Clinical trials: Multiple Congenital Anomaly
Prader-Willi Syndrome, Hyperphagia
Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2)…
- Trial name
- OXT-PWS (NCT02629991)
- Testing
- Intranasal Oxytocin (IN-OXT)
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Montefiore Medical Center
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Congenital Adrenal Hyperplasia
Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1
Children with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency tend to have elevated circulating levels of androgens, which can accelerate skeletal maturation and adversely impact adult height.
- Trial name
- NCT02574910
- Testing
- Abiraterone acetate
- Where
- 4 sites in United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
Hemophilia A
BAX 855 PK-guided Dosing
1. To compare the efficacy and safety of pharmacokinetic (PK)-guided treatment with BAX 855 targeting FVIII trough levels of 1-3% and approximately 10% (8-12%) 2.
- Trial name
- PROPEL (NCT02585960)
- Testing
- PEGylated Recombinant Factor VIII
- Where
- 83 sites in United States, Australia and 20 more
- Volunteers
- 135 people
- Sponsor
- Baxalta now part of Shire
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex
The purpose of the study is to evaluate the safety and potential side effects of everolimus (an experimental drug) on a person with Tuberous Sclerosis Complex who also has been diagnosed with a brain tumor (astrocytoma) The…
- Trial name
- NCT00411619
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Hypothyroidism, Trisomy 21
Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients
Children with levothyroxine (T21) have developmental delay and other functional gastrointestinal (GI) issues that may negatively affect L-T4 tolerability and absorption.
- Trial name
- NCT04747275
- Testing
- Liquid stable levothyroxine (L-T4) Tirosint-SOL, Oral tablet levothyroxine (L-T4)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Children's Mercy Hospital Kansas City
Atopic Dermatitis
Long-term Extension Trial in Subjects With Atopic Dermatitis Who Participated in Previous Tralokinumab Trials - ECZTEND
The purpose of this extension trial is to evaluate the long-term safety of tralokinumab.
- Trial name
- NCT03587805
- Testing
- Tralokinumab
- Where
- 309 sites in United States, Belgium and 9 more
- Volunteers
- 1,672 people
- Sponsor
- LEO Pharma
Atopic Dermatitis
JAK1 Inhibitor With Medicated Topical Therapy in Adolescents With Atopic Dermatitis
This is a randomized, double blind, placebo controlled, parallel group, Phase 3 study to evaluate the efficacy and safety of PF 04965842 in adolescent participants 12 to <18 years of age with moderate to severe AD.
- Trial name
- JADE TEEN (NCT03796676)
- Testing
- PF-04965842, PF04965842
- Where
- 136 sites in United States, Australia and 12 more
- Volunteers
- 287 people
- Sponsor
- Pfizer
Late Infantile Metachromatic Leukodystrophy
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
This is a single center, open-label study of patients with late infantile MLD. All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561).
- Trial name
- NCT00633139
- Testing
- Recombinant human Arylsulfatase A (rhASA)
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
Primary Immunodeficiency
A Study to Evaluate IGSC 20% Biweekly Dosing in Treatment-Experienced Participants and Loading/Maintenance Dosing in Treatment-Naïve Participants With Primary Immunodeficiency
The purpose of the study is to determine whether biweekly (every 2 weeks) administration of Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) produces a steady-state area under the…
- Trial name
- NCT04566692
- Testing
- IGSC 20%
- Where
- 12 sites in United States
- Volunteers
- 33 people
- Sponsor
- Grifols Therapeutics LLC
Down Syndrome
Treadmill Training and Orthotic Use in Infants With Down Syndrome
This study determines the effect of orthotic use in combination with treadmill training on the development of gross motor skills and walking onset in infants with Down syndrome.
- Trial name
- NCT00825175
- Testing
- Treadmill Training, Supramalleolar Orthoses
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- University of Michigan
Infant, Newborn, Infant, Low Birth Weight
Multi-dose Pharmacokinetics and Dose Ranging of Inositol in Premature Infants (INS-2)
This pilot study is a randomized, placebo-controlled, clinical trial to measure changes in blood and urine levels of inositol in premature infants at high risk for retinopathy of prematurity (ROP) following repeated doses of…
- Conditions
- Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature
- Trial name
- INS-2 (NCT01030575)
- Testing
- Inositol lower volume, Inositol mid-level volume, Inositol high volume
- Where
- 17 sites in United States
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- NICHD Neonatal Research Network
Chronic Myeloproliferative Disorders, Leukemia
Reduced-Intensity Busulfan and Fludarabine With or Without Antithymocyte Globulin Followed by Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease
RATIONALE: Giving low doses of chemotherapy, such as busulfan and fludarabine, before a donor stem cell transplant helps stop the growth of cancer and abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00448201
- Testing
- anti-thymocyte globulin, sargramostim, therapeutic allogeneic lymphocytes
- Where
- United States
- Volunteers
- 71 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Atopic Dermatitis
Study of 0417 Ointment in the Treatment of Atopic Dermatitis
The aim of this trial is to assess the efficacy of 0417 Ointment in the Treatment of Atopic Dermatitis. Treatment medication will be administered as follows: Apply a thin layer of ointment to affected skin areas twice daily and…
- Trial name
- NCT01139450
- Testing
- 0417, Vehicle of 0417 test product, Tacrolimus Ointment 0.03%
- Where
- United States
- Volunteers
- 899 people
- Sponsor
- Fougera Pharmaceuticals Inc.
Congenital Hyperinsulinism
An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism
The objective of this trial is to evaluate the safety, tolerability and glucose-raising effects of RZ358 in patients with Congenital Hyperinsulinism (HI).
- Trial name
- NCT04538989
- Testing
- RZ358 Sequential Group Cohort 1, RZ358 Sequential Group Cohort 2, RZ358 Sequential Group Cohort 3
- Where
- 17 sites in United States, Bulgaria and 9 more
- Volunteers
- 23 people
- Sponsor
- Rezolute
Tuberous Sclerosis Complex
Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy.
- Trial name
- NCT05323734
- Testing
- Ganaxolone
- Where
- 63 sites in United States, Australia and 8 more
- Volunteers
- 129 people
- Sponsor
- Marinus Pharmaceuticals
Osteochondrodysplasia
Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia
This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.
- Trial name
- upreACH-2 (NCT06067425)
- Testing
- SAR442501
- Where
- 9 sites in Australia, China and 3 more
- Volunteers
- 16 people
- Sponsor
- Sanofi
Kidney, Polycystic
HALT Progression of Polycystic Kidney Disease Study A
The efficacy of interruption of the renin-angiotensin-aldosterone system (RAAS) on the progression of cystic disease and on the decline in renal function in autosomal dominant kidney disease (ADPKD) will be assessed in two…
- Trial name
- HALT PKD A (NCT00283686)
- Testing
- Lisinopril, Telmisartan, Standard Blood Pressure Control
- Where
- 7 sites in United States
- Volunteers
- 558 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Tumor, Tumor, Solid
Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)
A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes
- Conditions
- Tumor, Tumor, Solid, Metastasis, Metastatic Cancer
- Trial name
- NCT05103358
- Testing
- nab-sirolimus
- Where
- 166 sites in United States, Puerto Rico and 1 more
- Volunteers
- 128 people
- Sponsor
- Aadi Bioscience, Inc.
Cystic Fibrosis
MP-376 (Aeroquin™, Levofloxacin for Inhalation) in Patients With Cystic Fibrosis
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- NCT01180634
- Testing
- Aeroquin
- Where
- 112 sites in United States, Australia and 3 more
- Volunteers
- 330 people
- Sponsor
- Amgen