Diseases and Abnormalities at or Before Birth

All Conditions

Heart and Blood Diseases

Neoplasms

Blood and Lymph Conditions

Rare Diseases

Congenital Abnormalities

Vascular Malformations

Lymphangioma

Lymphatic Abnormalities

Lymphatic Diseases

Neoplasms by Histologic Type

Lymphatic Malformations

Microcystic Lymphatic Malformation

Anti-Infective Agents

Antineoplastic Agents

All Drugs and Chemicals

Sirolimus

Temsirolimus

MTOR Inhibitors

Anti-Bacterial Agents

Antibiotics, Antitubercular

Clotrimazole

Miconazole

Antifungal Agents

Immunosuppressive Agents

Immunologic Factors

QTORIN 3.9% Rapamycin Anhydrous Gel Topical

QTORIN 3.9% Rapamycin Anhydrous Gel

Placebo

Kathy Goin

Emily Cook

Study Coordiantor

Study Coordinator

Steve Kempers, MD, FAAD

Milton Waner, MD, FCS (SA)

Joyce Teng

The main purpose of this study is to assess the change in clinician global impression after 24 weeks of treatment with QTORIN 3.9% Rapamycin Anhydrous Gel compared to placebo in approximately 50 participants with microcystic lymphatic malformations.

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations

A Multicenter, Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations

Palvella Therapeutics, Inc.

Nutritional and Metabolic Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Digestive System Diseases

Behaviors and Mental Disorders

Heart Diseases

Heart Defects, Congenital

Nutrition Disorders

Premature Birth

Hirschsprung Disease

Child Nutrition Disorders

Cardiovascular Abnormalities

Obstetric Labor, Premature

Obstetric Labor Complications

Pregnancy Complications

Urogenital Diseases

Female Urogenital Diseases

Female Urogenital Diseases and Pregnancy Complications

Digestive System Abnormalities

Gastrointestinal Diseases

Megacolon

Colonic Diseases

Intestinal Diseases

Quality of Life

Pharmaceutical Solutions

The overall incidence of congenital malformations in the gastrointestinal and urinary tract needing surgical interventions is about 1:1000 (Swedish national malformation registers) with a morbidity during childhood about 20-60%. Advanced paediatric surgery for the diagnosis Hirschsprung's disease, anorectal malformations, bladder extrophy, congenital diaphragmal hernia, and esophageal atresia is from July 2018 only performed at two NCSM in Sweden. The NCSM at Skåne University Hospital (SUS) in Lund forms one context. The quality of postoperative care is of immense importance both for short and long-term outcome. Legal guardians describe their situation after leaving the hospital as extremely stressful as they have not only to take responsibility for their new-born child but also of surgical wounds, medications, treatments, and special nutritional needs.

In Sweden, about 8-10 in 1000 children per year are born with congenital heart disease (CHD). CHD is a birth defect that leads to frequent hospitalisation, long hospital stays, and extreme anxiety for parents (18). In Sweden, paediatric heart surgery is concentrated to two NCSM of which one is situated at SUS, Lund where 250-300 children have cardiac surgery every year. Children with complicated CHD require contact and follow-up visits for a long time after the heart surgery and many families have to travel long for surgery (for example from Iceland), postoperative care and follow-up visits. Telemedicine after reconstructive cardiac surgery in children is shown to be feasible, although challenging and reduced unscheduled visits.

Most prematurely born children grow up to be healthy, but as a group, they are at a greater risk of developing cognitive, emotional and behavioural problems. Every year 7% of all children are born prematurely (gestational age of less than 37 weeks) and the numbers of preterm births are rising in Sweden as well as internationally. Preterm births often involve long hospitalisations for children and parents, and discharge from the hospital often means a difficult transition for parents in both short and long-term perspectives. Traditionally, communication with parents following discharge has been through home visits or telephone calls. By communicating through digital technology, it may be possible to improve the support to parents and thereby make the transition from hospital to home less stressful.

For children with cancer, treatment and follow-up at home is common. At the same time, families wish to minimize the negative impact on family members' social and everyday life. Home medication management is a high-risk area and medication errors are common, particularly among parents of children with cancer. Thus, parents are responsible for complex care and regular follow-up in their home with an increased need for education as well as clinical management support. At present, there are no, or limited, professional outreach support to support them and their families. Communication with parents following discharge has been through e-mail and/ or telephone calls. By communicating through digital technology, it may be possible to improve the support to children and parents.

For children with LTI administration of intravenous infusion therapy at home is an increasingly important area. Home medication management is a high-risk area and medication errors are common, particularly among parents of children with cancer. Thus, parents are responsible for complex care in their home with an increased need for educational as well as clinical management support during home infusion therapy. At present, children and adolescents with LTI at the University Hospital of Copenhagen receive home infusion therapy by a portable pump with no assistance from an outreaching team to support them and their families.

Cerebral palsy (CP) is the most common physical disability in childhood. Approximately 2-2.5/1000 children have CP with affected muscle tone, movement and motor skills, often accompanied by pain, epilepsy and intellectual, communicational and behavioural impairment. Early detection is challenging but important for minimizing the consequences from neurodevelopmental impairment by an early and right treatment. General Movement Assessment (GMA), an observational method for classification of spontaneous movements in young infants, is currently the most accurate method for early identification of CP. Video recordings are taken with a standardised video set-up in the hospitals regular follow-up clinics when the child is 10 to 20 weeks post-term age. Performing video recordings at home by the parents at a time, which suits the family and the child, would optimize the chances for a successful recording.

In this project we focus on eHealth solutions for patients and/or their families in situations where they could benefit from enhanced communication options with specialist staff. In this design space we use a model where a 4G-enabled tablet computer is lent to the families for the length of the study, thus avoiding economic requirements on the families and keeping the equipment uniform. The tablets run an application that enables multiple forms of direct and bi-directional communication in real-time with specialist staff at the hospital. For the staff, the technical situation is different; here we have in earlier demonstrators used web-based solutions accessed with existing computer equipment.

e-health device with application

Inger Kristensson Hallström, PhD

The overall aim is twofold: 1) to stretch the borderline regarding the present knowledge of clinical and economic cost-effectiveness of eHealth as an aid for facilitating and supporting self-management in families with long-term childhood illness, and 2) to develop a sustainable multidisciplinary research environment for advancing, evaluating, and implementing models of eHealth to promote self-management for children and their families.

A number of clinical studies are planned for, covering different parts of paediatric healthcare. The concept of child-centred care is essential. Experienced researchers from care science, medicine, economics, technology, and social science will collaborate around common issues. Expertise on IT technology will analyse the preconditions for using IT; economic evaluations will be performed alongside clinical studies; and cultural and implementation perspectives will be used to analyse the challenges that arise from the changes in relations among children, family and professionals, which may occur as a result of the introduction of eHealth.

Child health is not only important in itself. Investments in child health may also generate significant future gains, such as improved educational and labour market performance. Six complex, long-term and costly challenges in paediatric healthcare are planned for, involving eHealth technology such as interactive video consultation, pictures, on-line monitoring, and textual communication. The research follows an international framework for developing and evaluating complex interventions in healthcare. End-users (families) and relevant care providers (professionals in health and social care) will participate throughout the research process. The overall aim is certainly to analyse eHealth as an aid for facilitating and supporting self-management. However, the plan also includes the research issue whether eHealth at the same time improves the allocation of scarce health care- and societal resources.

The vision for the coming 6-year period is to establish multi-disciplinary research, designed to strengthen and advance the knowledgebase of eHealth as an aid for facilitating and supporting self-management among children with long-term illness and their families. The programme builds on interventions and implementation in three research domains: (1) eHealth to enable and promote self-management in advanced paediatric care, (2) eHealth for early diagnosis and treatment in paediatric healthcare, and (3) Co-creation of multi-disciplinary knowledge for the translation of eHealth in practice.

The overall goals of the research programme are to:

Evaluate, advance and implement models of eHealth to improve self-management for children and adolescents with or at risk for long-term illness and their families for better allocation of family-, healthcare- and societal resources.

Increase the knowledge of cultural factors and implementation strategies to facilitate implementation processes in general, and more specifically generate theoretical and methodological models on how to successfully implement eHealth in paediatric care.

Enhance the translation and integration of new research findings within eHealth into care services and the wider society by supporting healthcare professionals in their provision of evidence-based care and by translating and communicating research results to them as well as stakeholders and families.

Enhance the research proficiency of junior researchers in paediatric care and eHealth nationally and internationally through collaborative approaches in both inter- and multidisciplinary groups.

Two features of healthcare systems today are easily detected: 1) a trend towards more reliance on the individual and family to take active part in the healthcare process - with self-management being a keyword, and 2) great expectations regarding the potential benefits of using modern information technology in the domain of health and health care. The complex and costly needs for care of an increasing number of children with long-term illness (LTI) i.e. an illness that lasts for three months or more, generally has a slow progression, and can be controlled but not cured \[1\]) is one of the most critical challenge for healthcare systems all over the world. In addition to poorer health, people with LTI generally have lower educational achievements, fewer economic opportunities, and higher rates of poverty. Ensuring healthy growth and development in children is a principal concern of all societies. In this context, eHealth is of special interest as an aid for facilitating and supporting self-management for children with and at risk for LTI.

Self-management is the use of own time and money for individual and family health promotion. It is an ingredient in almost all measures for promoting health. Pure self-management in health relies on own efforts without formal intervention of health care services. On the other extreme, there are formal healthcare activities that require 100 percent of healthcare inputs for example surgery under anaesthesia. In most instances, both types of inputs are necessary - in varying degrees. The choice of self-management activities and its results depend on family-member preferences, knowledge, and attitudes. In order for healthcare system initiatives to be accepted and successful, they must be attuned to these characteristics. Individual tailoring is all the more important, the more reliance is on self-management. Thus, in the process of developing and evaluating new treatment modes, user participation is essential.

eHealth is a broad concept as indicated by the WHO definition: "eHealth is the transfer of health resources and healthcare by electronic means", i.e. also including older techniques such as telemedicine. Great expectations are associated with the realisation of the term. It may: 1) increase communication between healthcare providers and patients, 2) increase accessibility and patient participation in healthcare, and 3) support information and data sharing between patients, family and health-service providers http://www.euro.who.int/en/ehealth. It might be observed that the term eHealth does not preclude the use of electronic means to improve communication between providers within the healthcare system, potentially increasing the internal efficiency of formal healthcare, without any direct effects on patients and families. Here, however, the focus is on the role of eHealth as an aid for facilitating and supporting self-management, i.e. a concern with a wider, societal perspective on the allocation of scarce resources for health.

Cost-effectiveness is one of the ethical principles that are supposed to govern decisions on the allocation of scarce healthcare resources in Sweden. Thus, new healthcare technology should not be introduced if not proven cost-effective in comparison to existing (best) practice. Requirements for the economic evaluation of new healthcare technology exist in most Western countries. To date, economic evaluations of eHealth reported in the literature are scare and suffer from lack of randomised control trials (RTCs), small sample sizes, and the absence of high-quality data or appropriate measures.

Paediatric healthcare is rapidly evolving towards outpatient and community-care and from public to parental responsibility. This form of care is however rarely backed-up with communication support. In Sweden and most Western countries, highly specialised paediatric healthcare is confined to few National Centres of Specialized Medicine (NCSM) http://www.socialstyrelsen.se/rikssjukvard. This means that following birth a large majority of children with congenital malformations are transported to hospitals far away from home for advanced surgery. Following discharge, the outermost medical and nursing competence for the child will be at the NCSM. International policy and practice aim for infants, older children and adolescents with LTI to be cared for at home as much as possible to reduce the distress of hospital admissions and to keep up the normal family life. Limited research exists on the effect of digital interactive care on parents and children. eHealth in the home setting is often preferred by parents, especially by parents of newborns but has still not been widely adopted. Digital techniques can aid safer post-operative healthcare at home and may reduce costs to patients and society. This in turn might lead to more confident use of self-management and an increased sense of security at home. In some areas in paediatric care telemedicine initiatives is shown to benefit resources, enrich the community, and improve care that ultimately serve to better patient health.

Six clinical research projects are planned, illustrating complex, highly specialized, often long-term and costly decision-making challenges in paediatric care, for developing, evaluating implementing, and utilising different devices of eHealth technology. In addition, four more research projects will be performed in parallel to provide general analyses of eHealth in paediatric care and to facilitate and support economic, technical, cultural, and organisational analyses of the clinical data.

Bridging the hospital care and safe care at home for children with LTI with the aid of eHealth is a vital clinical area, especially for professionals in nursing, physiotherapy and medicine. However, also for the scientific area in health science it is of great importance as eHealth change the communication between children, parents and health professionals. This is a new research area that needs to be explored.

To develop, evaluate and implement eHealth devices in clinical care, an inter- and multidisciplinary approach is needed. Therefore, the investigators well-functioning and long-lasting clinical and research collaboration in health science, medicine, global health, health economics, technology, and social science has been enhanced to respond to the needs in paediatric care. Thereby, it is possible to address the important study of encounters with technology and various methods of communications and how eHealth aid can capture complex exchange of wellbeing and health, and feelings and perceptions of for example anxiety, stress levels, safety, many of which are culturally related. Also, values and contexts are sensitive to communication methods. The studies will therefore contribute significantly to the research area of eHealth as an aid for facilitating and supporting self-management in paediatric care, but not only. The programme also includes the research issue whether eHealth at the same time improves the allocation of scarce healthcare- and societal resources.

The multi-method design including both qualitative and quantitative methods will enhance the understanding to advance and evaluate routines and practices for better physiological and psychological outcomes in children with LTI and their families. The clinical projects will also contribute to a deeper understanding on identifying risk factors for complications and how to prevent adverse events in care performed at home.

In this programme, the focus is on eHealth solutions for situations where children and their families can be supported by enhanced communication for self-management with specialist professionals most commonly with specialist nurses. In such situations, it is important to find technical solutions that are both easy to use for all involved, easily accessible for families, and efficient to use for the professionals. Design options include web browser-based solutions, application-based solutions for larger mobile devices such as touchpads and application for mobile phones while observing the strict security rules for personal health information. An issue of accessibility is also to avoid economic demands on e.g. families not having access to appropriate equipment. Furthermore, relying on patient's equipment increases the technical demand and testing as the variation of equipment is huge, while the number of patients is comparatively small. Typically, technologies and applications develop at a high pace driven by commercial interests and may not be synchronised with a corresponding development of the understanding of possibilities and limitations of the applications in specific areas of care.

Importantly, the studies within different clinical contexts and considering cost-effectiveness, technical development, cultural and implementation aspects contribute to the knowledge and understanding about how eHealth can reduce the information asymmetry between providers, developers and consumers for further implementation into clinical practices, policies, and procedures. This programme also provides a significant contribution to a grounded and evidence-based understanding of both possibilities and limitations of eHealth applications in this area not only in terms of their efficacy but also in terms of the possible ethical conflicts involved in the introduction of new technology.

Consecutive series of legal guardians to children fulfilling the inclusion criteria in each arm. The legal guardians will be asked to 1) be involved in the development of the eHealth application, 2) evaluate the eHealth application at home, or 3) serve as a control group receiving traditional follow-up after discharge.

eHealth as an Aid for Facilitating and Supporting Self-management in Families With Long-term Childhood Illness

eHealth as an Aid for Facilitating and Supporting Self-management in Families With Long-term Childhood Illness - Development, Evaluation and Implementation in Clinical Practice

The PedsQL Healthcare Satisfaction Generic Module is composed of 24 items comprising 6 dimensions. Item scaling: 5-point Likert scale: 0 (Never) to 4 (Always) and Not Applicable. Higher scores indicate higher satisfaction. The scale includes the variables: information, family inclusion, communication, technical skills, emotional needs, and overall satisfaction.

This questionnaire contains information about the health of each child both at the time when the study begins and when the study ends. It asks for information on the weight of the child, the diagnosis, the treatment, specific needs when released from hospital, general health status when released from hospital, general health status at end of study. The answers are either given as multiple choice or as free text.

The PedsQL 2.0 Family Impact Module is composed of 36 items comprising 8 dimensions. Item scaling: 5-point Likert scale: 5-point Likert scale from 0 (Never) to 4 (Almost always). Higher scores indicate better functioning.

The Parental Stress (Parental - Persistent Role Problems) is composed of 9 items. Item scaling: 5-point Likert scale: 5-point Likert scale from 0 (Does not happen) to 4 (Happens always). Lower scores indicate less stress.

Any adverse events occurring during the study period will be recorded in free text format by the staff for all participants.

The therapy used to correct a specific complication is the basis of this classification in order to rank a complication in an objective and reproducible manner. The scale consists of 7 grades (I, II, IIIa, IIIb, IVa, IVb and V). A high grade indicates a more severe problem.

Healthcare expenditures by type of resource

The EQ-5D-3L descriptive system comprises the following five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: no problems, some problems, and extreme problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions. This decision results into a 1-digit number that expresses the level selected for that dimension. The digits for the five dimensions can be combined into a 5-digit number that describes the patient's health state.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations	NCT06239480	Microcystic Lym...	QTORIN 3.9% Rap... Placebo	6 Years -	Palvella Therapeutics, Inc.
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations	NCT06239480	Microcystic Lym...	QTORIN 3.9% Rap... Placebo	6 Years -	Palvella Therapeutics, Inc.
eHealth as an Aid for Facilitating and Supporting Self-management in Families With Long-term Childhood Illness	NCT04150120	Preterm Birth Pediatric Cance... Hirschsprung Di... Congenital Malf... Congenital Hear... Nutrition Disor...	e-health device...	3 Days - 47 Months	Lund University
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations	NCT06239480	Microcystic Lym...	QTORIN 3.9% Rap... Placebo	6 Years -	Palvella Therapeutics, Inc.

Spots Global Cancer Trial Database for congenital malformation