7 Clinical Trials for Various Conditions
This study is evaluating the use of two painless, non-invasive technologies in the assessment of muscle health over time in both healthy volunteers and patients who have diseases that affect the nervous system.
Swallowing dysfunction and delay of oral feeding skills in premature infants significantly delay discharge from the hospital. Thickening oral formula or breast milk feeds with commercial thickeners and rice cereal has been used in some nurseries with the goal of promoting development of adequate feeding skills. The objective of this pilot study is to examine the effect of thickened feeds on premature infants with oral feeding difficulties due to dysfunction secondary to immature swallowing. Hypotheses: * Thickened feeds will lead to a reduction in symptoms secondary to immature swallowing in 32-40 weeks gestational age infants when compared to non-thickened feeds * Thickened feeds will promote transition to hospital discharge.
This was a multi-center, open-label, Phase Ib dose escalation /Phase II study in recurrent or metastatic head and neck squamous cell carcinoma (RM HNSCC) patients considered to be resistant, ineligible or intolerant to platinum-based chemotherapy. The Phase Ib included three arms. Three different methods of administration and two different BYL719 formulations were studied to determine the MTD and/or RP2D of BYL719 in combination with cetuximab: Arm A - film-coated whole tablets were orally administered to patients who were able to swallow the tablets; Arm B - a drinkable suspension prepared from crushed film-coated tablets was administered orally to patients with swallowing dysfunction Arm C - a suspension from a dispersible tablet administered via G-tube, in patients with swallowing dysfunction. Arm C was used to investigate the pharmacokinetics (PK), compared to Arm A (film coated tablet), and safety of the dispersible tablet of the dispersible tablet formulation of BYL719. The Phase II investigated the clinical efficacy of BYL719 and consisted of an open label, randomized Phase II part investigating BYL719 in combination with cetuximab compared to cetuximab alone in patients resistant or intolerant to platinum and naïve to cetuximab (Scheme 1: Arm 1 and Arm 2), and a non-randomized Phase II part Scheme 2: Arm 3. In addition, patients who experienced disease progression in Arm 2 (cetuximab) were allowed to switch to the combination regimen (cross-over, Arm 2B). The safety of the BYL719 in combination with cetuximab was also further characterized in Arms 1, 2B and 3. Patients were treated until progression of disease), unacceptable toxicity, or withdrawal of informed consent, whichever occurred first (except for phase II Arm 2 had the opportunity to crossover to the combination treatment (Arm 2B). In the follow-up period all patients had to complete the safety follow-up assessments within 30 days after the last dose of the study treatment. Patients who did not have disease progression at the time of discontinuation of study treatment were radiologically followed for disease status until disease progression, initiation of subsequent anticancer therapies, or death, whichever occurred first. In addition, all patients enrolled in Phase II were followed for survival.
This study is being done to assess the esophageal muscle (swallowing tube) in patients with and without achalasia to further understand and help direct the treatment for patients with achalasia.
This study will identify clinical signs and symptoms critical for diagnosing swallowing disorders and will characterize swallowing problems in various patient populations, such as patients with Parkinson's disease, stroke, post-polio syndrome, multiple sclerosis and other conditions that cause swallowing abnormalities. Patients with swallowing difficulties who are enrolled in NIH neurology or speech pathology protocols may be eligible for this study. Participants will undergo the following procedures: 1. Oral examination-A neurologist and speech pathologist examine the patient's swallowing function. The patient is interviewed about difficulties with food intake, chewing and swallowing during meals. 2. Ultrasound examination-Ultrasound creates image of areas inside the body using sound waves. With the patient in a sitting position, a 3/4-inch transducer (device for transmitting and receiving sound waves) is placed under the chin to visualize tongue movements during swallowing. 3. Modified barium swallow-While standing or sitting, the patient swallows 1/2 teaspoon of flavored barium (a radioactive substance) six times (a total of 3 teaspoons), while the tongue and pharynx (tube leading from the mouth to the esophagus) are scanned and videotaped. The barium is given in three consistencies-thin, medium and thick (pudding-like). 4. Electromyography-A small plastic strip with wires attached is placed under the patient's chin. The patient then swallows 1/2 ounce of barium three times in a row, and the movement of the chin muscles during swallowing is displayed. Patients may also be asked to swallow 5/8 cup of barium twice; once with the head tilted upward and once with the head untilted. Depending on the test results, patients may be asked to return for follow-up study and monitoring.
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here. This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords). In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control). In Part C, all participants will receive study drug.
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844. This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords). In Parts A/B, two out of every three patients will receive the study drug. The third patient will receive 1 to 2 small needle pricks at the location where the LP and IT injection is normally made (sham control). In Part C, all participants will receive study drug, as described in the Part C registration record. Start date for this record is the first day a participant was enrolled in Parts A/B. The trial is actually continuing until the last primary outcome measure of safety data are collected from Part C participants. The last primary outcome measure of safety, along with final adverse events results will be posted in the separate Part C registration record.