders
- Trial name
- NCT03718234
- Testing
- Subcutaneous hydrocortisone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Minnesota
Gastric Adenocarcinoma
Derazantinib Alone or in Combination With Paclitaxel, Ramucirumab or Atezolizumab in Gastric Adenocarcinoma
The purpose of this study was to evaluate the efficacy of derazantinib monotherapy or derazantinib in combination with paclitaxel and ramucirumab in patients with gastric adenocarcinoma (GAC) i.e.
- Trial name
- FIDES-03 (NCT04604132)
- Testing
- Derazantinib, Derazantinib-paclitaxel-ramucirumab combination
- Where
- 81 sites in United States, Argentina and 13 more
- Volunteers
- 47 people
- Sponsor
- Basilea Pharmaceutica
Atopic Dermatitis
Secukinumab for Treatment of Atopic Dermatitis
Atopic Dermatitis, also known as atopic eczema, or eczema, is a common skin disease that can affect males and females of all ages, but often starts in childhood.
- Trial name
- NCT02594098
- Testing
- Secukinumab
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Chronic Hepatitis C, Hemophilia
Viral Kinetics in HCV Clearance in Subjects With Hemophilia
This study will examine viral dynamic responses in subjects with chronic hepatitis C and hemophilia when treated with pegylated interferon + ribavirin and telaprevir.
- Trial name
- HCV/Hemophil (NCT01704521)
- Testing
- PegInterferon, Ribavirin, Telaprevir
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Kenneth Sherman
Hereditary Angioedema (HAE)
CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema
The purpose of this study is to investigate the safety, PK / PD, and efficacy of SC CSL312 for prophylactic treatment of pediatric subjects with HAE.
- Trial name
- NCT05819775
- Testing
- CSL312
- Where
- 13 sites in United States, Australia and 3 more
- Volunteers
- 22 people
- Sponsor
- CSL Behring
Adolescent Behavior, Intellectual Disability
Promotion of Physical Activity in Adolescents With Intellectual and Developmental Disabilities
The objective of this study is to compare the effect of two strategies to increase MVPA in adolescents with intellectual and developmental disabilities (IDD): a single level intervention delivered to the adolescent only, and a…
- Conditions
- Adolescent Behavior, Intellectual Disability, Down Syndrome, Physical Activity
- Trial name
- NCT03684512
- Testing
- Group Exercise Sessions, Individual Support Sessions, Facebook Group
- Where
- United States
- Volunteers
- 116 people
- Sponsor
- University of Kansas
Pleuropulmonary Blastoma, Recurrent Malignant Peripheral Nerve Sheath Tumor
Lorvotuzumab Mertansine in Treating Younger Patients With Relapsed or Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, Pleuropulmonary Blastoma, Malignant Peripheral Nerve Sheath Tumor, or Synovial Sarcoma
This phase II trial studies how well lorvotuzumab mertansine works in treating younger patients with Wilms tumor, rhabdomyosarcoma, neuroblastoma, pleuropulmonary blastoma, malignant peripheral nerve sheath tumor (MPNST), or…
- Conditions
- Pleuropulmonary Blastoma, Recurrent Malignant Peripheral Nerve Sheath Tumor, Recurrent Neuroblastoma, Recurrent Rhabdomyosarcoma
- Trial name
- NCT02452554
- Testing
- Laboratory Biomarker Analysis, Lorvotuzumab Mertansine, Pharmacological Study
- Where
- 69 sites in United States
- Volunteers
- 62 people
- Sponsor
- Children's Oncology Group
Huntington's Disease (HD)
Creatine Safety & Tolerability in Huntington's Disease
The purpose of this study is to extend findings from the creatine dose-finding study (CREST-UP1) in Huntington's disease to evaluate the long-term safety, tolerability, and clinical impact of high dose creatine.
- Trial name
- CREST-X (NCT01412151)
- Testing
- Creatine monohydrate
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Massachusetts General Hospital
Zollinger-Ellison Syndrome, Multiple Endocrine Neoplasia
Treatment of Zollinger-Ellison Syndrome With Prevacid
The purpose of this study is to study the safety and efficacy of high dose Prevacid in the long-term treatment of patients who secrete abnormally large amounts of gastric acid.
- Trial name
- NCT00204373
- Testing
- Lansoprazole (Prevacid)
- Volunteers
- 72 people
- Sponsor
- Charles Mel Wilcox, MD
HER2 Positive Breast Cancer
A Study of Tucatinib or Placebo With Trastuzumab and Pertuzumab for Metastatic HER2+ Breast Cancer
This study is being done to see if tucatinib works better than placebo when given with other drugs to treat participants with HER2-positive breast cancer.
- Trial name
- HER2CLIMB-05 (NCT05132582)
- Testing
- Tucatinib, Trastuzumab, Pertuzumab
- Where
- 307 sites in United States, Australia and 21 more
- Volunteers
- 654 people
- Sponsor
- Seagen, a wholly owned subsidiary of Pfizer
Friedreich Ataxia
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
The purpose of this study is to evaluate the efficacy of TAK-831 versus placebo on upper extremity (arm and hands) motor function and manual dexterity.
- Trial name
- NCT03214588
- Testing
- TAK-831
- Where
- 6 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurocrine Biosciences
Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors
Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
Urea cycle disorders are inherited illnesses in which the body does not produce enough of the chemicals that remove ammonia, a byproduct of protein metabolism, from the blood stream.
- Conditions
- Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
- Trial name
- NCT00345605
- Testing
- Sodium Phenylbutyrate, Arginine
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Brendan Lee
Cystic Fibrosis, Allergic Bronchopulmonary Aspergillosis
Open-label Vitamin D Trial for Patients With Cystic Fibrosis and Allergic Bronchopulmonary Aspergillosis
The purpose of this study is to see if giving people with CF and ABPA enough vitamin D to make their blood levels of the vitamin higher, will reduce the allergic response in their body and make the symptoms caused by ABPA better.
- Trial name
- NCT01222273
- Testing
- cholecalciferol (Vitamin D3)
- Where
- 2 sites in United States
- Volunteers
- 7 people
- Sponsor
- University of Pittsburgh
Neurofibroma, Plexiform, Neurofibromatosis Type I
R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas
This study will examine whether the experimental drug R115777 (Tipifarnib) can shrink or slow the growth of plexiform neurofibromas in children and young adults with neurofibromatosis type 1 (NF1) and determine what side effects…
- Trial name
- NCT00021541
- Testing
- tipifarnib
- Where
- 12 sites in United States, Germany
- Volunteers
- 62 people
- Sponsor
- National Cancer Institute (NCI)
Tourette's Syndrome, Tic Disorders
Open Label Trial of Aripiprazole in Children and Adolescents With Tourette's Disorder
The purpose of this study is to determine if Abilify will reduce tics (repetitive, uncontrollable movements or vocalizations) in children and adolescents ages 7-18 with Tourette's Disorder (TD) or a chronic motor tic disorder…
- Trial name
- NCT00241176
- Testing
- Aripiprazole
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- NYU Langone Health
Sickle Cell Anemia, Stroke
Silent Cerebral Infarct Transfusion Multi-Center Clinical Trial
The goal of this study is to determine the effectiveness of blood transfusion therapy for prevention of silent cerebral infarct (stroke) in children with sickle cell anemia.
- Trial name
- SIT (NCT00072761)
- Testing
- transfusion therapy
- Where
- United States
- Volunteers
- 196 people
- Sponsor
- Washington University School of Medicine
Dermatitis, Dermatitis, Atopic
Study Evaluating Efficacy and Safety of PF-04965842 and Dupilumab in Adult Subjects With Moderate to Severe Atopic Dermatitis on Background Topical Therapy
B7451029 is a Phase 3 study to investigate PF-04965842 in adult patients who have moderate to severe atopic dermatitis and use background topical therapy.
- Conditions
- Dermatitis, Dermatitis, Atopic, Eczema, Skin Diseases
- Trial name
- JADE Compare (NCT03720470)
- Testing
- PF-04965842 100 mg, PF-04965842 200 mg, Dupilumab
- Where
- 216 sites in United States, Australia and 16 more
- Volunteers
- 838 people
- Sponsor
- Pfizer
Autosomal Dominant Polycystic Kidney Disease
Short-term Renal Hemodynamic Effects of Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
The purpose of the trial was to determine the short-term effects of tolvaptan in patients with autosomal dominant polycystic kidney disease (ADPKD) at various levels of renal function.
- Trial name
- NCT01336972
- Testing
- Tolvaptan
- Where
- Netherlands
- Volunteers
- 29 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-B (NCT03905694)
- Testing
- Lumasiran
- Where
- 9 sites in United States, France and 3 more
- Volunteers
- 18 people
- Sponsor
- Alnylam Pharmaceuticals