cyrilzakka/clinical-trials huggingface dataset:
Expanded Access Program of Palopegteriparatide in Patients With Hypoparathyroidism

To provide palopegteriparatide (TransCon PTH), an investigational parathyroid hormone (PTH) replacement therapy in an expanded access setting for adult patients with hypoparathyroidism who have previously received PTH-treatment, who meet the eligibility criteria for this protocol as described below and have a clear unmet medical need that cannot be adequately treated by a commercial product or a clinical trial.

This is an expanded access program for eligible participants who previously received PTH treatment and who are ineligible for an ongoing TransCon PTH clinical trial. The treating physician/investigator contacts Ascendis Pharma when, based on their medical opinion, a patient meets the criteria for inclusion in the expanded access program.

Participants will start with a fixed dose of study drug and will be individually and progressively titrated to an optimal dose based on serum calcium levels, followed by an individualized dosing period until palopegteriparatide is commercially available to the participant. For patients taking conventional therapy with calcium and or active forms of vitamin D, these therapies will be reduced in dose and/or discontinued during titration of palopegteriparatide.

Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of hypoparathyroidism.
* Patients with previous PTH-treatment experience.
* Serum albumin-adjusted calcium level ≥7.8 mg/dL and 25(OH) vitamin D in the normal range within 2 weeks before first dose.
* Body mass index (BMI) 17 - 40 kg/m2.
* Be willing and able to give written informed consent by signing an Institutional Review Board (IRB)-approved Informed Consent Form (ICF).

Exclusion Criteria:

* Diagnosis of pseudohypoparathyroidism.
* Currently enrolled in an investigational drug or device study or has used an investigational drug or device within 30 days or 5 half-lives (whichever is longer) of Day 1.
* Severe renal impairment (estimated glomerular filtration rate \<30 mL/min/1.73m2).
* Increased risk for osteosarcoma, such as those with Paget's disease of bone or unexplained elevations of alkaline phosphatase, hereditary disorders predisposing to osteosarcoma, or with a prior history of substantial external beam or implant radiation therapy involving the skeleton
* Active malignancy within past 2 years excluding successfully resected thyroid carcinoma and non-melanoma skin cancer.
* Severe or decompensated cardiac disease within 26 weeks, including but not limited to class IV or Stage D heart failure, unstable angina, myocardial infarction or uncontrolled arrhythmias.
* Pregnant or lactating females, or females intending to become pregnant.
* Known allergy and/or sensitivity to palopegteriparatide or its excipients or prior PTH therapy.
A Study to Evaluate Sampling Methods for Subgingival Plaque

To identify a sampling method for collecting subgingival plaque


Eligibility Criteria:
Inclusion Criteria:

* Provide written informed consent to participate in the study;
* Be 18 years of age or older;
* Agree not to participate in any other oral/dental product studies during the course of this study;
* Agree to delay any elective dentistry (including dental prophylaxis) until the study has been completed;
* Agree to use the acclimation toothpaste together with their regular brush and refrain from any other oral care products (including floss and mouth rinse) between the Screening and Baseline Visit;
* Agree to use the treatment products and refrain from any form of non-specified oral hygiene during the treatment periods (after the Baseline Visit), including but not limited to the use of products such as floss, mouth rinse, or whitening products;
* Agree to return for all scheduled visits and follow study procedures;
* Must have at least 16 natural teeth;
* Be in good general health, as determined by the Investigator/designee based on a review of the health history/update for participation in the study;
* Agree to refrain from all oral hygiene for at least 12 hours prior to each visit;
* Agree to refrain from eating, chewing gum, drinking and using tobacco for 4 hours prior to each visit;
* Have 10-20 bleeding sites; and
* Have minimum 6 sampling sites with bleeding and pocket depth ≥2mm but not deeper than 4mm.

Exclusion Criteria:

* Have had a dental prophylaxis within 2 weeks of plaque sampling visits;
* Have taken antibiotics or used anti-gingivitis / anti-bacterial oral care products such as chlorhexidine or Listerine within 2 weeks of plaque sampling visits;
* Have rampant caries, open or untreated caries, or advanced periodontitis requiring prompt treatment;
* Taking allergy medication or any other medication that can affect salivary flow such as an antihistamine;
* Need an antibiotic prophylaxis prior to dental visits;
* A history of hypersensitivity to oral care products containing hydrogen peroxide or stannous fluoride;
* A history of hypersensitivity to products containing sodium lauryl sulfate (SLS);
* Are pregnant (Self-reported) or lactating; or
* Have any condition or disease, as determined by the Investigator/Designee based on a review of the medical history, which could be expected to interfere with examination procedures or with the subject's safe completion of the study.
DHEA Replacement in Adrenalectomized/Hypoadrenal Patients

The role of dehydroepiandosterone (DHEA) and its sulphated ester (DHEAS) \[together known as DHEA(S)\] in humans remains to be fully elucidated. They are the most abundant circulating steroid hormones in humans with the principle source of production being the adrenal glands. In adrenalectomised or hypoadrenal subjects it is not standard clinical practice to replace this hormone. Whilst this lack of DHEA(S) is clearly not life threatening, the published literature on the psychological effects of replacement in this population has shown some benefit in quality of life but data concerning the metabolic effects is conflicting and incomplete. We wish to carry out a study to measure the effect of DHEA(S) replacement in a group of adrenalectomised / hypoadrenal subjects and measure the outcome of a number of psychological and metabolic parameters both prior to and after twelve weeks of 50 mg/day DHEA(S) in a double blind randomised cross over design.

Mood, quality of life and sexual well being will be assessed using validated questionnaires. In addition, we will utilise the GCRC facility to measure body composition using total body water and DEXA. We will measure insulin sensitivity, exercise performance, muscle fatigability and VO2 max. Pre- and post- intervention muscle biopsies will assess DHEA(S) effects on myosin heavy chain isoform expression; skeletal muscle mitochondrial ATP production and be used to assess muscle enzyme levels.


Eligibility Criteria:
Inclusion criteria:

* Subjects who have been adrenalectomised or have been hypoadrenal (from whatever cause) for \> 24 months;
* Subjects who have been on a steady glucocorticoid replacement regime for \>12 months;
* Women of child bearing age in whom oestrogen status has been steady for \> 6 months - i.e. either on or off the oral contraceptive pill for that time;
* Subjects on other forms of hormone replacement therapy (e.g. thyroxin) in whom dose has remained the same for \> 6 months.

Exclusion criteria:

* BMI \>35 Kg/m2;
* Individuals with fasting blood glucose above 120mg/dl;
* A history of sex hormone dependant malignancy;
* A history of liver disease;
* Renal failure;
* Cardiovascular disease (other than hypertension);
* Polycythaemia;
* Pregnancy or breastfeeding;
* Cerebrovascular disorders;
* Neurological disorders;
* PSA levels above the age related reference range;
* Use of drugs known to alter mood within the 6 months prior to enrolment;
* Any drug known to affect hepatic biotransformation;
* Evidence of psychiatric disease or dementia;
* Postmenopausal women who have been on hormone replacement therapy for less than six months.
Hysteroscopic Intrauterine Suturing

Clinical trial The aim of the study is to evaluate the safety and feasibility of intrauterine suturing with Apollos X-tack, inserted into the uterus by hysteroscope.

Primary outcome:

Intrauterine suture will sustain the procedure - when the uterus will be opened the suture will be observed.

Secondary outcome:

Procedure complications. Study Design Interventional pilot Study

Study group:

Women undergoing laparoscopic hysterectomy.

Introduction:

A hysteroscopy is a procedure used to examine and treat the inside of the uterus.

Apollo X-tack is an endoscopic apparatus for stomach defect closure by gastroscopy.

The aim of this study is to evaluate the safety and feasibility of intrauterine suturing with Apollos X-tack inserted into the uterus by hysteroscope.

Study Design Interventional pilot study

Study group:

Women undergoing laparoscopic hysterectomy.

Inclusion criteria:

Benign indication for hysterectomy

Exclusion from the study:

Evidence or suspicious for malignancy

Sample size calculation:

Pilot study of 2 patients.

Primary outcome:

Intrauterine suture will sustain the procedure - when the uterus will be opened the suture will be observed.

Secondary outcome:

Procedure complications.

Recruitment:

The recruitment of the patients will be done at the gynecology department before the surgery.

Procedure:

After general anesthesia and entering the abdomen with the laparoscope, Truclear hysteroscope with X-tack attached will be inserted to the uterine cavity. Insertion of normal saline to the cavity and applying 4 points suturing to the uterus. The procedure will be monitored by external laparoscopic camera located in the abdominal cavity. After the hysterectomy the uterus will be opened and the suture will be examine.

A preoperative intrauterine incision using a hysteroscope, followed by suturing, will be considered based on the success of the first procedure.

CRF:

Demographics information, obstetrics and gynecology information, surgery type, surgery indication.

Procedure duration, success, amount of fluid used, cervical dilatation, complications.

Eligibility Criteria:
Inclusion Criteria:

* Benign indication for hysterectomy

Exclusion Criteria:

* Evidence or suspicious for malignancy
Prismaflex HF20 Set and Prismaflex® System 7.10/7.20 for Acute Continuous Renal Replacement Therapy (CRRT) in Children

The primary objective of this study is to evaluate the efficacy of the Gambro Prismaflex® HF20 Set based on testing the hypothesis that it delivers sufficient renal replacement therapy to effectively treat acute kidney injury (AKI) in pediatric patients by reducing blood urea nitrogen (BUN).


Eligibility Criteria:
Inclusion Criteria:

1. Patients with a hospital admission body weight ≥8 and \<20 kg (ie, ≥17.6 and \<44.09 lbs).
2. Patients with AKI defined as either 1) AKI by the Kidney Disease Improving Global Outcomes (KDIGO) Acute Kidney Injury Guideline serum creatinine criteria, which is a \>50% rise in serum creatinine over baseline or a 0.3 mg/dL SCr rise in 48 hours OR 2) as AKI by the Pediatric modified Risk, Injury, Failure, Loss, End-Stage Renal Disease (pRIFLE) criteria which is a 25% reduction in estimated creatinine clearance,16 OR 3) a serum creatinine \>1.2 mg/dL. OR Patients with severe fluid overload, defined as a \>10% fluid accumulation relative to the ICU admission.
3. Patients who have received RRT previously can be included in the study if \>24 hours have elapsed since their previous RRT treatment.
4. Provide written informed consent from one or both parents, as required by the local IRB or legal guardians, unless one parent is deceased, unknown, incompetent or not reasonably available, or when only one parent has legal responsibility for the care and custody of the child per 21 CFR Part 50.55(e).

Exclusion Criteria:

1. Patients a) weighing 8.0-20.0 kg with a hemoglobin of \<7.0 g/dL, b) weighing 8.0-12.0 kg with a hemoglobin of \<8.0 g/dL (unless blood prime used), and c) weighing 12.1-20.0 kg with a hemoglobin of \<7.5 g/dL (unless blood prime used).
2. Children who are wards of the state.
National Register of Oesophageal Atresia

The esophageal atresia is a group of birth defects including a break in continuity of the esophagus with or without persistent communication with the trachea (tracheoesophageal fistula), sometimes associated (from 50%) of other malformations (heart, kidney, digestive ...).

The current prognosis for this ailment is good. However he persists a mortality (\<10%) and significant morbidity, firstly related malformations (heart, kidney, for example), and secondly with particularly difficult anatomical forms (Forms long defect) .

The prevalence of this condition is estimated to be 1/2500 in 3000 live births, making an estimated ± 2,500 new cases over to 10 years in France.

The current project aims to set up a national registry (Metropolitan France and Dom Tom) to measure the prevalence of esophageal atresia among live births, phenotypic characteristics, the circumstances of their diagnosis, and their initial future at short-term during the first year of life, at which time occurs the vast majority of deaths and complications in this disease.


Eligibility Criteria:
Inclusion Criteria:

* To be born in France
* To have a esophageal atresia

Exclusion Criteria:

* To be born abroad
Safety of Cultured Allogeneic Adult Umbilical Cord Stem Cells for Peyronie's Disease, ED, and Interstitial Cystitis

This trial will study the safety and efficacy of intravenous and intracavernosal or interstitial delivery of cultured allogeneic adult umbilical cord derived mesenchymal stem cells for the treatment of Peyronie's disease, erectile dysfunction , and Interstitial Cystitis

This patient funded trial aims to study the safety and efficacy of intravenous and intracavernosal or interstitial delivery of cultured allogeneic adult umbilical cord derived mesenchymal stem cells (UC-MSCs) for the treatment of Peyronie's disease, erectile dysfunction , and Interstitial Cystitis. The total dose will be 100 million cells. Patients will be evaluated within one month pre treatment and at 1, 6, 12, 24, 36, and 48 months post treatment for safety and efficacy.

Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of Peyronie's Disease, erectile dysfunction, or Interstitial Cystitis
* Understanding and willingness to sign a written informed consent document

Exclusion Criteria:

* Active infection
* Active cancer
* Chronic multisystem organ failure
* Pregnancy
* Clinically significant Abnormalities on pre-treatment laboratory evaluation
* Medical condition that would (based on the opinion of the investigator) compromise patient's safety.
* Continued drug abuse
* Pre-menopausal women not using contraception
* Previous organ transplant
* Hypersensitivity to sulfur
Investigation of the Relationship Between Pulmonary Function and Muscle Strength in Athletes With Disability

To investigate the relationship between respiratory function and core muscles strength in athletes with disabilities and compare with healthy athletes.

The determination of parameters affecting the performance of high performance athletes that are effective in performance is very important to determine the physical performance potential. The body is a kinetic chain and core muscles play an important role in this chain. The relationship between the performance ability of the athletes and the relationship between the core muscles and performance abilities and respiratory function is extremely important. Different functional classifications caused by different barriers (physical, mental or visual) in many paralympic branches affect physical training because it is directly related to the classification, performance and training adaptations of an athlete. Determining the parameters that affect the respiratory function of paralympics is very important to develop a training program with strategies that can affect the physical performance of athletes and thus to achieve the desired results in training and competitions. Therefore, the aim of this study was to investigate the relationship between respiratory muscle strength and core muscle strength in paralympic athletes (athletes with disability).

Eligibility Criteria:
Inclusion Criteria:

* Being professional athletes for at least three years.

Exclusion Criteria:

* Having any lumbar injuries in the last three months,
* Having systemic problems other than their disabilities(for athletes with disabilities),
* Having any disability related to vision and hearing,
* Acute or chronic respiratory infection,
* Spinal cord injury level up to the innervation of the abdominal muscles (for paraplegic athletes).
Effect of Aerobic Exercises With Selected Phytoestrogens on Hot Flushes in Menopausal Women

50 menopausal women with the diagnosis of hot flushes were randomized to 1 of 2 groups

Group (A):

It consists of 25 women who practiced treadmill exercise program for 30 minutes at 60% to 70% of maximum heart rate. The treatment sessions will be repeated 3 times per week for 12 weeks.

Group (B):

It consists of 25 women who received diet modification contains soy products (phytoestrogen) such as soy milk and soy beans every day for 12 weeks only.

50 menopausal women with the diagnosis of hot flushes were randomized to 1 of 2 groups

Group (A):

It consists of 25 women who practiced treadmill exercise program for 30 minutes at 60% to 70% of maximum heart rate. The treatment sessions will be repeated 3 times per week for 12 weeks.

Group (B):

It consists of 25 women who received diet modification contains soy products (phytoestrogen) such as soy milk and soy beans every day for 12 weeks only.

Eligibility Criteria:
Inclusion Criteria:

* menopausal women minimum 2 years after menopause Patients were required to have at least moderate hot flashes at time of randomization, having a score ≥ 40 in Hot Flash-Related Daily Interference Scale (HFRDIS)

Exclusion Criteria:

* women with BMI \> 30kg/m2 ingestion of any psychotropic and antidepressant medications, any Selective Estrogen Receptor Modulator medications (e.g., tamoxifen and raloxifen), any Aaromatase inhibitor medications (e.g., anastrozole, letrozole, and exemestane), leuprolide acetate, clonidine, gabapentin, pregabalin, amino acid supplements, over the counter (OTC) medications that reduced hot flashes during the last 4 weeks, ingestion of estrogen and progesterone based medications, history of suicidal thoughts, substance or alcohol dependence (with the exception of nicotine dependence) during the last 3 months and Electroconvulsive therapy (ECT) during the last 2 months.
177Lu-JH04 in Patients with FAP-Positive Tumors

This is a pilot study to assess the dosimetry, toxicity and response of 177Lu-JH04, a new FAP-targeted radiopharmaceutical, in patients with FAP-Positive Tumors. All patients underwent 68Ga-FAPI PET/CT for selection and were successively divided into three groups of 3 people each.The three groups received successively an approximately 3.70 GBq (100 mCi), 5.55 GBq (150 mCi) and 7.40 GBq (200 mCi) of 177Lu-JH04 up to 4 cycles.

Cancer-associated fibroblasts (CAFs) are an integral part of the tumor microenvironment and present abundantly in the stroma of multiple tumor entities. Fibroblast activation protein (FAP), as a specific marker protein of CAFs, is overexpressed on more than 90% of malignant tumors\&amp; limited and expression in normal tissues, making it an appropriate target for diagnosis and therapy of numerous malignant tumors. Currently, several radiopharmaceuticals targeting FAP for radionuclide therapy have been developed, such as 177Lu-FAPI-46, 177Lu-FAP-2286, and showed high efficacy and reasonable toxicity profile in tumor treatment. 177Lu-JH04, a novel radiopharmaceutical targeting FAP, demostrated high stability in vitro and in vivo, and can accumulate specifically in tumors with high binding affinity, safety, and selectivity in preclinical studies. In this study, we assess the dosimetry, toxicity and response of 177Lu-JH04 for the treatment of patients with advanced metastatic cancers after exhaustion of all other treatment options.

Eligibility Criteria:
Inclusion Criteria:

* progressive advanced metastatic tumors
* tumors with high FAP expression confirmed on 68Ga-FAPI PET/CT
* adequate renal, haematological, and liver function
* an Eastern Cooperative Oncology Group performance status of 0-2

Exclusion Criteria:

* pregnant or lactating women
* received other radionuclide therapy in the past 6 months
* received chemotherapy, radiotherapy and other anti-tumor treatments in the past 28 days
Genetic Studies in Patients and Families With Infantile Spasms

Infantile spasms (IIS), a characteristic epilepsy syndrome of infancy with often catastrophic developmental consequences, is known in some patients to have many different genetic, metabolic and structural etiologies. However, for most patients IIS is the only presenting clinical feature and the specific cause is unknown. Only two FDA approved pharmacologic treatments for IIS exist, Adrenocorticotropic hormone (ACTH) and vigabatrin. While vigabatrin may be the treatment of choice for Tuberous Sclerosis as a cause for IS, ACTH is the treatment of choice for all others. Unfortunately, a substantial number of patients may still not respond to ACTH and there is no a priori way that suggests which patients may be responders. This has led to the following key questions:

Can novel genetic analyses determine known genetic causes of IS with greater efficiency (more timely and cost-effective)? Can novel genetic analyses determine previously unknown disease modifying genes that predispose individuals to develop IS? Can novel genetic analyses elaborate genes and gene polymorphisms that favor ACTH responsiveness? Do these polymorphisms suggest strategies to improve ACTH responsiveness?

Primary Aim 1: Apply whole-exome sequencing to determine possible causes of cryptogenic IS and evaluate adding whole-exome sequencing to standard practice for determining causes of IS. Sub-aim 1: Determine the effectiveness of whole-exome sequencing in suggesting disease-modifying genes that may contribute to triggering IS.

Primary Aim 2: Determine genes, through whole-exome sequencing, that may play a role in determining ACTH responsiveness for IS. Sub-aim 2: Correlate genes or genetic factors (haplotypes) associated with ACTH responsiveness and disease modification.

Eligibility Criteria:
Inclusion Criteria:

* Patient trios (both biological parents + patient with IIS = trio) with IIS retrospectively identified to have been treated with ACTH according to FDA-approved protocol (Table 1).
* Ability to provide informed consent (in case of severe to profound intellectual disability, consent provided by an legally authorized representative, as necessary)

Exclusion Criteria:

* IIS due to suspected or genetically proven tuberous sclerosis
* IIS but do not meet retrospective enrollment criteria (Table 1)
* Inability to complete consent process