Clinical trials: Newborn
Smith-Lemli-Opitz Syndrome
Simvastatin Therapy in Smith-Lemli-Opitz Syndrome
This study will evaluate the safety and effectiveness of simvastatin in treating children with Smith-Lemli-Opitz syndrome (SLOS).
- Trial name
- NCT00064792
- Testing
- Simvastatin Susp., OraPlus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Forbes Porter, M.D.
Atopic Dermatitis
Measuring Adherence to Topical Therapy in Children With Atopic Dermatitis and the Impact of a Return Visit
The purpose of this research study is to better understand how the study drug works when people use it to treat atopic dermatitis.
- Trial name
- NCT00654355
- Testing
- tacrolimus ointment
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Wake Forest University
Equinus Contracture
The Effect of Botulinum Toxin A Injections on Ankle Dorsiflexion Following Internal Fixation of Tibial Pilon Fractures
The purpose of this study is to determine if botulinum toxin type A (Botox) injections, at the time of surgery for pilon fractures, will improve ankle range-of-motion and functionality.
- Trial name
- Botox (NCT02051933)
- Testing
- Botulinum toxin type A, sodium chloride
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Wake Forest University Health Sciences
Neonatal Abstinence Syndrome, Aromatherapy
The Effect of Aromatherapy on Neonatal Abstinence Syndrome and Salivary Cortisol Levels
Determine the effectiveness of lavender and chamomile aromatherapy of mitigation of symptoms of Neonatal Abstinence Syndrome
- Trial name
- NCT03097484
- Testing
- lavender and chamomile essential oils
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- John M. Daniel
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
Congenital Adrenal Hyperplasia
Nevanimibe HCl for the Treatment of Classic CAH
This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT03669549
- Testing
- Nevanimibe hydrochloride
- Where
- 11 sites in Brazil, Czechia and 3 more
- Volunteers
- 15 people
- Sponsor
- Millendo Therapeutics US, Inc.
Gaucher Disease
Auto-antibodies Prevalence and CD1 Role in Gaucher Disease
Hypergammaglobulinaemia is frequently observed in type 1 Gaucher disease (GD1), being either polyclonal or monoclonal gammopathies. Polyclonal hypergammaglobulinemia may be related to the presence of autoantibodies.
- Trial name
- NCT02650219
- Testing
- genetic analyses
- Where
- 11 sites in France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Hospital St. Joseph, Marseille, France
Lymphoma
A Study of INCB050465 in Relapsed or Refractory Follicular Lymphoma
The purpose of this study is to assess the objective response rate of parsaclisib treatment in participants with relapsed or refractory follicular lymphoma.
- Trial name
- CITADEL-203 (NCT03126019)
- Testing
- Parsaclisib
- Where
- 83 sites in United States, Australia and 11 more
- Volunteers
- 126 people
- Sponsor
- Incyte Corporation
Congenital Heart Defects
Is Levosimendan Superior to Milrinone Regarding Acute Kidney Injury After Cardiac Surgery for Congenital Heart Disease?
The aim of the study is to assess the ability of Levosimendan to reduce the postoperative acute kidney injury in pediatric patients undergoing surgery for congenital heart disease (CHDs).
- Trial name
- MiLe-1 (NCT02232399)
- Testing
- Levosimendan, Milrinone
- Where
- 2 sites in Finland, Sweden
- Volunteers
- 72 people
- Sponsor
- Göteborg University
Cystic Fibrosis
A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis
This phase IV, multicenter, randomized, open-label, two-period crossover study will evaluate the comparable efficacy and safety of Pulmozyme (dornase alfa) delivered by the eRapid nebulizer system in patients with cystic…
- Trial name
- NCT01712334
- Testing
- dornase alfa [Pulmozyme®]
- Where
- 16 sites in United States
- Volunteers
- 99 people
- Sponsor
- Genentech, Inc.
Skin Laxity
Clinical Assessment of Skin Tightening and Contour Change of Submental Tissue Using Bipolar Radiofrequency Microneedling
The purpose of this study is to evaluate the safety and efficacy of bipolar fractional radiofrequency treatment via use of the Profound System to achieve skin tightening and contour change in lax submental (beneath the chin)…
- Trial name
- NCT04477187
- Testing
- Dermal Handpiece
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Adrenal Insufficiency, Excess Androgen
Comparison of Cortisol Pump With Standard Treatment for Congenital Adrenal Hyperplasia
Background: Congenital adrenal hyperplasia (CAH) is a genetic disorder of the adrenal gland. The adrenal gland is located in the abdomen and produces small amounts of hormones such as cortisol, aldosterone, and androgen.
- Conditions
- Adrenal Insufficiency, Excess Androgen, Congenital Adrenal Hyperplasia (CAH)
- Trial name
- NCT01859312
- Testing
- Hydrocortisone (Solucortef), Insulin pump (Medtronic)
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
A Study of NIDO-361 in Patients With SBMA
The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).
- Trial name
- PIONEER KD (NCT06411912)
- Testing
- NIDO-361
- Where
- 5 sites in Denmark, Italy and 2 more
- Volunteers
- 54 people
- Sponsor
- Nido Biosciences, Inc.
Atopic Dermatitis
SAR231893-LPS15497- "Dupilumab Effect on Sleep in AD Patients"
Primary Objective: To evaluate the effect of dupilumab on sleep quality in adult participants with moderate to severe atopic dermatitis (AD).
- Trial name
- NCT04033367
- Testing
- Dupilumab
- Where
- 42 sites in United States, Australia and 8 more
- Volunteers
- 188 people
- Sponsor
- Sanofi
Accelerated Phase Chronic Myelogenous Leukemia, Blastic Phase Chronic Myelogenous Leukemia
Etanercept in Treating Young Patients With Idiopathic Pneumonia Syndrome After Undergoing a Donor Stem Cell Transplant
This phase II trial is studying how well etanercept works in treating young patients with idiopathic pneumonia syndrome after undergoing a donor stem cell transplant.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Blastic Phase Chronic Myelogenous Leukemia, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission
- Trial name
- NCT00309907
- Testing
- etanercept, methylprednisolone
- Where
- 26 sites in United States
- Volunteers
- 39 people
- Sponsor
- Children's Oncology Group
Cystic Fibrosis Related Diabetes, Pancreatic Insufficiency
Pilot and Feasibility Study for the Treatment of Pre-diabetes in Patients With Cystic Fibrosis
The purpose of this study is to provide the necessary data and experience to design a larger, full scale clinical trial to determine if a certain medicine (repaglinide), which increases the amount of insulin secreted by the…
- Trial name
- NCT00763412
- Testing
- repaglinide
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Arbelaez, Ana Maria
Skin; Deformity
RES Prepared With RECELL® Compared to Conventional Care for Healing of Donor Sites in Ages 1-16 Years
To evaluate whether the time to complete closure is superior for RECELL-treated split-thickness donor sites, compared with Control (standardized dressings only).
- Trial name
- NCT03624192
- Testing
- RECELL® Autologous Cell Harvesting Device, Telfa™ Clear and Xeroform™ dressings
- Where
- 7 sites in United States
- Volunteers
- 3 people
- Sponsor
- Avita Medical
Cystic Fibrosis
Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion
The objective of this study was to evaluate the safety and efficacy of VX-661in combination with ivacaftor in participants with cystic fibrosis (CF) who are homozygous for F508del cystic fibrosis transmembrane conductance…
- Trial name
- NCT02070744
- Testing
- VX-661, Ivacaftor
- Where
- 23 sites in United States
- Volunteers
- 40 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hemophilia
A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis
Primary Objective: To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA)…
- Trial name
- ATLAS-PPX (NCT03549871)
- Testing
- Fitusiran, BPA prophylaxis, Factor (FVIII or FIX) prophylaxis
- Where
- 35 sites in United States, Australia and 13 more
- Volunteers
- 80 people
- Sponsor
- Genzyme, a Sanofi Company
Charcot-Marie-Tooth Disease, Hereditary Neuropathy With Liability to Pressure Palsies
Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.
- Conditions
- Charcot-Marie-Tooth Disease, Hereditary Neuropathy With Liability to Pressure Palsies, Genetic Disorders
- Trial name
- NCT01401257
- Testing
- PXT3003 Low dose, PXT3003 Intermediate Dose, PXT3003 High Dose
- Where
- 6 sites in France
- Volunteers
- 80 people
- Sponsor
- Pharnext S.C.A.
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