Kornimate/medical-research-clean huggingface dataset:
long-term follow-up for rgx-121

Conditions: Mucopolysaccharidosis II

Summary: rgx-121-5101 is the long-term follow-up study to the rgx-121-101 first in human study where participants received rgx-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene (ids) to the central nervous system. this study will evaluate the long-term safety and efficacy of rgx-121.

this is a prospective follow-up study to evaluate the long-term safety and efficacy after a single administration of rgx-121. eligible participants are those who previously have enrolled in clinical study rgx-121-101 and received a single intracisternal (ic) or intracerebroventricular (icv) infusion of rgx-121. enrollment of each participant will occur the same day or after the participant has completed the end of study (eos) visit or early termination visit (et) from the previous (parent) study. participants will be followed in this study cumulatively for up to 5 years after rgx-121 administration (inclusive of the parent study) or until rgx-121 is commercially available in the participant's country, whichever occurs first. no treatment will be directed under this observational protocol. the total study duration for each participant may vary depending on when they enroll in the current study following rgx-121 administration in the parent study.

Eligibility criteria:
inclusion criteria: * to be eligible, a participant must have previously received rgx-121 in a separate parent trial. * participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent exclusion criteria: * patient has not received rgx-121 previously in a separate parent trial.

Keywords: mps ii gene therapy hunter
safety and efficacy study of teduglutide in subjects with short bowel syndrome who completed protocol cl0600-004 (nct00081458)

Conditions: Short Bowel Syndrome

Summary: the purpose of this clinical study is to evaluate the long-term safety and efficacy of daily administration of teduglutide.

teduglutide is an analog of naturally occurring human glucagon-like peptide-2 (glp-2), a peptide secreted by l-cells of the distal intestine. glp-2 is known to increase intestinal and portal blood flow, and inhibit gastric acid secretion. teduglutide binds to the glucagon-like peptide-2 receptors located in intestinal subpopulations of enteroendocrine cells, subepithelial myofibroblasts and enteric neurons of the submucosal and myenteric plexus. activation of these receptors results in the local release of multiple mediators including insulin-like growth factor (igf)-1, nitric oxide and keratinocyte growth factor (kgf). this multicenter, double-blind, international, phase iii trial will have a treatment period of 28 weeks. subjects in this study are those who received teduglutide or placebo in protocol cl0600-004 (nct00081458). these subjects will receive daily subcutaneous injections of 0.05 milligrams or 0.10 milligrams of teduglutide per kilogram of body weight. subjects will have visits every 4 to 6 weeks and will be assessed for parenteral nutrition (pn) reduction with a follow-up period of 4 weeks duration for those subjects who do not complete this protocol, or do not enter into the long-term safety extension protocol cl0600-010.

Eligibility criteria:
inclusion criteria: at dosing week 24 of protocol cl0600-004 (nct00081458), subjects will be reviewed for their participation in this study. subjects who meet all of the following criteria can be enrolled in this study: * signed and dated informed consent form (icf) to participate before any study-related procedures are performed * completion of protocol cl0600-004 (nct00081458) exclusion criteria: * history of cancer or clinically significant lymphoproliferative disease with fewer than 5 years documented disease-free state * history of alcohol or drug abuse (within previous year) * prior use of native glucagon-like peptide 2 (glp-2) within 3 months of screening visit * pregnant or lactating women * any condition or circumstance, which in the investigator's opinion would put the subject at any undue risk, prevent completion of the study, or interfere with analysis of the study results

Keywords: short bowel syndrome parenteral nutrition sbs teduglutide
effect of hepatic and renal impairment on the pharmacokinetics, safety and tolerability of bay1841788 (odm-201)

Conditions: Pharmacokinetics; Hepatic Insufficiency; Renal Insufficiency

Summary: evaluate the potential effect of hepatic or renal impairment on the pharmacokinetics, safety and tolerability of bay 1841788 (odm-201).

the study was closed after part 1 because additional investigation in volunteers with moderate renal impairment in part 2 was not deemed to be ethically or scientifically justified.

Eligibility criteria:
inclusion criteria: * all subjects -- male and white subjects between 45 and 79 years of age with a body mass index between 18 to 34 kg/m\*2 (both inclusive). * patients with moderate hepatic impairment (part 1) -- patients with documented liver cirrhosis confirmed by histopathology, e.g., previous liver biopsy, laparoscopy, ultrasound, or fibroscan and with moderate hepatic impairment (defined as child pugh class b). * patients with severe renal impairment (part 1) -- patients with severe renal impairment with an estimated glomerular filtration rate 15-29 ml/min/1.73 m\*2, who are not on dialysis and are not expected to start dialysis in the next 3 months (stage 4). * healthy subjects -- healthy as determined by the investigator or medically qualified designee based on a medical evaluation including medical history, physical examination, laboratory tests and cardiac monitoring and with estimated glomerular filtration rate \>90 ml/min (according to modified diet of renal disease equation). * patients with moderate renal impairment (part 2) -- patients with moderate renal impairment with an estimated glomerular filtration rate 30-59 ml/min/1.73 m\*2 (stage 3). * patients with mild renal impairment (part 2) -- patients with mild renal impairment with an estimated glomerular filtration rate (egfr) 60-79 ml/min/1.73 m\*2 (stage 2). * patients with mild hepatic impairment (part 2) * patients with documented liver cirrhosis confirmed by histopathology, e.g., previous liver biopsy, laparoscopy, ultrasound, or fibroscan. * patients with mild hepatic impairment (defined as child pugh class a). exclusion criteria: * severe cerebrovascular or cardiac disorders, e.g., myocardial infarction less than 6 months prior to dosing, congestive heart failure of new york heart association (nyha) grade iii or iv. * subjects with percutaneous transluminal coronary angioplasty or coronary artery bypass graft less than 6 months prior to study drug administration. * strong cytochrome p450 (cyp) 3a4 inhibitors or strong cyp3a4 inducers within 28 days or 5 drug half-lives (if drug half-life in patients is known), before start of study treatment. * known bcrp (breast cancer resistant protein) and oatp (organic anion-transporting polypeptide) substrates not specifically mentioned in the protocol within 28 days or 5 drug half-lives (if drug half-life in patients is known), before start of study treatment. * smoking more than 20 cigarettes daily.
hip fracture impact on vascular events in noncardiac surgery patients: a cohort evaluation (hip vision): pilot study

Conditions: Hip Fractures

Summary: hip vision (pilot study) is a prospective observational cohort study to evaluate the incidence of overall and cause-specific mortality among consecutive patients aged ≥ 18 years presenting with hip fracture to the juravinski hospital of the hamilton health sciences. this pilot study will assess the feasibility of a larger prospective international cohort study. after eligibility has been confirmed and informed consent has been obtained, participants will be registered in the study. troponin level, complete blood counts and serum creatinine level will be collected at day 1 through day 10 post admission. cam instrument will be employed at admission and once daily post admission day 1 through day 10. fim instrument will be administered within 72 hours of admission to establish pre-fracture functional independence and disability. patients will be contacted by research personnel by telephone 30 days after study registration.


Eligibility criteria:
inclusion criteria: 1. patients aged ≥ 18 years with hip fractures (i.e. fractures involving the subcapital, femoral neck, or intertrochanteric regions), treated either operatively or non-operatively 2. mechanism of injury consistent with either: 1. a fall from a standing height or; 2. another mechanism of injury which, in the clinical judgment of an orthopedic surgeon, would impart the same or less traumatic energy as a fall from a standing height exclusion criteria: 1. patients with fractures isolated to the proximal femoral shaft, with no involvement of the intertrochanteric, femoral neck, or subcapital region 2. patients with hip fractures resulting from high energy mechanisms, such as motor vehicle accidents or falls from a substantial height 3. patient who refuse 30-day or 6-month follow up 4. patients who refuse to consent either by themselves or through a substitute decision-maker (for patients unable to consent, we will use a deferred consent process, as described below).
observational study investigating the effectiveness of fentanyl matrix in relieving pain and improving function in patients with osteoarthritis of the knee or hip

Conditions: Pain; Osteoarthritis, Hip; Osteoarthritis, Knee

Summary: the purpose of this study is to investigate the utility of fentanyl matrix for the treatment of pain due to osteoarthritis (oa) of knee or hip, which was not adequately controlled by non-opioid analgesic (e.g. nsaids or cox-ii inhibitors) or weak opioids \[tramadol hclacetaminophen, tramadol or codeine (or combinations with acetaminophen/ibuprofen)\].

this study is a prospective, open-label, non-interventional study. the objective of this study is to investigate the utility of fentanyl matrix after 8 week treatment of fentanyl matrix for the treatment of pain due to osteoarthritis (oa) of knee or hip, which was not adequately controlled by non-opioid analgesic (e.g. nsaids or cox-ii selective inhibitors) or weak opioids \[tramadol hcl with acetaminophen, tramadol or codeine (or combination with acetaminophen/ibuprofen)\] in clinical practice according to the investigator's discretion. this study will investigate the usefulness of fentanyl matrix based on improvement in pain and function after fentanyl matrix administration in patients who complained of pain caused by osteoarthritis and indicated for fentanyl matrix administration in clinical practice at the investigator's discretion. this study will also measure the patients' functional improvement through korean version of womac. observational study - no investigational drug administered

Eligibility criteria:
inclusion criteria: * patients who complain of chronic pain caused by osteoarthritis of the knee or hip persisting for 3 months or longer * patients who can fully observe the overall clinical study requirements including k-womac completion at the investigator's discretion * patients who complain of severe pain (nrs score = 7) because pain was not treated enough with the previous analgesic * patients who have never been administered strong opioid analgesics over the last one month exclusion criteria: * patients with a history of the drug or alcohol abuse in the past or now * childbearing women who are pregnant or likely to be pregnant during the study period and male subjects who are neither infertile nor willing to refrain from sexual relations but whose partner does not conduct an effective contraception (implant, injections, oral contraceptives, intrauterine device, etc.) * patients who are unable to use a transdermal system due to skin disease * patients with serious mental disorder * patients with history of hypersensitivity to opioid analgesics * patients with history of co2 retention * patients who are not eligible for the study participation based on warnings, precautions and contra medications in the package insert of the study drug at the investigator's discretion

Keywords: chronic pain fentanyl matrix arthritis osteoarthritis
transmasculine quality of life and sexual function at pre and post gender-affirming hormonal therapy

Conditions: Transgender Men

Summary: the goal of this prospective observational study is to evaluate the short-term effect of testosterone therapy on these issues among hormone naïve tm individuals. the main questions that aim to answer are : 1. comparison quality of life in transgender men at pre and post testosterone therapy 2. to evaluate the most affected domain of quality of life (qol) and fsfi of this participants participants will be ask to complete two questionnaires: the world health organization quality of life bref thai (who-qol-bref-thai) and the fsfi (female sexual function index) before testosterone administration and both questionnaires will be re-evaluated at 12 weeks after initiation of testosterone therapy

this study was designed as a single-center, prospective trial at king chulalongkorn memorial hospital, bangkok, thailand between september 2022 to february 2024. all hormone-naïve tm aged above 18 years who planned to start testosterone at gender health clinic, king chulalongkorn memorial hospital, thailand were approached. all the enrolled participants will be received information about the study and written consent was obtained from all participants before the start of the study. the demographic data were recorded. blood samples of serum estradiol, total testosterone and other laboratory investigations will be assessed including complete blood count, liver function test and lipid profiles. then, the whoqolbrefthai questionnaire and thai version of female sexual function index questionnaire (fsfi) will be assessed before testosterone administration. all tm participants will be received 250 mg of testosterone enanthate intramuscularly every 4 weeks for 12 weeks. after 12 weeks of treatment, another laboratory investigations and 2 questionnaires, (whoqolbrefthai and fsfi) will be assessed again.

Eligibility criteria:
inclusion criteria: * compatible with diagnostic and statistical manual of mental disorders, 5th edition (dsm-5) * had never used testosterone therapy before the enrollment exclusion criteria: * history of abnormal hormone functions * ongoing treatment or current use of medications that affect hormone functions * diagnosis of psychiatric illnesses beyond gender dysphoria by a psychiatrist * prescription of psychiatric or neurological medications within the past 3 months up to the present study participation * presence of chronic illnesses or severe age-related conditions, such as chronic kidney disease, chronic liver disease, or any type of cancer

Keywords: transgender men sexual function gender-affirming hormone therapy testosterone quality of life fsfi
analysis of data collected in the european group for blood and marrow transplantation (ebmt) registry for off-label transplant use of plerixafor

Conditions: Acute Leukemia; Chronic Leukemia; Myelodysplastic Syndrome; Myeloproliferative Neoplasm; Solid Tumors; Lymphoma; Plasma Cell Disorders

Summary: genzyme will evaluate/monitor the off label transplant use of plerixafor using data in the european group for blood and marrow transplantation (ebmt) registry. off-label use of plerixafor will be collected for data entered over a 5 year time span (i.e., data entered into the registry between the date of european union (eu) marketing authorization \[31 july 2009\] and 31 july 2014). the ebmt is a non-profit, scientific society representing more than 600 transplant centers mainly in europe. the ebmt promotes all activity aiming to improve stem cell transplantation or cellular therapy, which includes registering all the activity relating to stem cell transplants. data are entered, managed, and maintained in a central database with internet access; each ebmt center is represented in this database. the collection by the ebmt registry of reasons for the off-label transplant use of plerixafor shall provide information of a substantial number of patients who are representative of the patient population receiving plerixafor off-label.


Eligibility criteria:
inclusion criteria: patients treated with plerixafor who have 1 or more of the following: * background disease other than lymphoma or multiple myeloma (mm) * are younger than 18 years of age. * received transplant using ex vivo plerixafor-mobilised cells (umbilical cord cell, peripheral blood (pb), bone marrow (bm) cell collection) * received treatment with plerixafor alone (i.e., without granulocyte colony stimulating factor (g-csf)) * contraindication for g-csf * transplants using plerixafor-mobilised cells from allogeneic donor * received transplant using plerixafor-mobilised bone marrow cells * routes of plerixafor administration other than subcutaneous * patients whose cells do not mobilize poorly * other exclusion criteria: * adults diagnosed with lymphoma or multiple myeloma (mm) and have been treated with plerixafor according to the european union (eu) label.
photocil (topical) for the treatment of vitiligo

Conditions: Vitiligo

Summary: photocil is a topical drug (cream) that selectively delivers narrow band - ultraviolet b (nb-uvb) therapy when exposed to sunlight. photocil is intended to help protect users from non-therapeutic ultraviolet b (uvb) radiation while selectively passing wavelengths of light in the nb-uvb range with peak transmission of 308nm. the aim of the study is to assess the safety and efficacy of photocil in the treatment of vitiligo.

nb-uvb phototherapy is a common treatment for patients with psoriasis, and has been reported to be safe and effective in numerous clinical trials. clinical trials have reported achievement of vitiligo area severity index (vasi)-75 in 50-70% of patients after 4-6 weeks of nb-uvb treatment. many drawbacks limit patients compliance, access, and acceptance of traditional nb-uvb phototherapy. the strict treatment regimen (2-3 sessions per week for an average of 12 weeks or more) performed at a specialized phototherapy clinic combined with high cost and low or no reimbursement make compliance and access a major drawback. in order to address the drawbacks of phototherapy, we developed a novel topical cream - photocil - that selectively delivers nb-uvb therapy when exposed to sunlight. when used with natural sunlight, photocil provides a convenient alternative to traditional clinic based phototherapy; thus, has the potential to dramatically increase patient compliance and treatment outcome.

Eligibility criteria:
inclusion criteria: * diagnosed with vitiligo confirmed by a dermatologist * vitiligo lesions affecting at a minimum 5% of the facial, legs, or arms surface area * age: 18 to 65 * participants able to give informed consent exclusion criteria: * subject did not respond to prior phototherapy treatment * subject completed phototherapy for same lesion(s) in last 6 months * subject has previous history of skin cancer * subject has previous history of photosensitivity * subject has a history of herpes (hsv i or ii) outbreaks * subject has previous history of autoimmune disease may be excluded at investigator's discretion * subject is currently taking of immunosuppressive or photosensitizing drugs * subject plans to use antibiotics, anti-fungal, calcineurin inhibitors or other drugs that may cause photosensitivity during the study period. these patients may be excluded at investigator's discretion * subject is pregnant or lactating women
an exploratory trial investigating the neurogenic component in psoriasis vulgaris by use of botulinum toxin a

Conditions: Psoriasis Vulgaris

Summary: the purpose of this study is to evaluate the anti-psoriatic effect of injected botulinum toxin type a compared to that of injected vehicle.


Eligibility criteria:
inclusion criteria: * age 18 years or above * a diagnosis of psoriasis vulgaris with lesions located on arms and/or legs and/or trunk * chronic stable psoriasis vulgaris diagnosed more than 6 months prior to screening. * women must use a reliable contraceptive during the trial. exclusion criteria: * pregnant or breast feeding women, or women planning to become pregnant. * skin infection at injection sites * use of biological therapies or small molecules (marketed or not marketed) with a possible effect on psoriasis vulgaris * use of systemic treatments with a potential effect on psoriasis vulgaris * exposure to phototherapy within 4 weeks for puva and 2 weeks for uvb prior to randomisation * use of potent or very potent (who group iii-iv) corticosteroids for topical treatment of psoriasis within a 4 week period prior to randomisation * use of muscle relaxants * history of dysphagia or aspiration * current diagnosis of guttate, erythrodermic, exfoliative or pustular psoriasis
survey of patient and physician awareness and values to the diagnosis and treatment of reduced kidney function (chronic kidney disease) in patients with high blood sugar level (type 2 diabetes) (aware-ckd in t2d)

Conditions: Renal Insufficiency, Chronic

Summary: in this survey study, researchers want to find out more about how physicians are treating patients diagnosed with reduced kidney function (chronic kidney disease, ckd) and high blood sugar level (type 2 diabetes); what physicians know about the different treatment options available; and how they use this knowledge to treat their patients successfully. additionally, researchers want to gather information about patient's awareness of their disease and which criteria they consider important for their treatment of the disease. the study will survey a total of 150 patients with type 2 diabetes: 50 patients with mild ckd; 50 patients with moderate ckd; and 50 patients with severe ckd. patients eligible for this study can be male or female and should be at least 18 years old. in addition, a total of 150 physicians will be surveyed. physicians eligible for this study may be general practitioners, endocrinologist, or nephrologists.

the primary objective in this study is to assess the awareness, values and preferences of patients and physicians with respect to the risks, consequences and management of chronic kidney disease (ckd) in patients with type 2 diabetes. secondary objectives aim to assess awareness of available ckd treatments, referral tools and communication between patients and physicians. both patients and physicians will complete a survey that consists of various questions regarding the knowledge of disease, values and treatments, patient-physician interaction, physician referral tools and disease risk management.

Eligibility criteria:
inclusion criteria: * for patient cohort: * diagnosis of type 2 diabetes * egfr \< 60 ml/min per 1.73 m2 (measured within the last year) or egfr ≥ 60 ml/min per 1.73 m2 and uacr ≥ 2 (measured within the last year) * no documented non-diabetic etiology for renal disease * for physician cohort: * licensed general practitioner, endocrinologist or nephrologist * currently providing care for patients with chronic kidney disease and type 2 diabetes exclusion criteria: * patients with chronic kidney disease and type 2 diabetes who are physicians * physicians with chronic kidney disease and type 2 diabetes

Keywords: chronic kidney disease (ckd) type 2 diabetes (t2d) patient awareness physician awareness interaction patient physician