Kornimate/medical-research-clean huggingface dataset:
metabolic study of cockayne syndrome

Conditions: Cockayne Syndrome

Summary: cockayne syndrome (cs) is related to defective dna transcription and/or repair and belongs to the family of nucleotide excision repair. it is an autosomal recessive multisystemic disorder characterized by mental retardation, microcephaly, severe growth failure with lipoatrophia, sensorial impairment, cutaneous photosensitivity, dental decay, enophtalmios. the disease is progressive causing severe impairments but there's currently no therapeutics for the disease. growth failure, feeding difficulties and lipoatrophia are prognostic keys of cs but physiopathology is unknown. according to preliminary assays, our goal is to test the hypothesis that cachexia is due to hypometabolism. we also want to test the potential link between this basal metabolism modification and mitochondrial dysfunction and somatotrope axis, and correlation between the basal metabolism degree and global severity of the disease.


Eligibility criteria:
cockayne patients: inclusion criteria : * male and female with cockayne syndrome * age\>6 months exclusion criteria: * intercurrent diseases * subject in period of exclusion * pregnancy and breastfeeding control subjects : inclusion criteria : * sister or brother of cockayne patients * age\>6 months exclusion criteria: * intercurrent diseases * subject in periods of exclusion * pregnancy and breastfeeding

Keywords: rest energetic cost measured by indirect calorimetry hormonal axis mitochondrial oxidative phosphorylation basal metabolism
a glomerular filtration rate (gfr) measuring study of rta 402 in chronic kidney disease (ckd) patients with type 2 diabetes mellitus

Conditions: Chronic Kidney Disease; Type 2 Diabetes

Summary: a phase 2 glomerular filtration rate (gfr) measuring study of rta 402 in chronic kidney disease (ckd) patients with type 2 diabetes mellitus.

an exploratory study to investigate the effects of rta 402 on glomerular filtration rate in ckd patients with type 2 diabetes mellitus. to evaluate the safety of rta 402.

Eligibility criteria:
inclusion criteria: * ckd patients with type 2 diabetes mellitus * patients whose egfr levels are eligible for this study * patients being treated with stable dose of ace inhibitors and/or arb etc. exclusion criteria: * patients with type 1 diabetes * patients with known non-diabetic renal disease * patients with a history of renal transplantation * patients with mean sbp \> 160 mmhg or mean dbp \> 90 mmhg * patients with hba1c \> 10% * patients with cardiovascular disease specified in the study protocol * patients for whom inulead® injection is contraindicated etc.

Keywords: ckd patients with type 2 diabetes
perioperative blood pressures and chronic hypertension

Conditions: Hypertension

Summary: this study aims to assess the performance of presurgical clinic blood pressure measurements in predicting home blood pressure readings among surgical patients. it also aims to measure the association between home blood pressures and patient perspectives on blood pressure control.

a convenience sample of presurgical patients seen in a preadmission testing clinic were enrolled and sent home with a home blood pressure monitor that stored up to 50 readings. participants were asked to return the bp cuff on the day of their surgery.

Eligibility criteria:
inclusion criteria: * ambulatory patients \>= 21 years of age presenting for surgery to a tertiary care preadmission testing clinic. exclusion criteria: * inability or unwillingness to independently operate a home blood pressure monitor.

Keywords: hypertension perioperative care
role of nlrp3 in periodontitis and diabetes

Conditions: Metabolic Disease; Periodontal Diseases

Summary: nlrp3 plays a key role in endothelial function and may be a link for the known interaction of periodontitis and diabetes. the investigators compared the impact of gingival health, periodontitis (cp), diabetes or of both diseases (cp +diabetes) on saliva and serum nlrp3levels.

the aim of this study was to evaluate a possible association between nlrp3 and gingival health, periodontitis (cp), diabetes or of both diseases (cp +diabetes) on saliva and serum nlrp3 levels.

Eligibility criteria:
inclusion criteria: * presence of at least 15 teeth * cp with a minimum of 40% of sites with a clinical attachment level (cal) ≥2mm and probing depth (pd) ≥4mm; * presence of at least ≥2 mm of crestal alveolar bone loss verified on digital periapical radiographs * presence of ≥40% sites with bleeding on probing (bop) exclusion criteria: * intake of contraceptives * intake of immunosuppressive or anti-inflammatory drugs throughout the last three months prior to the study * status of pregnancy or lactation * previous history of excessive drinking * allergy to local anaesthetic * intake of drugs that may potentially determine gingival hyperplasia such as hydantoin, nifedipine, cyclosporin a or similar drugs.
correlation between clinical and electrophysiological phenotypes in a population of patients with neuropathy charcot-marie-tooth disease type 1a

Conditions: Charcot-Marie-Tooth Type 1A Neuropathy

Summary: this exploratory cross-sectional study proposes, firstly, to objectify in a population of charcot-marie-tooth disease type 1a (cmt 1a)if there is a correlation between the recording of electrical parameters and upper limb muscle strength of the thigh and in a second step, to seek a relationship between the measured parameters.

patients suffering from cmt 1a neuropathy will be invited to go through a series of tests such as: * electromyography * isokinetic test * podiatric assessment * functional evaluations: hand dexterity, limbs muscles strength * quality of life assessment with questionnaires

Eligibility criteria:
inclusion criteria: * patients older than 18 years * diagnosis of charcot marie tooth disease, type 1a confirmed by molecular biology (duplication 17.p11.2) * listening and written french * agreement after written information, clear and honest about the purpose of the study, the nature of the tests and their possible side effects or bothersome. * strength of the quadriceps superior to 2/5 mmt mrc exclusion criteria: * presence of other neurological comorbidity * presence of coronary artery disease unstabilized * peripheral neuropathy of other causes: diabetes, monoclonal gammopathy, malignancy, solid cancer, systemic autoimmune disease (lupus, sjögren's disease, wegener sarcoidosis ...), infectious disease (viral hepatitis, hiv ...), drugs known to be responsible for iatrogenic neuropathy. * gait trouble of other origin * patients unable to give their consent. * intellectual deficit that does not allow to comply with tests

Keywords: charcot-marie-tooth disease type 1a neuropathy isokinetic electromyography walking
probiotics for eradication of carbapenem resistant klebsiella pneumonia

Conditions: Anti-Biotic Resistance; Klebsiella Pneumoniae

Summary: infection by klebsiella pneumonia producing class a carbapenemases is a major clinical and public health problem in israel and worldwide. the aim of this study is to determine the safety and efficacy of alteration of the gut microflora by probiotics, alone or in combination with mechanical bowel cleansing, as a strategy to eradicate colonization of the gastrointestinal tract by crkp. we hypothesize that administration of probiotics will decrease the rate of gi colonization by crkp.

crkp colonization will be determined by culture and pcr of rectal swabs. the study will include 3 arms - no intervention, probiotics and mechanical bowel cleansing + probiotics. followup examination of rectal swab samples for the presence of crkp will be performed at 4 weeks and 12 weeks from study entry.

Eligibility criteria:
inclusion criteria: 1. subjects who are \> 18 years of age. 2. signing of informed consent by subject or legal custodian. 3. colonization of the gastrointestinal tract by crkp as determined by stool culture or pcr of stool for the blakpc gene. 4. negative cultures for crkp from extra-intestinal sites excluding urine. exclusion criteria: 1. subjects who have participated in another clinical trial within the last three months. 2. women with childbearing potential unless using adequate contraception (iud, oral or depot contraceptive or barrier plus spermicide). 3. subjects who are pregnant or breast feeding. 4. subjects who will be unavailable for the duration of the trial, are likely to be non-compliant with the protocol, or who are felt to be unsuitable by the investigator for any other reason. 5. subjects with chronic diarrhea (\>4 weeks). 6. subjects with inflammatory bowel disease. 7. subjects whose stool is positive for clostridium difficile toxin. 8. subjects receiving immunosuppressive treatment (i.e. corticosteroids; azathioprine or 6-mp; cyclosporine; tacrolimus; okt3, methotrexate; anti tnf agents; chemotherapy). 9. subjects who underwent solid organ or hematopoietic stem cell transplantation. 10. subjects with primary or secondary immunodeficiency disorders, including hiv. 11. subjects with chronic advanced cardiac, respiratory, renal or liver disease. 12. subjects with advanced malignant disease. 13. subjects with severe acute organ failure. 14. subjects in whom crkp is isolated from sites other than stool or urine (i.e. blood, sputum or wound cultures).

Keywords: carbapenem resistant klebsiella pneumoniae carpapenemases probiotics vsl3
thalidomide at low dose for the treatment of patient with myelodysplastic syndromes - thal-smd-200

Conditions: Myelodysplastic Syndromes

Summary: the gfm previously conducted a dose-escalating phase ii trial of thalidomide in mds with a minimum dose of 200mg/d and a maximum dose 800mg/d. responses were evaluated according to iwg criteria at week 16 and thalidomide continued up to week 56 in responders. 82% patients received at least 8 weeks of treatment and were evaluable. 59% had hematological improvement, mainly on the erythroid lineage (increase of hemoglobin). most responses were observed at low doses and between 4 and 8 weeks. the objectives of this trial (thal-smd-20) are to evaluate the efficacy and tolerance of lower doses thalidomide in low risk mds patients with transfusion-dependant anemia.

thalidomide: first part of the trial: 82 patients at 200mg/day given at bedtime x 12 weeks, decreased to 100mg/day if grade 1 or 2 side. stopped temporally for 1 week if grade 3 or 4 side effects. then reintroduced at the same dose. if side effects again, definitively stopped. responses evaluated at 12 weeks according to iwg criteria for the erythroid lineage at week 12: * if no hematological improvement (hi): increased to 300mg/day for 8 weeks and then eventually to 400mg/day for 8 weeks more, if no hi. * if hematological improvement (hi): continued at the same dose. second part of the trial: 30 patients treated at 50mg/day x 12 weeks. responses evaluated at 12 weeks according to iwg criteria for the erythroid lineage at week 12: * if no hematological improvement (hi): increased to 100mg/day for 8 weeks and then eventually to 200mg/day for 8 weeks more, if no hi. * if hematological improvement (hi): continued at the same dose.

Eligibility criteria:
inclusion criteria: * patients ≥18 years, with ipss low or int-1 mds * transfusion dependant anemia above 2 packed red blood cells (prbc)/month * ecog index = 0, 1, 2 * no peripheral neurological disease exclusion criteria: * mds patients with ipss int-2 or high * patients with less than 2 packed red blood cells (prbc)/month * patients with previous history of venous thrombosis * patient treated with epo +/- g-csf in the 2 months before inclusion in the protocol * patient having received intensive chemotherapy in the 3 months before inclusion in the protocol * patient having received thalidomide in a previous protocol * patient presenting an iron, b12 vitamin or folic acid uncorrected deficiency * patient with peripheral neurological disease * patient not being able to subject itself to a regular clinical and biological follow-up * pregnant patient or patient in a period of lactation * patient refusing to take a contraceptive treatment through out all the study * patient receiving drugs able to interfere with the mechanism of action of thalidomide * patient refusing to sign the informed consent.

Keywords: low risk myelodysplastic syndromes mds bone marrow diseases thalidomide cytopenias anemia
a clinical study to compare long-term efficacy and safety of an aliskiren based regimen to a hydrochlorothiazide based treatment regimen with optional addition of amlodipine

Conditions: Hypertension

Summary: this study is designed to compare the long-term efficacy and safety of an aliskiren based regimen to an hctz based treatment regimen with optional addition of amlodipine in patients with essential hypertension


Eligibility criteria:
inclusion criteria: * patients with essential hypertension exclusion criteria: * severe hypertension * history or evidence of a secondary form of hypertension * history of myocardial infarction. * other protocol-defined inclusion exclusion criteria also apply.

Keywords: hypertension, aliskiren, hydrochlorothiazide, amlodipine
postoperative positioning after surgery for macular holes

Conditions: Macular Holes

Summary: idiopathic macular holes are an important cause of visual loss. macular holes can be treated by surgically removing the vitreous gel and injecting intraocular gas. following macular hole surgery, face-down positioning is often advised with the aim of improving the likelihood of macular hole closure. the current evidence of postoperative positioning protocols is insufficient to draw firm conclusions and guide practice. the investigators wish to compare non-face-down positioning and face-down positioning after surgery for macular holes in a randomized trial. hypothesis: non-face-down positioning is equivalent to face-down positioning after surgery for macular holes.


Eligibility criteria:
inclusion criteria: * idiopathic macular hole stage ii-iv * informed consent * duration of symptoms ≤ 24 months exclusion criteria: * previous vitreomacular surgery * myopia ≥ 8 diopters * ocular trauma * disease affecting visual function (proliferative diabetic retinopathy, diabetic macular edema, exudative macular degeneration)

Keywords: macular holes non face down positioning face down positioning
tibulizumab skin healing and inflammation evaluation for lasting defense

Conditions: Hidradenitis Suppurativa (HS)

Summary: the study is a phase 2, multi-center, randomized, double-blind, placebo-controlled study to evaluate the effects of tibulizumab over 16 weeks (period 1) in adults with hidradenitis suppurativa, followed by a 16-week open-label extension period in which all participants will receive tibulizumab (period 2)


Eligibility criteria:
inclusion criteria: * male or female, 18 to 70 years of age * ≥6-month history of hidradenitis suppurativa (hs) * total an (abscesse and inflammatory nodule) count ≥5 * hs lesions in ≥2 distinct anatomical areas, at least one of which is hurley stage ii or iii exclusion criteria: * draining tunnel count \>20 * presence of another inflammatory condition or a skin condition that may interfere with study assessments * known to have immune deficiency or is immunocompromised * evidence or suspicion of active or latent tuberculosis * history of opportunistic, chronic, or recurrent infection requiring chronic antibiotic use, serious or life-threatening infection within 2 months, or had an infection requiring systemic antibiotics within 2 weeks * has active systemic candidiasis * unable to tolerate subcutaneous drug administration

Keywords: hidradenitis suppurativa hs tibulizumab hidradenitis suppurativa clinical response hiscr skin diseases inflammatory skin disease chronic skin condition sweat gland disease skin diseases bacterial suppurative hidradenitis hidradenitis acne acne inversa inversa
effects of trunk stabilization versus activation exercises on pain and disability in postpartum lumbo-pelvic pain

Conditions: Pelvic Pain

Summary: to compare the effects of trunk stabilization versus activation exercises on pain and disability in postpartum lumbo-pelvic pain.

effects of stabilizing exercises on postpartum pelvic floor muscle function, lumbo-pelvic pain and disability. in results, stability exercises can help women with lpp enhance the function of their pfms. pregnancy and childbirth weakens pfms and disrupts the load-transfer system in the lumbo-pelvic area. the use of pfms in the right pattern can help these muscles operate better. local stabilizing muscles are targeted during stabilization exercises, which increase pelvic motor control and stability. as per researcher knowledge there was limited evidence present on the comparison of trunk stabilization and trunk activation exercises. therefore, this study was conducted to find the effectiveness of trunk stabilization versus activation exercises on disability and pain in postpartum women with lumbo-pelvic pain.

Eligibility criteria:
inclusion criteria: * age 20-40 * female after postpartum * two or more positive posterior provocation test * active straight leg raises * pain onset during pregnancy exclusion criteria: * orthopedic or rheumatologic disorders * intervertebral disc pathology * neoplasm or previuos surgery of spine * history of fracture.

Keywords: lumbo-pelvic pain postpartum stabilization exercises trunk activation exercises
a trial to investigate the dose-linearity of biochaperone® combo 75/25 and the safety at three different doses in subjects with type 2 diabetes

Conditions: Type 2 Diabetes Mellitus

Summary: this is a bicentric, double-blinded, randomised, four-period crossover phase 1 trial, using automated 30-hour euglycemic clamp in subjects with type 2 diabetes mellitus.

this is a bicentric, double-blinded, randomised, four-period crossover phase 1 trial, using automated 30-hour euglycemic clamp in subjects with type 2 diabetes mellitus. each subject will be randomly allocated to a sequence of four treatments, three single doses of biochaperone® combo 75/25 (0.6 u/kg, 0.8 u/kg or 1.0 u/kg) and one single dose of humalog® mix25 at 0.8 u/kg on four separate dosing visits. subjects will come in a fasted state to the clinical trial centre in the morning of each dosing day and stay at the clinical trial centre until the 30-hour clamp procedures have been terminated.

Eligibility criteria:
inclusion criteria: * male or female subject aged 18-70 years (both inclusive) * type 2 diabetes mellitus (as diagnosed clinically) for ≥ 12 months * hba1c level between 6.5% and 9.0 % (both inclusive) * body mass index between 20.0 and 40.0 kg/m2 (both inclusive) * body weight \<= 125.0 kg at the screening visit * insulin-treated subjects. total insulin dose of \<= 1.2 (i)u/kg/day exclusion criteria: * type 1 diabetes mellitus * known or suspected hypersensitivity to imp(s) or related products * previous participation in this trial. participation is defined as randomised. * receipt of any medicinal product in clinical development within 60 days before randomisation in this trial. * clinically significant abnormal values for haematology, biochemistry, coagulation, or urinalysis as judged by the investigator considering the underlying disease. * supine blood pressure at screening (after resting for at least 5 min in supine position) outside the range of 90-160 mmhg systolically or 50-95 mmhg diastolically (excluding white-coat hypertension; therefore, if a repeated measurement shows values within the range, the subject can be included in the trial); symptoms of arterial hypotension and/or a heart rate at rest outside the range of 50-90 beats per minute. this exclusion criterion also pertains to subjects being on anti-hypertensives. * women of child bearing potential not willing to use contraceptive methods.
adaptive therapy for resected stage ⅰb-ⅱ nsclc patients with undetectable mrd

Conditions: NSCLC

Summary: it is a prospective, multicenter, randomized controlled trial to investigate the predictive value of mrd on adjuvant therapy. stage ib-ii resected egfr/alk negative nsclc patients will undergo two-round mrd tests after surgery, first in 3-7 days and second in 1 month after surgery. and patients who confirm two-round landmark undetectable mrd will be enrolled and randomly assigned to the conventional treatment group or the adaptive therapy group . patients in the the adaptive therapy group will undergo closely mrd and imaging monitoring without adjuvant therapy if the mrd was negative.


Eligibility criteria:
inclusion criteria: * stage ib-ii non-small cell cancer patients who after complete resection. * egfr/alk negative * two-round mrd tests confirm landmark undetectable mrd * expected survival ≥24 months * ecog ps 0-1 * willing to accept mrd monitoring every 3 months for a total of 2 years after surgery exclusion criteria: * patients who had previously undergone radiotherapy or chemotherapy or any other anti-tumor therapy * patients with a history of other malignancies in the past 5 years * any unstable systemic disease (eg, active infection, high-risk hypertension, unstable angina, congestive heart failure, etc.) or other conditions that investigators considered would limit the ability of the patient to participate in the study

Keywords: mrd ctdna
metoprolol treatment for cytokine release syndrome in patients treated with chimeric antigen receptor t cells

Conditions: Solid Tumor; Hematological Malignancy

Summary: the aim of this prospective study is to evaluate the feasibility and efficacy of metoprolol, a beta-1 adrenergic receptor blocker, in the treatment of cytokine release syndrome (crs) caused by chimeric antigen receptor t (car t) cell infusions, its effects on the serum levels of interleukin-6 (il-6) and other cytokines.


Eligibility criteria:
inclusion criteria: * patients with lymphoma, leukemia or other malignant diseases who were enrolled for car t cell therapy. exclusion criteria: patients with contraindications indicated in metoprolol instruction, including: * significant bradycardia (heart rate \< 45/min) * cardiogenic shock * severe or acute heart failure * poor peripheral circulation perfusion * grade ii or iii atrioventricular block * sick sinus syndrome * severe peripheral vascular disease

Keywords: metoprolol cytokine release syndrome chimeric antigen receptor t cells
aripiprazole in patients with psychosis associated with parkinson's disease

Conditions: Parkinson's Disease; Psychoses

Summary: the purpose of this clinical research study is to assess the safety and tolerability of aripiprazole in patients with psychosis associated with parkinson's disease.


Eligibility criteria:
inclusion criteria: * primary diagnosis of idiopathic parkinson's disease. * psychosis related to parkinson's disease. exclusion criteria: * psychosis present prior to diagnosis of parkinson's disease.

Keywords: psychosis, parkinson's disease