Kornimate/medical-research-clean huggingface dataset:
a clinical study evaluating the safety and efficacy of cs-101 in treating subjects with β-thalassemia

Conditions: Beta-Thalassemia

Summary: the goal of this open label, single-arm clinical study is to learn about the safety and efficacy of cs-101 in treating β-thalassemia.

cs-101 is an autologous cd34+ cell suspension, edited by ex vivo base editing technology, which modifies the bcl11a binding site in hbg promoter, so that it loses the ability to bind to bcl11a, which can re-induce the production of γ-globin chain and increase the concentration of fetal hemoglobin(hbf) in the blood, compensating for the function of missing adult hemoglobin hba to achieve clinical cure. the therapy addresses two major challenges in the current treatment of the disease: lack of matching donors and graft-versus-host diseases in allogeneic hematopoietic stem cell transplantation.

Eligibility criteria:
key inclusion criteria： * 6 to 35 years old(inclusive) male or female subjects at the time of informed consenting * diagnosis of β-thalassemia, genotypes include but are not limited to β+β0，βeβ0，β0β0, etc * history of at least≥8 units/year of packed rbc transfusions in the prior 12 months prior to the screening period * generally in good condition, karnofsky performance score≥60 points for subjects≥16 years old at the time of autologous hematopoietic stem cell collection, or lansky play-performance score≥60 points for subjects under 16 years old, or equivalent clinical evaluation as the investigator site's common practice key exclusion criteria: * treatment with other investigational medications or other experimental interventions 30 days prior to signing informed consent or within 6 half-lives of the drug, whichever is longer. * subjects who have received or are receiving thalidomide and/or luspatercept, when their drug-drug interaction on the efficacy and safety of cs-101 cannot be ruled out, unless at least there are 3 test results showing the total hemoglobin level before transfusion is below 9g/dl in the past 6 months before screening. * previously received allogeneic hematopoietic stem cell transplantation or gene(edited) therapy. * subjects have available related fully matching donors and are eligible and prepared for allogeneic hematopoietic stem cell transplantation. * those with active infections, including but not limited to: hiv, hepatitis b, hepatitis c, cytomegalovirus, epstein-barr virus and treponema pallidum test positive, or known tuberculosis, parasitic infection, etc. who are judged by the investigator to be unsuitable to participate in this study. * echocardiography results with ejection fraction below 45%. * advanced liver disease, defined as： aspartate aminotransferase (ast), alanine aminotransferase (alt) \>3 × upper limit of normal (uln) or: baseline international normalized ratio (inr) \>1.5 × uln. * mri during the screening period showed heavy iron overload and is judged by the investigator to be unable to participate in the study.
mri and microbiota analysis in constipation

Conditions: Constipation

Summary: the purpose of the study is to test how well magnetic resonance imaging (mri) can measure whole gut transit time in people with constipation, and how readily it can detect a change in transit time induced by taking a dietary supplement with laxative effects.the investigators expect to demonstrate that ispaghula (psyllium) accelerates movement of matter through the intestinal tract. the investigators will also assess whether a change in gut bacteria and the chemicals that they release can be detected.


Eligibility criteria:
inclusion criteria: * meets rome iii criteria for diagnosis of functional constipation5 on questionnaire (nb. both those who do and do not meet ibs criteria will be eligible) * at least one bowel motion per week while taking usual laxatives * able to give informed consent * aged 18 or older exclusion criteria: * history declared by the candidate of other pre-existing gastrointestinal disorder, including but not limited to: * inflammatory bowel disease * coeliac disease diagnosed in the last year * pancreatitis * cancer of the gastrointestinal tract * any reported history of gastrointestinal resection (excluding appendicectomy or cholecystectomy) * presence of an intestinal stoma * pregnancy declared by candidate (no formal testing) * contraindications for mri scanning i.e. metallic implants, pacemakers, history of metallic foreign body in eye(s) and penetrating eye injury * reported alcohol intake of \>28 units/ week with daily drinking * any use of a product containing ispaghula or psyllium in the 4 weeks prior to consent * unable to avoid use of dihydrocodeine or morphine during the study * if taking other regular opiates such as codeine, fentanyl or oxycodone, participants should be able to maintain a stable dose throughout the study * any reported history of hypersensitivity or significant adverse reaction to ispaghula, maltodextrin or bisacodyl * unable to stop drugs and other agents used primarily for their laxative effect, during periods of screening, washout, baseline and treatment (maximum 15 days - rescue medication will be provided). * antibiotic or prescribed probiotic treatment in the past 4 weeks * inability to lie flat or exceed scanner limits of weight \<120kg * poor understanding of english language * participation in any medical trials for the past 3 months * any condition where the candidate is likely to require a course of antibiotics in the next 3 months e.g. severe chronic respiratory disease, recurrent urinary tract infection, lower limb ulceration * judgement by the pi that the candidate who will be unable to comply with the full study protocol e.g. diabetes, severe copd during the screening 2 weeks off laxatives * no bowel motions recorded during screening period * ≥3 complete spontaneous bowel motions (csbms - bowel motion with the feeling of complete evacuation, without the use of rescue therapy in the preceding 24 hours) per week

Keywords: constipation psyllium ispaghula magnetic resonance imaging mri microbiota
reducing obesity and type 2 diabetes in high risk youth

Conditions: Obesity; Type 2 Diabetes

Summary: compare the effect of the healt\[e\]teen program and a health\[e\]teen plus coping skills training delivered to high school students on bmi, nutrition and physical activity behaviors, and self-efficacy over 6 months.


Eligibility criteria:
inclusion criteria: * student enrolled in health, biology, or public health course exclusion criteria: * cognitive ability unable to read or respond to questions in program

Keywords: internet school based obesity prevention program adolescents
safety, attenuation and immunogenicity of gap3ko administered via a stephensi mosquitoes

Conditions: Malaria

Summary: study designed to evaluate safety and tolerability of a genetically attenuated p. falciparum (gap3ko) that arrests early in the liver stage of the parasite life cycle. study will also confirm the attenuation of the gap3ko parasites using peripheral blood smears. secondary objectives are to evaluate the humoral immune responses to gap3ko.

this single arm, open-label, phase 1 safety study is designed to evaluate the safety and tolerability of a genetically attenuated p. falciparum (gap3ko) that arrests early in the liver stage of the parasite life cycle. the study will also confirm the attenuation of the gap3ko parasites using peripheral blood smears. the secondary objectives of the study are to evaluate the humoral immune responses to gap3ko. a total of 10 healthy, malaria-naïve adult subjects will be enrolled to receive gap3ko via the bite of 150-200 gap3k0-infected a. stephensi mosquitoes under controlled conditions. subjects will be evaluated for safety, reactogenicity, and signs and symptoms of malaria to confirm attenuation for 28 days, including monitoring in a hotel setting 8-18 days post gap 3ko administration.

Eligibility criteria:
inclusion criteria: * good general health * no hematologic, hepatic, or renal disease * weight greater than 50 kg * assessment of understanding completed and passed prior to enrollment * availability and reliable access to trial center * females must use two forms of pregnancy prevention exclusion criteria: * recent (within 6 months) or planned travel to malaria endemic area * history of confirmed malaria diagnosis * anticipated use of the following: * investigational malaria vaccine at any time * malaria chemoprophylaxis within 6 months * chronic systemic immunosuppressive medications within 6 months * blood products or immunoglobulin within 120 days * systemic antibiotics with antimalarial effects within 30 days * investigational product or vaccine within 30 days * live vaccine within 28 days; killed vaccine within 14 days of gap3ko * medications known to significantly interact with chloroquine or malarone * history of: * sickle cell trait or other hemoglobinopathies * splenectomy or functional asplenia * systemic anaphylaxis * severe allergic reaction to mosquito bites or malaria treatment drugs * history of chronic or active neurologic disease * cardiac disease or stroke * clinically significant medical condition, abnormal lab results * clinically significant abnormal ecg * moderate or high risk for coronary heart disease * acute illness * pregnant or nursing female * hiv, hepatitis b, or hepatitis c * psychiatric condition that precludes compliance with the protocol * suspected or known alcohol or drug abuse * staff with direct involvement in conduct of the study or gap activities
physiopathology and sequelae of covid-19 infection

Conditions: Covid19; TSH; Cortisol

Summary: the data obtained from covid-19 infections seem to suggest that the immunogenesis of covid-19 could in some cases be the result of immune dysregulation. on the other hand, endocrine damage is possible at the tile of covid-19 infection (mainly thyroid,adrenal, and hypothalamus). these disorders are autoimmune or linked to degeneration. the main objective is to assess the thyroid function (thyrotropic axis) as well as the corticotropic adrenal function of patients who have had copvid-19 pneumonia. the secondary objectives is to describe the pathophysiological mechanisms of pulmonary and vasculothrombotic involvement of covid-19


Eligibility criteria:
inclusion criteria: * patient having a covid19 infection hospitalized at the amiens picardie university hospital * patient who is seen in pneumology consultation for a respiratory assessment at 3 months of his hospitalization (between m2 and m4) to evaluate presence of respiratory sequelae * patient who agreed to participate in the protocol * patient affiliated to a social security exclusion criteria: * patient under guardianship or curators * patient refusing to participate

Keywords: covid19 tsh cortisol
a survey on attitudes on financial incentives

Conditions: Obesity; Colon Cancer

Summary: the study concerns a survey of attitudes of the public on issues around personal responsibility for health and the use of financial incentives to promote health in the u.s. and germany. all data are gathered anonymously through online platforms. two separate professional panel providers are used for access to members of the public. all participants are people who have voluntarily joined a survey panel in the past through an informed consent process and currently serve as panel members.


Eligibility criteria:
inclusion criteria: * none other than age exclusion criteria: * none other than age

Keywords: cancer obesity personal responsibility
study to evaluate the effects of kuvan on individuals with phenylketonuria (pku) with maladaptive behaviors

Conditions: Phenylketonuria

Summary: to validate the outcome measures and the tolerability of kuvan treatment in the improvement of behavioral symptoms in 10 selected adults with phenylketonuria (pku) with or without mental retardation.


Eligibility criteria:
inclusion criteria: * biochemical confirmation of pku, * measurable maladaptive behavior at baseline, * signed informed consent, * not pregnant or lactating, * females on birth control if applicable, * willing to comply. exclusion criteria: * subjects or care provider unreliable, use of investigational products within 30 days prior to enrollment, * pregnant or lactating, * concurrent disease or condition that would interfere with study participation or safety, ie multiple psychotropic meds, any condition the pi renders the subject at high risk, on drugs that inhibit folate synthesis, * concurrent use of levodopa.

Keywords: pku, phe, pah, kuvan, trp, tyr
evaluation of covid-19 vaccination in elderly subjects in retirement home (ehpad) and long-term care units (usld) in ile-de-france

Conditions: Reside Either in Retirement Home or Long-term Care Units

Summary: the main objective of this non-interventional, multi-center study is to compare the incidence of serious health events (death, unscheduled hospitalizations) in subjects vaccinated against coronavirus with that of unvaccinated subjects, in retirement home (ehpad) and long-term care units (usld) in ile-de-france during the same observation period 12-months minimun and up to18 months according vaccinal schedule. the primary outcome will be the incidence of serious health events (death and unscheduled hospitalizations) in subjects vaccinated against coronavirus compared to that of unvaccinated subjects.

this multicenter study is a parallel cohort to the first phase of the vaccination campaign against coronavirus, in particular for elderly subjects in institutions. all subjects who will not oppose the study will be included and this date will correspond to day 0. d0 will correspond to the date of the first vaccine injection for subjects in the vaccine group. the subjects included will be followed up on d21, d90, m6, 12 months (m12) and up to 18 months in the case of 3rd vaccinal injection the following data will be collected at inclusion and during follow-up: * age, weight, sex, level of autonomy * chronic diseases * any hospitalizations * possible symptoms after vaccination for subjects who choose to be vaccinated * the fate of the subjects and the evaluation of the immune response * hospitalizations and vital status of subjects serology data prescribed by doctors as part of routine care will also be collected.

Eligibility criteria:
inclusion criteria: * subject in retirement home (ehpad) or long-term care units (usld) during the anti-coronavivus vaccination period. * subject not opposing the study or whose legal representative does not oppose the study. exclusion criteria: * subject not affiliated to a social security scheme.

Keywords: retirement home (ehpad) long-term care units (usld) coronavirus vaccination
shotblocker during intramuscular injection randomized control trial

Conditions: Neonatal Pain

Summary: the objective of this trial is to evaluate the effect of bionix shotblocker on pain of injection of the first hepatitis b vaccine in healthy newborns. shotblocker is a pain reducing tool used in babies, children, and adults for injections. swaddling during the injection and administration of oral sucrose prior to the injection are established standards of care for painful procedures in neonates. the investigators hypothesize that the use of shotblocker in addition to swaddling and oral sucrose administration will lessen the pain response.


Eligibility criteria:
inclusion criteria: * infants born at umass memorial medical center (ummmc) via vaginal delivery or cesarean delivery and under the care of the nursery team * term infants (37 to 42 weeks gestational age) * no acute illness that causes pain * apgar score above 7 at 5 minutes * have successfully attempted at least one oral feeding * no circumcision in the last 6 hours * parental consent for hepatitis b vaccine exclusion criteria: * swallow dysfunction * congenital or genetic abnormalities * infants who were exposed to sedatives within the last 12 hours * infants with skin on thigh, hand, or foot that is not intact * diagnosis of neonatal abstinence syndrome (nas) * infants who are simultaneously receiving hepatitis b immunoglobulin or nirsevimab (rsv vaccine) * department of children and families (dcf) custody

Keywords: shotblocker neonatal pain needlestick
integration of family planning and hiv services in tanzania

Conditions: HIV Infection; HIV Infections

Summary: hiv positive women and couples have broad reproductive health needs that are not always met within hiv services. the integration of family planning (fp) services into tanzania's hiv care and treatment clinics (ctc) will address the fertility desires of ctc clients and ultimately result in the reduction of unintended pregnancies and hiv incidence. one strategy for integrating fp and ctc services is to use a "facilitated referral" model. facilitated referrals are enhanced referrals for additional services that have been used in other settings and which are the preferred intervention strategy the government of tanzania would like to pilot test. this study will evaluate the feasibility, effectiveness, and costs of implementing a "facilitated referrals" intervention by examining fp use among female clients attending hiv/aids care and treatment centers. this study will measure whether there is a reduction in unmet need for contraception among female ctc clients after the facilitated referral integration intervention has been implemented.


Eligibility criteria:
inclusion criteria: 1. female clients of care \& treatment clinics with cd4 counts \>100, and are who stage i - iii. (ctc clients) 2. care and treatment providers who provide services to clients (ctc providers) 3. care and treatment supervisors who are the immediate in-charge supervisor for the ctc clinic. (ctc supervisor) 4. family planning providers who provide services to clients. (fp providers) 5. family planning supervisors who are the immediate in-charge supervisor for the fp clinic. (fp supervisor)

Keywords: ae adverse event aids acquired immunodeficiency syndrome alt (sgpt) alanine aminotransferase art antiretroviral therapy ast (sgot) aspartate aminotransferase dcf data collection forms dmc data monitoring committee fda (u.s.) food and drug administration gcp good clinical practice guidelines hb sag hepatitis b surface antigen ich international conference of harmonisation ind investigational new drug application irb institutional review board iu international units mg milligram(s) mm3 cubic millimeter(s) pcr polymerase chain reaction sae serious adverse event µg microgram uln upper limit of the normal range wb western blot to inform the ministry of health strategy for integrating fp and hiv/aids care and treatment services in tanzania hiv seronegativity
intracerebral hemorrhage acutely decreasing arterial pressure trial

Conditions: Intracerebral Hemorrhage

Summary: rationale: management of blood pressure (bp) in the acute phase of intracerebral hemorrhage (ich) remains controversial. although it has been established that there is a transient moderate reduction of perihematoma cerebral blood flow (cbf) in acute ich, the effect of bp treatment is unknown. the potential for exacerbation of cbf has precluded routine aggressive bp reduction. aim and hypothesis: the primary study aim is to demonstrate the feasibility and safety of acute bp reduction to \< 150 mmhg systolic using a standardized protocol in ich patients. it is hypothesized that ctp will not demonstrate evidence of perihematoma ischemia following acute bp reduction. design: ich adapt is a randomized blinded endpoint trial. acutely hypertensive ich patients are randomized to a target systolic bp of \< 150 mmhg or \< 180 mmhg. patients are treated with intravenous (iv) labetalol/hydralazine/enalapril. study outcomes: the primary outcome is cerebral blood flow in the perihematoma region, measured with ct perfusion, 2 hours after randomization. secondary outcomes include the difference in bp at 1 and 2 hours post-randomization in the two treatment groups and hematoma expansion rates at 24 hours. discussion: ich adapt is the only randomized trial designed specifically to identify any hemodynamic changes in the perihematoma region secondary to aggressive bp management. the results of this trial will facilitate ongoing and future studies aimed at determining the efficacy of rapid bp reduction in acute ich.


Eligibility criteria:
inclusion criteria: * age ≥18 years * acute primary ich demonstrated with ct scan * onset ≤ 24 h prior to randomization exclusion criteria: * contraindication to bp reduction i.e., severe arterial stenosis or high-grade stenotic valvular heart disease * indication for urgent bp reduction i.e., hypertensive encephalopathy, or aortic dissection * definite evidence that the ich is secondary to underlying cerebral or vascular pathology, i.e., avm, aneurysm, tumour, trauma, vasculitis, or hemorrhagic transformation of an ischemic infarct * previous ischemic stroke within 30 days of current event nb: prior ich is not an exclusion criterion * planned surgical resection of hematoma nb: extraventricular drain placement is not an exclusion criterion * contraindication to ct perfusion imaging (i.e. contrast allergy, metformin use or creatinine \>160 μmol/l)

Keywords: stroke hypertension cerebral blood flow ct perfusion
a study of qls1128 and in combination with ritonavir in healthy participants

Conditions: COVID-19

Summary: a study to evaluate safety, tolerability and pharmacokinetics of qls1128 sustained-release tablets and in combination with ritonavir tablets in healthy participants


Eligibility criteria:
inclusion criteria: * healthy male or female subjects between ages of 18-45 years. * male subjects' weight ≥ 50 kg, female subjects' weight ≥ 45 kg; body mass index ≥ 19 kg/m2 and ≤ 27 kg/m2. * female participants of child-bearing potential/ male subjects had fertile female partners must agree to use effective contraception from screening until 30 days after last dose. exclusion criteria: * participant who have a past history of covid-19 infection or are currently showing symptoms of covid-19 infection. * participant who have been treated with any sars-cov-2 neutralizing antibody or participated in clinical trials related to sars-cov-2 (e.g., sars-cov-2 neutralizing antibody, oral drug clinical trials, etc.). * participant who tested positive for sars-cov-2 nucleic acid during the screening period, or who were positive for covid-19 antibody if not vaccinated against covid-19. * participant who have a clear history of allergy to drugs, food or pollen, or are allergic to ritonavir tablets and qls1128 sustained-release tablets. * any clinical serious disease of cardiovascular, endocrine, neurological, digestive, respiratory, hematological, metabolic, psychiatric or other systems determined by the investigator may interfere with the results of this clinical trial.
organoid model predictive of response to immunotherapies

Conditions: Breast Cancer

Summary: the purpose of this study is to create models out of tissue samples and treat those models with the same immunotherapy treatment the patient will be receiving, in order to validate this process and to predict responses to therapies and use it to choose the best treatments for people in the future. the researchers will then examine the direct effects of the treatment on those models.


Eligibility criteria:
inclusion criteria: 1. ≥ 18 years old at the time of informed consent 2. ability to provide written informed consent and hipaa authorization 3. biopsy proven diagnosis of cancer 4. able to obtain at least 2 16 gage cores of fresh tissue safely (3 or more cores preferred) 5. planning to undergo standard of care immunotherapy 6. baseline standard of care ct within 8 weeks of starting immunotherapy exclusion criteria: 1. inability to provide fresh biopsy sample 2. any active infections 3. any conditions that in the opinion of treating physician and the study team will compromise the ability of the patient to receive prescribed treatment to assess the response.