Clinical trials: Newborn Diseases
Muscular Dystrophy, Oculopharyngeal (OPMD)
Continuation Protocol to Protocol BBCO-001
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized…
- Trial name
- HOPEMD (NCT02328482)
- Testing
- Tehalose 30gr
- Where
- Canada
- Volunteers
- 9 people
- Sponsor
- Bioblast Pharma Ltd.
Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia
Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
The primary objective of this study is to understand and characterize the health-related quality of life (HRQoL) and disease burden of adult participants with PK deficiency receiving routine clinical care.
- Conditions
- Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Anemia, Anemia, Hemolytic
- Trial name
- PKD (NCT04964323)
- Where
- 5 sites in United States, Italy and 3 more
- Volunteers
- 15 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Intermittent Claudication, Atherosclerotic Disease
Edwards Lifesciences Self-Expanding Stent Peripheral Vascular Disease Study
The main objective of this study is to assess the safety and effectiveness of the Edwards Lifesciences LifeStent nitinol self expandable stent device and its delivery system in the treatment of occlusive superficial femoral…
- Conditions
- Intermittent Claudication, Atherosclerotic Disease, Arterial Occlusive Disease
- Trial name
- RESILIENT (NCT00673985)
- Testing
- Percutaneous Transluminal Angioplasty, LifeStent NT™ Self-Expanding Peripheral Stent
- Volunteers
- 246 people
- Sponsor
- C. R. Bard
Alpha 1-Antitrypsin Deficiency
Pharmacokinetic Study of ARALAST (Human Alpha1- PI)
The primary purpose of this study is to characterize the pharmacokinetic profile of intravenous Aralast Fraction (Fr.) IV-1, a sterile, stable, lyophilized preparation of functionally intact human Alpha1- Proteinase Inhibitor…
- Trial name
- NCT00242385
- Testing
- Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor, Dose of 60 mg/kg alpha1-proteinase inhibitor
- Where
- 7 sites in Australia, New Zealand
- Volunteers
- 25 people
- Sponsor
- Baxalta now part of Shire
Skin Barrier to Water Loss, Skin Health
Apple Peptide Complex for Post-Surgical and Aesthetic Skin Recovery
This clinical study evaluates a topical botanical formulation for supporting skin recovery following invasive and non-invasive aesthetic procedures.
- Conditions
- Skin Barrier to Water Loss, Skin Health, Skin Discoloration, Skin Irritation
- Trial name
- APC-SKIN (NCT07807085)
- Testing
- Apple Peptide Complex Post-Aesthetic Care Protocol
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Grow Out Oils Clinical Aromatherapy Company
Thalassemia Major
Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
Patients with severe thalassemia (thalassemia major) present with severe anemia that required life-long transfusion therapy, spleen enlargement that led to increased transfusion requirement, and other serious complications as…
- Trial name
- NCT02049450
- Testing
- ruxolitinib
- Where
- 7 sites in Greece, Italy and 3 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Pompe Disease (Late-onset)
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Pompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha-glucosidase (or GAA).
- Trial name
- NCT06150820
- Testing
- No Intervention
- Where
- 53 sites in United States, Australia and 9 more
- Volunteers
- 119 people
- Sponsor
- Astellas Gene Therapies
Atopic Dermatitis
Evaluation of a Non-Invasive Device for Early Detection of Atopic Dermatitis Flares
This study will collect skin measurements from people with atopic dermatitis (AD) using the investigational Nevisense Go device.
- Trial name
- Detect-AD (NCT07645820)
- Testing
- Nevisense electrical impedance spectroscopy
- Where
- 13 sites in United States
- Volunteers
- 450 people
- Sponsor
- Castle Biosciences Incorporated
Cystic Fibrosis
A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)
This study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.
- Trial name
- NCT04969224
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 19 sites in Australia, Belgium and 2 more
- Volunteers
- 82 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Mucopolysaccharidosis (MPS)
Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)
Mucopolysaccharidosis (MPS) type II (MPS II; Hunter syndrome) is a rare, X-linked disease caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S) and occurs almost exclusively in boys, with an incidence of…
- Trial name
- NCT02095015
- Where
- 14 sites in United States, Canada and 5 more
- Volunteers
- 159 people
- Sponsor
- Shire
Pregnancy Related, Atopic Dermatitis
An Observational Retrospective Cohort Study Being Conducted in Women With Atopic Dermatitis Exposed to Ruxolitinib Cream Versus Topical Corticosteroids During Pregnancy: A US Claims Database Analysis
This study is to assess infant outcomes of women with AD who were exposed to ruxolitinib cream during pregnancy compared with a control cohort of women with AD who were exposed to a topical corticosteroid (TCS) during pregnancy.
- Trial name
- NCT06627335
- Testing
- Ruxolitinib Cream, TCS
- Where
- United States
- Volunteers
- 5,621 people
- Sponsor
- Incyte Corporation
Retinitis Pigmentosa
Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa
The purpose of this study is to evaluate the safety of intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa by a prospective, single-center, randomized, parallel, double-blind, phase I…
- Trial name
- TC/RP (NCT02280135)
- Testing
- Intravitreal injection of Autologous bone marrow Stem Cell, Subconjunctival injection of saline
- Where
- Spain
- Volunteers
- 8 people
- Sponsor
- Red de Terapia Celular
Cystic Fibrosis
Pharmacokinetic Study of Bramitob® Administered for Inhalation by PARI eFlow® vs PARI LC® PLUS Nebulizer
The purpose of this study is to assess pharmacokinetic and safety comparability of Bramitob® when administered for inhalation by using PARI eFlow® rapid electronic nebulizer vs PARI LC® PLUS nebulizer in Cystic Fibrosis Patients…
- Trial name
- NCT01116089
- Testing
- Bramitob® administered by PARI LC® PLUS nebulizer, Bramitob® administered by PARI eFlow® rapid electronic nebulizer
- Where
- 5 sites in Czechia, Moldova and 1 more
- Volunteers
- 25 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Leukemia
Vaccine Therapy and GM-CSF in Treating Patients With Acute Myeloid Leukemia in Remission
RATIONALE: Vaccines made from a peptide may help the body build an effective immune response to kill cancer cells. Colony-stimulating factors, such as GM-CSF, increase the number of white blood cells and platelets found in bone…
- Trial name
- NCT00454168
- Testing
- PR1 leukemia peptide vaccine, sargramostim
- Where
- 15 sites in United States
- Volunteers
- 244 people
- Sponsor
- The Vaccine Company
Homocystinuria Due to CBS Deficiency
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices
- Trial name
- NCT02998710
- Where
- 11 sites in United States, Ireland and 2 more
- Volunteers
- 110 people
- Sponsor
- Travere Therapeutics, Inc.
Neonatal Jaundice
Intermountain Inpatient Bilirubin Decision Accuracy Study Using Smartphone-based Measurements by Picterus Jaundice Pro
The goal of this project is to evaluate the performance of Picterus Jaundice Pro in an American population, including preterm and term newborns and pre- and post- phototherapy.
- Trial name
- NCT06329843
- Testing
- Picterus Jaundice Pro (JP)
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Picterus AS
Hemophilia A
A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates. The main aim of the study is to check for side effects from treatment with BAX855.
- Trial name
- NCT02615691
- Testing
- PEGylated Recombinant Factor VIII, ITI
- Where
- 91 sites in United States, Austria and 21 more
- Volunteers
- 120 people
- Sponsor
- Baxalta now part of Shire
Gout, Cardiovascular Diseases
Drug Use Study of Febuxostat After Recent Changes in the Prescribing Information
The prescribing information provides information on medicines. This study will check the number of patients starting febuxostat and the number of febuxostat users with cardiovascular disease after changes to the prescribing…
- Trial name
- NCT04853160
- Where
- United States
- Volunteers
- 24,046 people
- Sponsor
- Takeda
Genetic Disease, Nonsense Mutation
A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis
This is a Phase 2 open label study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 in patients with cystinosis with nonsense mutation in at least one allele.
- Conditions
- Genetic Disease, Nonsense Mutation, Cystinosis
- Trial name
- NCT04069260
- Testing
- ELX-02
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Fabry Disease
A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease
No investigational drug will be administered in this study for the treatment of Fabry disease. This will be a multicenter, multinational, non-treatment, cross-sectional study of young male patients with Fabry disease who have not…
- Trial name
- NCT01839526
- Testing
- Iohexol
- Where
- 22 sites in United States, Argentina and 12 more
- Volunteers
- 39 people
- Sponsor
- Genzyme, a Sanofi Company
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