Clinical trials: Congenital Disorder
Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia
Trial to Evaluate the Effect of ALN-PCSSC Treatment on Low Density Lipoprotein Cholesterol (LDL-C)
This study is a Phase II, placebo-controlled, double-blind, randomized trial in 480 participants with atherosclerotic cardiovascular disease (ASCVD) or ASCVD-risk equivalents (for example, diabetes and familial…
- Conditions
- Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia, Diabetes
- Trial name
- ORION-1 (NCT02597127)
- Testing
- ALN-PCSSC, Normal Saline
- Where
- 54 sites in United States, Canada and 3 more
- Volunteers
- 501 people
- Sponsor
- The Medicines Company
Severe Bronchopulmonary Dysplasia
Aerosolized Albuterol Use in Severe BPD
Currently several dose schedules of Albuterol are administered via nebulization to infants in the neonatal and infant intensive care unit (N/IICU).
- Trial name
- NCT02766673
- Testing
- Albuterol Sulfate, Sterile Saline
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Children's Hospital of Philadelphia
Tuberous Sclerosis Complex, Seizures
An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive the same dose of GWP42003-P.
- Trial name
- NCT02544750
- Testing
- GWP42003-P
- Where
- United States
- Volunteers
- 199 people
- Sponsor
- Jazz Pharmaceuticals
Epidermolysis Bullosa
ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0) cream in the treatment of skin lesions in participants with Epidermolysis Bullosa.
- Trial name
- NCT02384460
- Testing
- SD-101-6.0 cream
- Where
- 38 sites in United States, Australia and 11 more
- Volunteers
- 169 people
- Sponsor
- Scioderm, Inc.
Developmental Disabilities, Weight Loss
Weight Management for Adolescents With IDD
The goal of this study is to explore the impact of two different diets (conventional vs. enhanced stop light) and two different delivery systems (face-to-face vs.
- Conditions
- Developmental Disabilities, Weight Loss, Body Weight, Down Syndrome
- Trial name
- IDDAdol (NCT02561754)
- Testing
- Enhanced Stop Light Diet (eSLD), Conventional Diet (CD), Face-to-face (FTF)
- Where
- 2 sites in United States
- Volunteers
- 110 people
- Sponsor
- University of Kansas Medical Center
Fragile X Syndrome, FXS
A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome
This is a single-center, randomized, double-blind, 2-period crossover study to explore the effects of BPN14770 on cognitive function and behavior in subjects with Fragile X Syndrome.
- Conditions
- Fragile X Syndrome, FXS, Fra(X) Syndrome
- Trial name
- NCT03569631
- Testing
- BPN14770
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Tetra Discovery Partners
Anemia, Sickle Cell
Examining Cognitive Function and Brain Abnormalities in Adults With Sickle Cell Disease
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes and may lead to organ failure.
- Trial name
- NCT00528801
- Testing
- NP Battery, MRI
- Where
- 12 sites in United States
- Volunteers
- 212 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Leukemia, Myelodysplastic Syndromes
Helical Tomotherapy, Fludarabine Phosphate, and Melphalan Followed By Allo-HSCT in Hematological Malignancies
RATIONALE: Giving chemotherapy drugs, such as fludarabine phosphate and melphalan, and HT before a donor stem cell transplant helps stop the growth of cancer cells.
- Trial name
- NCT00544466
- Testing
- fludarabine phosphate, melphalan, allogeneic hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- City of Hope Medical Center
Iron Overload, Sickle Cell Disease
Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron…
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Trial name
- NCT02443545
- Testing
- Deferiprone
- Where
- 13 sites in United States, Canada and 3 more
- Volunteers
- 134 people
- Sponsor
- ApoPharma
Eczema, Atopic Dermatitis
Study Testing Response Effect of KY1005 Against Moderate-to-Severe Atopic Dermatitis, The STREAM-AD Study
This is an interventional, randomized, parallel group, treatment, Phase IIb, double blind, 5-arm study to assess the effect of Anti-OX40L Monoclonal Antibody (KY1005) in adult participants with moderate to severe atopic…
- Trial name
- STREAM-AD (NCT05131477)
- Testing
- Amlitelimab
- Where
- 103 sites in United States, Australia and 10 more
- Volunteers
- 390 people
- Sponsor
- Kymab Limited
Neonatal Abstinence Syndrome
Fetal and Infant Effects of Maternal Buprenorphine Treatment
This research will track the longitudinal neurobehavioral development of the buprenorphine-exposed fetus across gestation through 1 month of age in an effort to determine the safety of this medication for use during gestation…
- Trial name
- NCT01561079
- Testing
- Buprenorphine
- Where
- 2 sites in United States
- Volunteers
- 127 people
- Sponsor
- Johns Hopkins University
Atopic Dermatitis, Healthy Volunteers
Safety Study of Tezepelumab (AMG 157) in Healthy Adults and Adults With Atopic Dermatitis
This study is a single dose escalation study of tezepelumab (AMG 157) in healthy adults (Part A) and adults with moderate to severe atopic dermatitis (Part B).
- Trial name
- NCT00757042
- Testing
- Tezepelumab
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Amgen
Thyroid Eye Disease
Twenty-four Hour Intraocular Pressure Patterns in Patients With Uncontrolled Thyroid Eye Disease
Patients with Thyroid Eye Disease (TED) often have enlarged extraocular muscles and higher orbital fat contents due to their disease process.
- Trial name
- NCT01798966
- Testing
- SENSIMED Triggerfish
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Sensimed AG
Cystic Fibrosis
To Assess the Safety, Tolerability and Pharmacokinetics of AZD5634 Following Inhaled and Intravenous (IV)Dose Administration
This is a Phase 1, first-in-human (FIH) single ascending dose study being conducted to better understand the safety, tolerability and pharmacokinetics of AZD5634 in healthy subjects
- Trial name
- NCT02679729
- Testing
- AZD5634 for inhalation, AZD5634 for infusion
- Where
- 2 sites in United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Homozygous Familial Hypercholesterolemia
A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
This study was a Phase III,A two-part (double-blind placebo-controlled/open-label) multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in subjects with homozygous familial hypercholesterolemia (HoFH).
- Trial name
- ORION-5 (NCT03851705)
- Testing
- Inclisiran Sodium for injection
- Where
- 13 sites in Hong Kong, Israel and 6 more
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Hereditary Tyrosinemia, Type I
Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)
The purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product 1) and Nitisinone 10 mg Tablets 'Baked' for 6 months @ 40°C/75% RH (Test Product 2) are bioequivalent to the reference product Orfadin 10 mg…
- Trial name
- NCT02750345
- Testing
- Nitisinone, Nitisinone Baked Tablet, Orfadin
- Where
- South Africa
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Neurofibromatosis 1
Medical Treatment of "High-Risk" Neurofibromas
Patients with neurofibromatosis type 1 (NF1) commonly develop non-cancerous tumors called plexiform neurofibromas. These tumors can be defined as "high-risk" when they result in severe pain, physical disability, organ dysfunction…
- Trial name
- NCT00846430
- Testing
- Peg-Interferon alpha-2b, Celecoxib (Celebrex), Temozolomide (temodar)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Corewell Health West
Huntington Disease
PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).
- Trial name
- NCT04556656
- Testing
- Pridopidine
- Where
- 58 sites in United States, Austria and 9 more
- Volunteers
- 499 people
- Sponsor
- Prilenia
Cystic Fibrosis
Safety and Efficacy of Sildenafil in Cystic Fibrosis (CF) Lung Disease
The purpose of this study is to determine whether sildenafil can decrease inflammation in CF lung disease.
- Trial name
- NCT00659529
- Testing
- sildenafil
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- National Jewish Health
Growth Hormone Deficiency, Panhypopituitarism
Growth Hormone and Endothelial Function in Children
Objective: This study is designed to determine whether growth hormone treatment in children 8 to 18 years of age alters function of the lining of the arteries.
- Conditions
- Growth Hormone Deficiency, Panhypopituitarism, Short Stature
- Trial name
- NCT00373386
- Testing
- growth hormone
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Ohio State University
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