Clinical trials: Congenital Malformation
Facioscapulohumeral Muscular Dystrophy
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
Study A083-02 is a multi-center, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of locally-acting ACE-083 in patients with Facioscapulohumeral muscular dystrophy…
- Trial name
- NCT02927080
- Testing
- ACE-083
- Where
- 24 sites in United States, Canada and 1 more
- Volunteers
- 95 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Urea Cycle Disorders, Inborn, Inborn Errors of Metabolism
Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
Hyperammonemia, which can cause brain damage, occurs in many different kinds of inborn errors of metabolism. The investigators propose to determine if short-term (3 day) treatment with N-carbamylglutamate can diminish…
- Conditions
- Urea Cycle Disorders, Inborn, Inborn Errors of Metabolism, Propionic Acidemia, Methylmalonic Acidemia
- Trial name
- NCT01341379
- Testing
- N-carbamylglutamate
- Volunteers
- healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Polycystic Kidney, Type 1 Autosomal Dominant Disease, Polycystic Kidney, Type 2 Autosomal Dominant Disease
Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease
Sirolimus (SIR) has lead to a reduction of overall kidney size, a decrease in cyst density and general tubular cell proliferation in animal models, and to a reduction of the increase in creatinine and blood urea nitrogen by 34…
- Trial name
- RAP (NCT02055079)
- Testing
- Sirolimus
- Where
- Austria
- Volunteers
- 68 people
- Sponsor
- Medical University of Vienna
Blood and Lymphatic Diseases
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the…
- Trial name
- NCT05145062
- Testing
- BIVV003, ST-400
- Where
- 7 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sangamo Therapeutics
Fragile X Syndrome
Trial of Sertraline to Treat Children With Fragile X Syndrome
This study is a control trial of sertraline (Zoloft) in fragile X syndrome children aged 2 years to 5 years 8 months old. The trial is six months long, and each participant will receive a series of tests at both the beginning and…
- Trial name
- NCT01474746
- Testing
- Sertraline
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Randi J. Hagerman, MD
Skin Laxity
Hybrid Fractional Laser for Treatment of Off Face Body Skin Quality
To demonstrate effectiveness of hybrid fractional laser for the treatment of poor skin quality on the decolletage, back of the hands and lower legs
- Trial name
- NCT03969485
- Testing
- Hybrid Fractional Laser
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Sciton
Duchenne Muscular Dystrophy (DMD)
Peabody Developmental Motor Scaling In Children With Period Duchenne Muscular Dystrophy
To perform the reliability and validity study of the Peabody Developmental Motor Scale (Peabody Developmental Motor Scales-2) in children with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT05990608
- Testing
- Peabody Developmental Motor Scales-2, PDMS
- Where
- Turkey (Türkiye)
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Ondokuz Mayıs University
Severe Combined Immunodeficiency Syndrome
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency
This study will evaluate a new method for delivering gene transfer therapy to patients with severe combined immunodeficiency disease (SCID) due to a defective adenosine deaminase (ADA) gene.
- Trial name
- NCT00018018
- Testing
- CD34+ cells transduced with ADA retrovir
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Acute Gout Flare
Study Utilizing Rilonacept in Gout Exacerbations
This purpose of this clinical research was to determine the efficacy and safety of an experimental drug called Rilonacept in participants with an acute gout attack. Participants participated in this study for 30 days.
- Trial name
- SURGE (NCT00855920)
- Testing
- Rilonacept, Indomethacin
- Where
- 44 sites in United States
- Volunteers
- 225 people
- Sponsor
- Regeneron Pharmaceuticals
Prader-Willi Syndrome
Study of Emotion and Cognition Abilities of Children With PWS and Proposition of an Innovative Remediation
The present project project is divided in two parts. The primary aim of the part 1 of this study is to evaluate emotional control abilities of children with Prader-Willi syndrome (PWS) aged from 9 to 15 years and to study…
- Trial name
- PRACOM1 (NCT04526379)
- Testing
- Questionnaires, t-VNS
- Where
- France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital, Toulouse
Hemophilia B
HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061).
- Trial name
- NCT03569891
- Testing
- AAV5-hFIXco-Padua, Factor IX (FIX)
- Where
- 33 sites in United States, Belgium and 6 more
- Volunteers
- 67 people
- Sponsor
- CSL Behring
Fetal Growth Retardation, Preeclampsia
Management of Early-onset Fetal Growth Restriction: Angiogenic Factors Versus Feto-placental Doppler
This is a multicentre, open-label, randomized controlled trial. A total of 340 singleton pregnancies with an EFW ≤10th percentile between 26+0 and 31+6 weeks will be recruited and randomly allocated to either the control or the…
- Conditions
- Fetal Growth Retardation, Preeclampsia, Placenta Diseases
- Trial name
- earlyGRAFD (NCT05284474)
- Testing
- soluble fms-like tyrosine kinase to placental growth factor ratio (sFlt-1/PlGF)
- Where
- 27 sites in Spain
- Volunteers
- 340 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Huntington's Disease
A Study in Subjects With Late Prodromal & Early Manifest HD to Assess the Safety, Tolerability, pk, and Efficacy of Pepi
The purpose of this study is to evaluate the safety, tolerability, PK, and efficacy of Pepinemab in subjects with late prodromal and early manifest Huntington's disease.
- Trial name
- SIGNAL-HD (NCT02481674)
- Testing
- VX15/2503
- Where
- 32 sites in United States, Canada
- Volunteers
- 301 people
- Sponsor
- Vaccinex Inc.
Cystic Fibrosis, Cystic Fibrosis-related Diabetes
FEED-Cystic Fibrosis (FEED-CF)
The goal of this study is to determine the extent to which excess dietary sugars serve as a precipitating factor in glucose intolerance in adults with cystic fibrosis (CF), a population at especially high risk for a unique form…
- Trial name
- FEED-CF (NCT05766774)
- Testing
- Low-added sugar, high-fat diet, High-added sugar, high-fat CF diet
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Emory University
Tuberous Sclerosis Complex, Lymphangioleiomyomatosis
Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)
This study is an observational registry designed to gather information about Tuberous Sclerosis Complex (TSC) and Lymphangioleiomyomatosis (LAM) in pregnant women and their child.
- Trial name
- NCT06160310
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- David M. Ritter
Patent Foramen Ovale, Migraine
Comparison of the Effect of Medication Therapy in Alleviating Migraine With Patent Foramen Ovale
Migraine attack is an episodic disorder that affects approximately 12% of the population. Previous studies have shown that 41-48% of migraineur have a combination of patent foramen ovale (PFO).
- Trial name
- COMPETE (NCT05546320)
- Testing
- Aspirin 300mg, Metoprolol 25mg, Clopidogrel 75mg
- Where
- 39 sites in China
- Volunteers
- 1,000 people
- Sponsor
- Chinese Academy of Medical Sciences, Fuwai Hospital
Tetralogy of Fallot
Comparison of RVOT Gradient Under Anaesthesia With Post-operative Gradient in Patients Undergoing TOF Repair
The primary objective of the study will be to compare intraoperative post TOF repair RVOT gradient under two different anaesthetic depths.
- Trial name
- NCT03234582
- Where
- India
- Volunteers
- 20 people
- Sponsor
- Post Graduate Institute of Medical Education and Research, Chandigarh
Colorectal Cancer, Hereditary Non-polyposis Colon Cancer
Educational CD-ROM Compared With Standard Informed Consent for Patients With Colorectal Cancer or a Family History of Colorectal Cancer
RATIONALE: The use of a CD-ROM may help patients with colorectal cancer or a family history of colorectal cancer make informed decisions about undergoing microsatellite instability (MSI) testing.
- Trial name
- NCT00450424
- Testing
- counseling intervention, CD-ROM intervention
- Where
- 3 sites in United States
- Volunteers
- 239 people
- Sponsor
- Fox Chase Cancer Center
Hearing Loss, Mental Retardation
Postnatal HCMV Infection in Very Preterm Infants. Implications, Morbidity, Growth and Neurodevelopmental Outcomes.
The aim of this study is to investigate short and long term consequences from early postnatal HCMV infection transmitted via human milk in very preterm infants (birth weight < 1500 g or gestational age < 32 weeks).
- Conditions
- Hearing Loss, Mental Retardation, Microcephaly, Chorioretinitis
- Trial name
- NCT01151462
- Where
- Norway
- Sponsor
- Oslo University Hospital
Pre-Eclampsia, HELLP Syndrome
Evaluation of the Serum Markers sFLt1 and PlGF for the Prediction of the Complications of the Placental Vascular Pathologies in the 3rd Quarter of the Pregnancy.
The pre-eclampsia is a frequent pathology, concerning approximately 5 % of the pregnancies.The pre-eclampsia can evolve into severe maternal and\or foetal complications and is a major cause of mortality.
- Conditions
- Pre-Eclampsia, HELLP Syndrome, Fetal Death, Eclampsia
- Trial name
- PRECOPE (NCT03455387)
- Testing
- sampling of the serum marker sFlt1 and PlGF
- Where
- 7 sites in France
- Volunteers
- 233 people
- Sponsor
- Centre Hospitalier Metropole Savoie
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.