louisbrulenaudet/clinical-trials huggingface dataset:
Clinical trial NCT06369974: Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy
Official title: An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Genetic Disease
Keywords: leukodystrophy
antisense oligonucleotide
TUBB4A-related leukodystrophy
Hypomyelination
ASO
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: n-Lorem Foundation
First posted: 2024-04-17
Enrollment: 1
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single participant interventional study
Primary purpose: TREATMENT

Brief summary: This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single pediatric participant with TUBB4A associated leukodystrophy.

Detailed description: This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single pediatric participant with a de novo pathogenic gain of function TUBB4A mutation associated with severe leukodystrophy with hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC)

Eligibility criteria: Inclusion Criteria:

* Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s).
* Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records.
* Clinical phenotype and neuroimaging consistent with a diagnosis of TUBB4A-related leukodystrophy/Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum (H-ABC)
* Documented genetic mutation in TUBB4A

Exclusion Criteria:

* Participant has any known contraindication to or unwillingness to undergo lumbar puncture
* Use of investigational medication within 5 half-lives of the drug at enrolment
* Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.

Primary outcomes:
measure: Neurological assessments
timeFrame: Baseline to 24 months
Secondary outcomes:
measure: Feeding and swallow evaluation
timeFrame: Baseline to 24 months
measure: Safety and tolerability
timeFrame: Baseline to 24 months
Other outcomes:
Locations:
Study officials:
affiliation: Massachusetts General Hospital
name: Florian Eichler, MD
role: PRINCIPAL_INVESTIGATOR
References:
Clinical trial NCT02120274: Vitamin D and Vitamin B12 Supplementation With Pegylated Interferon-Alfa Plus Ribavirin for Treating Chronic Hepatitis C
Official title: Randomized Clinical Trial to Assess the Effectiveness of Vitamin D and Vitamin B12 Supplementation in Combination With Pegylated Interferon-Alfa Plus Ribavirin for Treating Chronic Hepatitis C.
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: RNA Virus Infections
Flaviviridae Infections
Hepatitis C
Hepatitis C, Chronic
Keywords: RNA Virus Infections
Flaviviridae Infections
Hepatitis C
Hepatitis C, Chronic
Vitamin D
Cholecalciferol
Vitamin B 12
Lead sponsor: Federal University of São Paulo
Sponsor class: OTHER
First posted: 2014-04-22
Enrollment: 85
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the effectiveness of the supplementation of vitamins D and B12 in combination with Pegylated Interferon-Alfa and Ribavirin in the treatment of genotype 1 chronic hepatitis C, who do not qualify to receive protease inhibitor in Brazil.

Detailed description: Randomized clinical trial comparing rates of RVR, eRVR, EOT and SVR during standard treatment with Pegylated Interferon-Alfa plus Ribavirin compared to the same treatment supplemented with B12 and vitamin D during the treatment period.

Patients assigned to the vitamin supplementation group will receive the standard treatment of Pegylated Interferon-Alfa plus Ribavirin for 48 weeks together oral Vitamin D and intramuscular Vitamin B12. The control group will receive the same regimen of peguilated alfainterferon plus ribavirin for 48 weeks without any vitamin supplementation.

Eligibility criteria: Inclusion Criteria:

* Genotype 1 HCV infection confirmed a positive test for anti-HCV antibody and detectable serum HCV RNA by PCR
* Pegylated Interferon-Alfa treatment naïve
* Liver biopsy with Metavir F2 or less
* Cognitive capacity to understand and sign the informed consent

Exclusion Criteria:

* HBV or HIV co-infection
* Hemoglobin level less than 10 g/dL or total neutrophil count less than 1,500/mm3 or platlet count below 75,000/mm3
* Creatinin ≥ 1.5 mg/dL
* Severe cardiopathy
* Pregnancy or impossibility to use birth control methods by the couple, or breast-feeding
* Conditions that, according to the investigator's judgement, preclude participation in the study, including clinical, cognitive or behavioural conditions

Primary outcomes:
measure: SVR12
timeFrame: 12 weeks after treatment completion
Secondary outcomes:
measure: RVR, EVR, EOT
timeFrame: week 4, week 12 and at the end-of-treatment.
Other outcomes:
Locations:
city: São Paulo
country: Brazil
facility: Outpatient Clinic of Viral Hepatitis (NUPAIG)
Study officials:
affiliation: Federal University of São Paulo
name: Adauto Castelo, PhD
role: STUDY_DIRECTOR
affiliation: Federal University of São Paulo
name: Jorge F Senise, PhD
role: STUDY_CHAIR
References:
Clinical trial NCT06867874: Syncytiotrophoblast-derived Extracellular Vesicles in Placenta Previa and Accreta Spectrum
Official title: Shedding of Syncytiotrophoblast-derived Extracellular Vesicles is Increased in Placenta Previa and Accreta Spectrum: a Case-control Study.
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Placenta Previa
Placenta Accreta Spectrum
Lead sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Sponsor class: OTHER
First posted: 2025-03-10
Enrollment: 70
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: BASIC_SCIENCE

Brief summary: The main goal of this case-control study is to quantify the levels of circulating syncytiotrophoblast-derived extracellular vesicles (STBEVs) in maternal blood in a population of women with placenta previa and placenta accreta spectrum.

Detailed description: The main goal of this case-control study is to quantify the levels of circulating syncytiotrophoblast-derived extracellular vesicles (STBEVs) in maternal blood in a population of women with ultrasound diagnosis of PP with and without postpartum histopathologic diagnosis of PAS, compared to pregnant women with normal placentation.

The secondary aim of this case-control study is to characterize the multi-omics profiling of STBEVs cargo's (proteins, gene and transcripts) isolated from patients diagnosed with PP and PAS compared to the control population.

Eligibility criteria: Inclusion Criteria:

* Pregnant women with diagnosis of placenta previa and accreta spectrum (cases)
* Healthy pregnant women
* \> 18 years

Exclusion Criteria:

* \< 18 years
* Twin pregnancies
* Chronic Infections
* Cancers
* Unable to give the written consent

Primary outcomes:
measure: Concentration of circulating EVs from blood
timeFrame: 9 months (three trimester of pregnancy)
measure: Proteomics analysis of circulating EVs
timeFrame: 9 months (three trimester of pregnancy)
Secondary outcomes:
Other outcomes:
Locations:
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario A.Gemelli IRCSS
Study officials:
affiliation: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
name: Chiara Tersigni
role: PRINCIPAL_INVESTIGATOR
References:
Clinical trial NCT05953974: Validation of a Diagnostic Score for Encephalitis to Assess the Risk of Autoimmune Origin
Official title: Validation of a Diagnostic Score for Encephalitis to Assess the Risk of Autoimmune Origin
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Autoimmune Encephalitis
Autoimmune Encephalitis Caused by N-Methyl D-Aspartate Receptor Antibody (Disorder)
Keywords: Diagnostic score
auto-immune encephalitis
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2023-07-20
Enrollment: 350
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients \> or = 18 years old presenting with auto-immune encephalitis, defined according to the Graus criteria, with neuronal antibodies :

* Anti NMDAr
* Anti LGI1
* Anti CASPR2
* Anti GABAb
* Anti GAD

Brief summary: The investigators wish to test a diagnostic risk score for autoimmune encephalitis in case of encephalitis, previously validated by two American teams, in a retrospective analysis, according to the clinical and paraclinical data available in our database of the Reference Centre for Autoimmune Encephalitis and Paraneoplastic Neurological Syndromes of Professor Honnorat for patients with NMDAr, anti LGi1, anti CASPR2, anti GABAbr and anti GAD antibodies.


Eligibility criteria: Inclusion Criteria:

* Age \> 18 years old
* Patient with a definite auto-immune encephalitis (Graus and al. 2016)

Exclusion Criteria:

* Uncomplete data in acute phase
* Patient with several antibodies
* Opposition

Primary outcomes:
measure: Validation of a diagnostic score for encephalitis to assess the risk of autoimmune origin.
timeFrame: At baseline
measure: Symptoms in the acute phase
timeFrame: At baseline
measure: Mode of onset of disease
timeFrame: At baseline
measure: Results of lumbar puncture
timeFrame: At baseline
measure: Results of antineuronal antibodies in CSF
timeFrame: At baseline
measure: Results of antineuronal antibodies in serum
timeFrame: At baseline
Secondary outcomes:
Other outcomes:
Locations:
city: Lyon
country: France
facility: Centre de référence des syndromes neurologiques paranéoplasiques et encéphalites auto-immunes
Study officials:
References:
Clinical trial NCT01342874: Improved Outcomes Associated With Inositol Dietary Supplementation in Women With Gestational Diabetes Mellitus
Official title: Improved Maternal and Fetal Outcomes Associated With Inositol Dietary Supplementation in Women With Early Gestational Diabetes Mellitus
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Gestational Diabetes Mellitus
Keywords: inositol
gestational diabetes mellitus
obstetric adverse outcomes
Lead sponsor: G. d'Annunzio University
Sponsor class: OTHER
First posted: 2011-04-27
Enrollment: 30
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: pregnant women presenting random fasting glucose during the first half of pregnancy above 92 mg%

Brief summary: Objective: Insulin resistance during normal pregnancy and in gestational diabetes mellitus (GDM) are unknown. New criteria are based on fasting glucose levels since the beginning of pregnancy. Inositol, a putative second messenger of insulin, correlates with the degree of insulin resistance. Dietary supplementation of inositol improves insulin resistance in patients with GDM.

Detailed description: Methods: A randomized double-blind study was carried out in women with GDM. Patients were randomly exposed to inositol and placebo. Increase of BMI, blood glucose at 75 grams oral glucose tolerance test (OGTT), fetal and neonatal adverse outcome, and insulin treatment were correlated to inositol exposure.

Eligibility criteria: Inclusion Criteria:

* singleton pregnant women
* random fasting glucose above 92 mg%

Exclusion Criteria:

* BMI \>30
* random fasting glucose above 126 mg%

Primary outcomes:
measure: Insulin therapy in early diagnosis of GDM
timeFrame: 6 months
measure: altered OGTT
timeFrame: 6 months
Secondary outcomes:
measure: delivery and neonatal outcomes
timeFrame: 6 months
Other outcomes:
Locations:
city: Chieti
country: Italy
facility: University of Chieti
state: CH
Study officials:
affiliation: ObGyn Dept
name: Claudio Celentano, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Corrado F, D'Anna R, Di Vieste G, Giordano D, Pintaudi B, Santamaria A, Di Benedetto A. The effect of myoinositol supplementation on insulin resistance in patients with gestational diabetes. Diabet Med. 2011 Aug;28(8):972-5. doi: 10.1111/j.1464-5491.2011.03284.x.
Clinical trial NCT04885374: China Amyotrophic Lateral Sclerosis Registry of Patients With Traditional Chinese Medicine
Official title: China Amyotrophic Lateral Sclerosis Registry of Patients With Traditional Chinese Medicine
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Dongzhimen Hospital, Beijing
Sponsor class: OTHER
First posted: 2021-05-13
Enrollment: 2000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients who meet the criteria will be enrolled from multi-centers in China.

Brief summary: A multi-center registration study of clinical characteristics of amyotrophic lateral sclerosis (ALS) patients with traditional Chinese medicine (TCM).

Detailed description: The CARE-TCM is a prospective, multi-center, patient registry study. Eligible ALS patients with TCM treatments will be enrolled in the registry. After enrollment, this research collects the individual characteristics of patients' status. Longitudinal follow-up data are collected from both patients (primary caregivers) and their treating neurologists every 3 months for 5 years. The aims of the CARE-TCM are to: 1) explore the general characteristics of ALS patients with TCM and; 2) assess the effectiveness and safety of various TCMs on ALS based on nationwide registry outcome collecting process.

Eligibility criteria: Inclusion Criteria:

* Patients aged 18 years and older.
* Patients with possible, laboratory supported probable, probable, or definite ALS according to the revised El Escorial criteria.
* ALS patients treated with traditional Chinese medicine.

Exclusion Criteria:

* Patients who refuse informed consent.

Primary outcomes:
measure: Effective Survival
timeFrame: Through study completion, an average of 3 months
Secondary outcomes:
measure: Overall Survival
timeFrame: Through study completion, an average of 3 months
measure: Rate of Disease Progression
timeFrame: Through study completion, an average of 3 months
measure: Rate of Forced Vital Capacity (FVC) Decline
timeFrame: through study completion, an average of 3 months
measure: Rate of Body Mass Index (BMI) Decline
timeFrame: Through study completion, an average of 3 months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Dongzhimen Hospital, Beijing University of Chinese Medicine
Study officials:
affiliation: Dongzhimen Hospital, Beijing
name: Ying Gao, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Song Y, Li M, Sugimoto K, Han Y, Liu J, Ma B, Song H, Zhang C, Gao Y; CARE-TCM Group. China amyotrophic lateral sclerosis registry of patients with Traditional Chinese Medicine (CARE-TCM): Rationale and design. J Ethnopharmacol. 2022 Feb 10;284:114774. doi: 10.1016/j.jep.2021.114774. Epub 2021 Oct 23.
Clinical trial NCT03760874: Atrial Fibrillation Research Database
Official title: Prospective Register Study to Record Safety and Efficacy of Oral Anticoagulants in Atrial Fibrillation Patients
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Anticoagulants and Bleeding Disorders
Atrial Fibrillation
Lead sponsor: University of Campania Luigi Vanvitelli
Sponsor class: OTHER
Collaborators: name: Monaldi Hospital
First posted: 2018-11-30
Enrollment: 5000
Sex: ALL
Minimum age (years): 18
Maximum age (years): 110
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients with atrial fibrillation in clinical practice

Brief summary: All patients with atrial fibrillation who are treated with vitamin-k antagonists (warfarin, phenprocoumon) or non vitamin K oral anticoagulants (Dabigatran, Rivaroxaban, Apixaban, Edoxaban) in real world settings will be recorded in this register. Within this register a characterization of patients and therapy (with regard to medication, dose and duration) will be done. On basis of defined clinical relevant end points the long-term efficacy and safety will be evaluated.


Eligibility criteria: Inclusion Criteria:

* Patients with atrial fibrillation
* Indication for oral anticoagulant treatment

Exclusion Criteria:

* Missing Informed Consent Form
* Missing Contact Informations for Follow up

Primary outcomes:
measure: Thromboembolic Events
timeFrame: 1 year
measure: Bleeding Events
timeFrame: 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Naples
country: Italy
facility: University of Campania "Luigi Vanvitelli"
Study officials:
References:
citation: Russo V, Carbone A, Attena E, Rago A, Mazzone C, Proietti R, Parisi V, Scotti A, Nigro G, Golino P, D'Onofrio A. Clinical Benefit of Direct Oral Anticoagulants Versus Vitamin K Antagonists in Patients with Atrial Fibrillation and Bioprosthetic Heart Valves. Clin Ther. 2019 Dec;41(12):2549-2557. doi: 10.1016/j.clinthera.2019.10.008. Epub 2019 Nov 14.
Clinical trial NCT06294574: Improved Successful Retrieval Rate of HydroMARK Plus Breast Biopsy Site Marker in Comparison to HydroMARK as Well as Improved Surgeon Satisfaction
Official title: Improved Successful Retrieval Rate of HydroMARK Plus Breast Biopsy Site Marker in Comparison to HydroMARK as Well as Improved Surgeon Satisfaction
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Breast Neoplasm
Lead sponsor: University of Alabama at Birmingham
Sponsor class: OTHER
Collaborators: name: Mammotome
First posted: 2024-03-05
Enrollment: 150
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

Brief summary: The purpose of the study is to test how successful the retrieval rate of the new HydroMARK Plus Breast Biopsy Site Marker in comparison to HydroMARK.

Detailed description: Study objectives

Primary objective

Assess the successful retrieval rates of the HydroMARK Plus clip

Secondary objective

Determine the level of satisfaction of the surgeon with the retrieval of the HydroMARK Plus clip

Eligibility criteria: Inclusion Criteria:

* Patients with biopsy clips scheduled for surgery with SCOUT localization

Exclusion Criteria:

* Patients who didn't have surgery, patients younger than 18 years old
* Patients older than 90 years old
* Inmates
* Non-English speakers

Primary outcomes:
measure: Retrieval rate of the HydroMARK Plus clip.
timeFrame: Date of HydroMARK Plus Clip placement to 1 week plus or minus 5 days
Secondary outcomes:
measure: Level of satisfaction of the surgeon with the retrieval of the HydroMARK Plus clip.
timeFrame: Date of retrieval surgery to 1 week.
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: The Kirklin Clinic
state: Alabama
Study officials:
affiliation: University of Alabama at Birmingham
name: Ceren Yalniz, MD
role: PRINCIPAL_INVESTIGATOR
References:
Clinical trial NCT04019574: Study to Investigate the Effects of Single Intravenous Doses of Difelikefalin (CR845) on the QTc Interval in Healthy Subjects
Official title: A Randomized, Double-Blind, Placebo- and Positive-Controlled, Four-way Crossover Study to Investigate the Effects of Single Intravenous Doses of Difelikefalin (CR845) on the QTc Interval in Healthy Subjects
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Healthy
Keywords: Difelikefalin
CR845
QTc
TQT
Lead sponsor: Cara Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2019-07-15
Enrollment: 57
Sex: ALL
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: OTHER

Brief summary: This is a Phase 1, randomized, double-blind (except for moxifloxacin), placebo- and positive-controlled, single-site, 4-way crossover study to investigate the effects of single therapeutic and supratherapeutic IV doses of difelikefalin (CR845) on the QTc interval in healthy adult subjects. Subjects will be randomized to a treatment sequence consisting of 4 treatment periods with a minimum 5-day washout between treatments. Subjects will receive each of the study treatments over the course of the study. Randomized subjects will receive the assigned study treatment as a single dose in the fasted state in the morning on Day 1 of each treatment period.


Eligibility criteria: Key Inclusion Criteria:

* Body weight between 50 to 120 kg and body mass index within 18 to 29 kg/m2 at Screening;
* Current nonsmokers who have not used any tobacco- or nicotine-containing products (chewed or smoked) or replacement products, including, but not limited to, electronic cigarettes, in the 45 days prior to Screening.

Key Exclusion Criteria:

* Past or present diseases, which as judged by the Investigator, may affect the outcome of this study;
* Any condition or situation that, in the opinion of the Investigator, would prevent proper evaluation of the safety or efficacy of the study treatment according to the study protocol;
* History of hypersensitivity or allergy to moxifloxacin or any other study treatment or history of tendonitis or tendon rupture with moxifloxacin or any other quinolone type drug.

Primary outcomes:
measure: QTc change from baseline following CR845 treatment.
timeFrame: Baseline, Day 1
Secondary outcomes:
measure: Change from baseline in QTc with Bazett correction (QTcB).
timeFrame: Baseline, Day 1
measure: Change from baseline in Heart Rate.
timeFrame: Baseline, Day 1
Other outcomes:
Locations:
city: Dallas
country: United States
facility: Cara Therapeutics Study Site
state: Texas
Study officials:
affiliation: Cara Therapeutics
name: Frédérique Menzaghi, PhD
role: STUDY_DIRECTOR
References: