Unique difficulties and challenges can arise for rare diseases and orphan disease indications within a clinical trial. Some of the challenges encountered by trials that are working on rare diseases can include recruitment and enrollment. One of the explanations for the challenges that are encountered in rare disease trials is because of the trial design of study, where the investigational product is being compared to the placebo. This review paper discusses the usage of placebo and when other options could be taken into consideration while creating the protocol, specifically with consideration to rare disease studies. It is essential for the investigators and those designing the protocol to consider alternative options from the standard randomized controlled trial. The aim of this paper is to review alternative trial design options. The trial designs discussed in this review paper include crossover trials, single arm studies and historical data, and n-of-1 trials.

Keywords

Recruitment; Enrollment; Placebo controlled; Clinical trials; Rare disease; Crossover trials; Single arm study;
Historical data; n-of-1 trials.

Recruitment in clinical research trials can be challenging in trials that are time-sensitive and/or are rare disease and critical care trials. One of the hurdles for recruitment in these types of clinical trials is due to the consent process, and the need to have consent of the patient within a certain timeframe, or the patient unable to consent for themselves. This paper will discuss the usage of the utilization of remote consent options for these trials.

Keywords

Recruitment; e-consent; Remote consent; Clinical trials; Rare disease; Time-sensitive.

To study the role of bactiguard infection technology (BIP) in preventing Catheter-associated urinary tract infections. This study was conducted in a tertiary care centre in the Department of Plastic Surgery, JIPMER, Pondicherry, India in the month of August 2019. The study subject was a 55-year-old gentleman with Fournier’s gangrene and type 2 diabetes mellitus (DM). The Foley’s catheter with BIP technology was used and its role in preventing urinary tract infections (UTI) was studied over a period of 10-days. During the period of the catheter use in our subject, patient did not develop features of urinary tract infections. BIP technology is claimed to be effective in preventing Catheter-associated urinary tract infections but further studies in more individuals is required to validate the same.

Keywords

Bactiguard infection technology (BIP); Catheter associated urinary tract infection (CAUTI); Biofilm.

Aim

To study the role of sound enhancer device in teleconsultation.

Methods

This study was conducted in the plastic surgery department in a tertiary care centre in the month of May-June 2019. A sound enhancer device was used with mobile phone for videoconferencing as form of teleconsultation by plastic surgery trainees in operation theatre and outpatient department. At the end of the study feedbacks were taken from the trainees.

Results

It was found that on using the sound enhancer device, the sound quality improved and it was helpful in the teleconsultation sessions using videoconferencing.

Conclusion

The use of sound enhancer is a simple, cost-effective, innovative method of using a simple technology to aid in utilisation of teleconsultation facilities in a very non-sophisticated way.

Keywords

Sound enhancer device; Teleconsultation; Telemedicine.

Cancer immunotherapy has evolved enormously in the recent years with better understanding of immune reactions, immune microenvironment and immunosurveillance. Breast cancer is characterized by large heterogeneity, a fact which rather complicated the development and the approval of novel therapeutic options in comparison to the majority of other solid tumors since each subtype has required a unique scientific approach and different targets and goals. Triple negative breast cancer (TNBC) is considered the most aggressive of the breast cancer subtypes with limited treatment options and worse outcome compared to others. This article summarizes some of the early clinical studies and the recently presented phase III clinical study of immunotherapy checkpoint inhibitors in this difficult setting.

Keywords: Cancer; Immunotherapy; Triple negative breast cancer (TNBC); Breast cancer.

Objective: This study aimed to conduct an evaluation of music therapy clinical trials
worldwide, to understand what trials have been conducted and to show the chronological
changes. Additionally, we sought to clarify issues related to providing clinical trial registration
information.
Methods: We searched the International Clinical Trials Registry Platform (ICTRP) database for
“music therapy,” and identified the disease target for each article found.
Results: A total of 150 clinical trial studies were found in the ICTRP using the term “music
therapy.” In these trials, music therapy was used for improvement of social functioning in
schizophrenia and/or serious mental disorders, anxiety and depressive symptoms, and cancer
symptoms. Twenty-five clinical trials were actively recruiting. Sixteen of the 25 trials were
registered in the United States at ClinicalTrials.gov, of which 9 trials were conducted in the US.
Seven trials were conducted in other countries such as Spain, Taiwan, and China.
Conclusion: A search for music therapy clinical trials retrieved 150 trials from the ICTRP, and
the number of clinical trial registrations has increased yearly. Music therapy is widely used in
patients with various diseases, including Alzheimer’s disease, anxiety, and arthritic pain and
has the potential to improve certain disease outcomes, but there is not enough evidence to
substantiate its efficacy. It is important to enlighten researchers and pharmaceutical companies
on the proper management of the quality of such clinical trial information, as this is an important
issue.