cyrilzakka/clinical-trials huggingface dataset:
Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)

The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis) by transplantation of a single dose of autologous CD34+ cells transduced ex vivo with the G2ARTE lentiviral vector expressing the DCLRE1C cDNA.


Eligibility Criteria:
Inclusion Criteria:

* Patient to 47 months
* SCID patients with confirmed biallelic mutations in the Artemis (DCLRE1C) gene even in the case of leaky forms characterised by a residual activity
* Absence of an HLA genoidentical donor or without rapidly available HLA-compatible unrelated donor (within six weeks of diagnosis)
* The patient can be treated by gene therapy without delay in case of active life threatening infections compromising the short-term prognosis and for which the delay in finding a phenoidentical donor is incompatible with the patient's condition of health. Active life threatening infections are defined as: viral respiratory infection, CMV infection, adenovirus infection, disseminated BCGitis or other infections grade ≥ 4 according to CTCAE scale
* Beneficiary of a social security scheme
* Parental, guardian's patient signed informed consent.

Exclusion Criteria

* Unwillingness to return for follow-up during the first 2 years study and the long term follow-up
* HIV-1 or 2 or HTLV1 infections
* Hypersensitivity to G-CSF, busulfan or Fludarabine
* Unable to tolerate general anesthesia and/or marrow harvest or peripheral blood stem cell collection (apheresis) or insertion of central venous catheter.
Clinical Results of Odyliresin (Iresine Celosia) in Symptomatic Benign Prostatic Hyperplasia

Odyliresin (Iresine Celosia) is a cytochrome-flavoprotein with a powerful anti-oxydant action on cells, has a therapeutic effect on BPH-related LUTS, reducing symptoms, prostate volume, improving the patient's quality of life and eventually limiting the number of patients who require surgery or endoscopy after medical treatment.

The aim of this phase II open clinical trial was to determine if Odyliresin, given its unique chemical structure, has a therapeutic effect on BPH-related LUTS, so as to reduce symptoms, prostate volume, improve the patient's quality of life and eventually to limit number of patients who require surgery or endoscopy after medical treatment. To this aim, patients who seek specialist advice for LUTS with no previous surgical treatment for BPH have been selected for treatment with Odyliresin in addition to an alpha-antagonist, in order determine whether at 12 months a clinical result is obtained, if after 6 and 12 months BPH symptoms are significantly improved, and a reduction of prostatic volume can be observed, thus changing the usual course of the disease. All patients with BPH-related LUTS received both alphalitic and Odyliresin treatment, clinical parameters and uroflowmetry parameters significantly improved, adenoma volume and post-void residual volume were significantly reduced at 12 month. This is the first clinical experience with Iresine Celosia in BPH.

Eligibility Criteria:
Inclusion Criteria:

* 45 years of age or older
* Clinically diagnosed with mild to moderate BPH
* Prostatic volume ≥ 30 ml determined by transrectal ultrasound
* Maximum flow rate (Qmax) \< 15 ml/sec for a voided volume 150-500 ml

Exclusion Criteria:

* Participants must not have severe BPH (IPSS symptom score \>21)
* Participants should not be currently undergoing any other form of medical therapy for BPH (5-PDE inhibitors, mepartricine, plant extracts such as Saw Palmetto, vitamin E, and quercetin).
* Patients must not have undergone prior transurethral resection of the prostate (TURP).
* Post void residual (PVD) \> 200 ml
* Previous urological history including urethral stricture disease and/or bladder neck disease, urinary retention, bladder stone, chronic prostatitis, bladder cancer, interstitial cystitis, active upper tract stone disease causing symptoms, insulin-dependent diabetes mellitus and non-controlled non-insulin-dependent diabetes mellitus, chronic renal failure
Kidney Protection Using the RenalGuard® System in Cardiac Surgery

This is a prospective randomised control trial 1:1 in patients at risk of developing Acute Kidney Injury after cardiac surgery comparing the RenalGuard® System to current medical treatment. 110 patients will be recruited for each group. The aim of the study is to assess whether the RenalGuard® system reduces Acute Kidney Injury (AKI) after cardiac surgery as compared to current practice.

Patients undergoing elective or in-house urgent cardiac surgery will be approached for the KIDNEY study if they fulfil the inclusion criteria. Upon consent pre-operative blood tests will be as per the usual practice. Moreover, 2ml of the serum will be stored for NGAL analysis.

Following the consenting process, patients will be randomised (envelop- based) into either having the RenalGuard® System (Study group) or current medical management (Control group).

Study group:

Patients in the study group will have the RenalGuard® System started in anaesthetic room once the peripheral line and arterial lines are sited. The RenalGuard® System will continue to run throughout the cardiac procedure in the operating room and up to 6 hours post-op after the patient has been transferred to the unit. Patients will be managed at a zero balance i.e. volume of the urine output will be matched to the volume of Hartmann's fluid infusion. Forced diuresis is then initiated.

Control group:

Patients in the control group will be managed as per current medical practice which will include no forced diuresis in operating room and the use of inotropes for maintenance of mean arterial pressure and intravenous furosemide for diuresis.

An additional blood test (NGAL) at six hours post-op will be taken and would require 2mls of blood. The NGAL specimens will be dealt with by the biochemistry lab (centrifuged \& stored) so that the specimens can be analysed in batches.

Patients kidney function (U\&Es) will be tested at the time of post-operatively as per usual practice (Day1, pre-discharge and as clinically indicated) and at the post-operative surgical out-patient visit.

Pre-operative, intra-operative and post-operative data will be collected for each patient group.

Eligibility Criteria:
Inclusion Criteria:

1. Patients undergoing surgery (elective or in-house urgent)
2. Patient 18 years old and over
3. Patient able to give written consent
4. Patient at risk of developing AKI after cardiac surgery (at least one factor)

   1. Diabetics (IDDM or NIDDM) with normal kidney function pre-op
   2. Patients with eGFR 20-60
   3. Patients undergoing combined cardiac procedures when the CPB time is likely to exceed 120 minutes
   4. Patients with Hb of 12.5 g/dl or below
   5. Logistic Euroscore of 5 and above
5. Patient not involved with another study

Exclusion Criteria:

1. Emergency surgery
2. Patient unable to give written consent
3. Patient already dialysis dependent or eGFR \<20
4. Patient partaking in another study
5. Pregnant patients
Human Samples and Data Repository

This protocol will enable IRB (institutional review board) review of proposed research using human samples and data collected under the terminated NIH studies 04-EI-0065 and 96-EI-0096. Data and samples may include demographic and personal health information, psychological or psychiatric testing, blood, urine, CSF or other body fluids or tissues and results of medical and physiological evaluation and medical imaging.

Data and sample analyses are limited to those approved under the original protocols unless additional specific institutional review board approval is obtained.

Objective: This protocol will enable prospective IRB review of research using human samples and data collected under other branch protocols.

Study population: Participants that were followed in terminated branch protocols under which data or samples were collected.

Design: Samples and data will be brought under this protocol, analyzed and stored for uses specified in the original protocols and as additionally approved by the IRB.

Outcome: The outcome of this protocol will be the storage, tracking, and utilization of samples and data obtained under terminated protocols.

Purpose: The purpose of this protocol is to enable prospective IRB review of research using human samples and data collected under other branch protocols.

Participants: Participants who previously participated in NIH protocols whose wishes regarding use of their samples or data for research other than that specified in the initial protocol is not known may be enrolled in this protocol. At this time, there are no additional analyses planned outside of the original protocols, apart from those listed below. Participant consent or IRB approval for a waiver of additional informed consent will be sought prior to initiation of additional analyses.

Eligibility Criteria:
* Data Analysis Study
The Effect of Povidone-iodine Ophthalmic Surgical Prep Solution on Respiration in Children Undergoing Strabismus Surgery With General Anesthesia.

Determine whether the application of povidone-iodine ophthalmic solution onto the ocular surface causes a change in respiration in children undergoing strabismus surgery with general anesthesia.

Hypothesis: The application of povidone-iodine ophthalmic solution to the ocular surface causes a change in respiration in children during general anesthesia prior to strabismus surgery.

The study is randomized and single blind. One hundred children were randomly assigned to have either povidone-iodine (group A) or balanced salt solution (BSS) (group B) eye drops instilled into their eyes during anesthesia just prior to strabismus surgery. After induction of anesthesia and insertion of an LMA while the patient was at a steady state of anesthesia and spontaneously breathing sevoflurane in oxygen, 3 drops of either povidone-iodine or BSS was instilled into each eye (right eye first). Respiratory rate was determined by measuring the peak-to-peak interval on the capnograph. This is similar to calculating heart rate by measuring the R-R interval on an ECG. HR, BP, SaO2, EtCO2, and expired sevoflurane were measured in each patient. Medical history information collected included history of prior surgeries, co-existing diseases, or apnea. Appropriate statistical analyses were performed for comparisons between the groups with respect to respiratory rate, HR, BP, expired sevoflurane, and co-existing diseases.

Eligibility Criteria:
* Inclusion Criteria

  * Children between the ages of 1 year and 17 years
  * Scheduled for strabismus surgery
  * Anesthesia plan includes inhalational induction with sevoflurane and the use of a laryngeal mask airway (LMA) with spontaneous ventilation, per the attending anesthesiologist.

Exclusion Criteria

* History of an adverse reaction to iodine
* History of any thyroid disease
* Patients who require tracheal intubation, as determined by the attending anesthesiologist; e.g. craniofacial anomalies.
* Patients with a contraindication to sevoflurane, such as malignant hyperthermia or severe left ventricular dysfunction.
* Inability or unwillingness of the subject or legal guardian/ representative to give informed consent.
Humeral Compression/Distraction Device

The proposed study of humeral shaft fractures in adults will examine outcomes in patients treated with the Humeral Trauma Nail System to treat displaced humeral shaft fractures (displaced OTA code 12A, B, or C), nonunions, or malunions. The device to be studied uses an internal gear system controlled wirelessly by rare earth magnets, which typically offers distraction for lengthening. In the humerus however, during the treatment of acute fractures, this percutaneously inserted nail can also offer compression during the healing process, thereby negating the effect of gravity, which may in turn lead to a faster and more consistent rate of union. Furthermore, in cases of slower healing the dynamic properties of the nail may employ an "accordion technique" (alternating compression-distraction, a feature unique to this implant.

The primary end point will be the union rate without secondary procedures. The hypothesis is that with the compression and distraction that this device makes possible, the investigators can achieve a higher union rate than with other forms of treatment, using a percutaneous approach, with a decreased complication rate, and can dynamically modulate the healing response when needed. The primary safety endpoint is the occurrence of adverse events from the time of treatment.

During the healing process an external remote controller (ERC) will be used to continually compress or distract the fracture as needed. The surgeon will monitor healing and decide, clinically based on sequential radiographs during subsequent office visits, whether the fracture needs additional compression or distraction, an accordion technique, or no additional external manipulation via the ERC. Participants will be followed for 12 months.

Eligibility Criteria:
Inclusion Criteria:

* Adult skeletally mature patients 18 years or older, treated by the Orthopaedic Trauma Service at Tampa General Hospital, and the Trauma Service at St. Louis University Hospital

  * Open or closed displaced humeral shaft fractures (displaced OTA code 12A, B, or C), nonunions or malunions
  * amenable to treatment with an intramedullary nail

Exclusion Criteria:

* Intra-articular extension of the fracture, prohibiting treatment with an intramedullary nail;
* Pathologic fracture
* Retained hardware
* Patients with pacemakers
* Age under 18 years.
* Patients who are prisoners or likely to become prisoners
* Homeless, or those likely to have difficulty making follow-up appointments
* Pregnancy
* Ipsilateral nerve or vascular injuries
* Immunocompromised patients
Study to Assess Safety, Pharmacokinetics, and Efficacy of Oral CC-223 for Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma or Multiple Myeloma

The main purpose of this first human study with CC-223 is to assess the safety and action of a new class of experimental drug (dual mTOR inhibitors) in patients with advanced tumors unresponsive to standard therapies and to determine the appropriate dose and tumor type for later-stage clinical trials.

Initially, patients will be treated with oral CC-223 for one month. During this time, various tests (involving blood and urine collections, ECGs, etc) will be performed. Those whose tumors stabilize or regress may continue receiving treatment for as long as they benefit from CC-223. Different dose levels of CC-223 will be tested in a dose-rising study design.

Eligibility Criteria:
Inclusion Criteria:

* Histologically-confirmed advanced solid tumor, Non-Hodgkin Lymphoma or multiple myeloma
* Patients have not tolerated or progressed on standard therapy, and no further standard therapy is available
* Archival and screening tumor biopsy
* Eastern Cooperative Oncology Group (ECOG) performance status 0-1 (solid tumors), 0-2 (hematologic malignancy)
* Adequate organ function

Exclusion Criteria:

* Prior systemic cancer-directed treatments or investigational drugs within 4 weeks or 5 half lives, whichever is shorter, prior to starting study drug or who have not recovered from side effects of such therapy. Subjects must have recovered from any effects of recent radiotherapy that might confound the safety evaluation of study drug
* Symptomatic brain metastases (prior Rx and stable metastases are OK)
* Acute or chronic liver or renal disease or pancreatitis
* Diarrhea ≥ Grade 2, impaired GI absorption
* Impaired cardiac function
* Diabetes requiring Rx, glucose \>126 mg/dL, HbA1c ≥6.5%
* Peripheral neuropathy ≥ Grade 2
* Pulmonary fibrosis
* Known HIV infection
* Known chronic hepatitis B or C virus (HBV/HCV) infection, unless comorbidity in subjects with HCC
* Pregnant, inadequate contraception
* Most concurrent second malignancies
Expanding the Cycle of Opportunity: Simultaneously Educating Parents and Children in Head Start

The CAP Family Advancement Study (CAP FAST) is a randomized control trial of CareerAdvance®, a dual-generation program providing education, career coaching, and soft-skills training for parents while their children attend CAP Tulsa's Head Start programs.

Randomization will take place with all applicants who have agreed to the study and are selected to participate in CareerAdvance®. Two-thirds of participants will be selected for the treatment group and will be able to enroll in CareerAdvance® immediately. The other one-third of the participants will be assigned to the control group and will not be able to enroll in CareerAdvance® for two years. Those who are selected for the control group will have access to CAP Tulsa's rich array of service outside of CareerAdvance®, including high quality early childhood education. The randomization process is introduced to parents during CareerAdvance® information sessions, and consent for the randomization process is required in order to apply to CareerAdvance®.

Eligibility Criteria:
Inclusion Criteria:

* Families whose children attend CAP Tulsa's Early Head Start and Head Start programs, and whose parents have applied to CareerAdvance®.

Exclusion Criteria:

* Participants who have not applied to CareerAdvance®..
First In Human Study Of Increasing Oral Doses Of PF-04634817

The study will evaluate the hypothesis that at doses and plasma concentrations which affect pharmacodynamic markers of activity at the chemokine receptors, CCR2 and CCR5, the compound is safe and well tolerated. It will also evaluate the hypothesis that the pharmacokinetic profile is robust and consistent with a once or twice a day therapeutic administration.


Eligibility Criteria:
Inclusion Criteria:

* Healthy male or female (of non-child bearing potential) subjects between 18 and 55 years of age.
* Body mass index of 17.5 to 30.5 kg/m2 and total body weight \> 50kg.

Exclusion Criteria:

* Evidence or history of any clinically significant disease.
* Treatment with an investigational drug within 30 days of study start
* Use of prescription and non-prescription medicines within 7 days of study start
CYP 2C19 Polymorphism and Voriconazole Trough Concentration in Chinese Adult Patients

To investigate the relationship between cytochrome P450 (CYP) 2C19 genetic polymorphism and the steady-state blood concentration of voriconazole in Chinese patients with invasive pulmonary aspergillosis (IPA), and to assess the effects of voriconazole trough concentration on the prognosis of IPA patients.

Each isolate of aspergillosis will be recovered from clinical specimens (sputum, bronchoalveolar lavage fluid, lung biopsy tissue) and the identification of species level will also be performed in Qilu Hospital by using conventional methods (both macroscopic and microscopic characteristics). The aspergillosis strains will be stored in 10 % glycerol broth at -80 °C. The in vitro antifungal susceptibility test of aspergillosis strains to voriconazole will be performed in the Centre for Medical Mycology and Mycoses, First Hospital, Peking University, and the performance will be according to the Clinical and Laboratory Standards Institute (CLSI) standard M38-A2 microdilution methods.

Serum galactomannan (GM) test will be performed twice per week for the first two weeks. A double-sandwich ELISA GM assay was used. A cut-off of optical density index (ODI) \>0.5 was taken as positive.

Voriconazole serum levels will be measured on day 4, day 7, day 10, and day 14 (all trough levels). In brief, quantitative analysis of voriconazole was performed using high-performance liquid chromatography coupled with tandem mass spectrometry.

Genotyping of CYP2C19 will be performed using 3 ml of peripheral blood sampled into EDTA (ethylenediaminetetraacetic acid) tubes at day 4. Genomic DNA was extracted from blood leukocytes with the use of a DNA extraction kit. Genotyping was confirmed by polymerase chain reaction (PCR)-restriction fragment length polymorphism (RFLP) analysis. Individuals can be divided into three groups according to the CYP2C19 genotype. Those who inherit two mutant CYP2C19 alleles (\*2 and/or \*3) have a reduced capacity to metabolize CYP2C19 substrates and are defined as poor metabolizers (PMs). Individuals who are homozygous (\*1/\*1) for wild-type CYP2C19\*1 or 1 wild-type allele and 1 CY¬P2C19\*17 have efficient enzymes to metabolize CYP2C19 substrates and are defined as extensive metabolizers (EMs). Subjects who are heterozygous (\*1/\*2, \*1/\*3) for wild-type CYP2C19\*1 are defined as intermediate metabolizers (IMs)

Eligibility Criteria:
Inclusion Criteria:

* Proven, probable, or possible invasive pulmonary aspergillosis (IPA)
* Acute IPA defined as duration of clinical syndrome of \<30 days.
* Treatment with voriconazole
* At least 18 years and older
* Weight \>40 kg and ≤120 kg
* Given the informed consent

Exclusion Criteria:

* Patients allergic to azole(s)
* Patients who heve been prescribed voriconazole before
* Positive urine pregnancy test (if female)
* Patients with aspergilloma or chronic aspergillosis ( \>1 month duration )
* Anticipated survival of less than 5 days or Karnofsky score \<=20
Synchrony and Reciprocity of Body Movements and Prosody Between Psychotherapist and Patient

Modern psychotherapy focuses on co-regulation, where therapist and patient interactively manage emotions. This co-regulation is seen in nonverbal communication like facial expressions, gestures, and prosody (vocal pitchs). Studies show body movements and even skin conductance synchronize between patients and therapists during sessions.

The RECiPROsody project builds on this by using technology (cameras, sensors) to record and analyze these synchronies in psychotherapy sessions. This project aims to understand how this "attunement" between therapist and patient contributes to successful therapy.

Researchers will explore how patients and therapists interact using a mobile app that records video, audio, physiological signals (heart rate), and movements. Questionnaires will assess patient well-being and therapist experience.

By studying these micro-processes, RECiPROsody hopes to gain insights into how the therapeutic relationship develops, including the connections between physical reactions, nonverbal communication, and overall progress. This knowledge can improve psychotherapeutic techniques and highlight the importance of the nonverbal communication in therapy.


Eligibility Criteria:
Inclusion Criteria:

* Male and female adults between the ages of 20 and 60 years old who are referred to the HCC Italy Clinical and Psychotherapy Research Center

Exclusion Criteria:

* Patients under 20 years of age
* Presence of paranoid or psychotic symptoms
* Admissions to psychiatric Operative Units that have occurred within the last year since the start of psychotherapy at the HCC Italy Clinical and Psychotherapy Research Center
REWARDS Premier Taxus-Liberte vs Xience V

Multicenter, retrospective registry to collect baseline, clinical, procedural, in hospital and 9-12 month follow-up data to compare major adverse cardiac events in patients receiving Promus Premier drug eluting stent to data already collected from the REWARDS-TLX Registry in which patients received either the Taxus Liberte or XIENCE V drug eluting stent.


Eligibility Criteria:
Inclusion Criteria:

* Patients 18 years of age or older
* Underwent PCI with Promus Premier (alone) DES

Exclusion Criteria:

* Underwent PCI with a non-Promus Premier DES during the same index procedure
* Patients not taking, or unable to take, dual antiplatelet therapy (aspirin plus clopidogrel, prasugrel, or ticagrelor)