Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Rare Diseases

Syndrome

Hemorrhage

Preleukemia

Myelodysplastic Syndromes

Thrombocytopenia

Hematologic Neoplasms

Anemia, Aplastic

Myeloproliferative Disorders

Anemia

Bone Marrow Diseases

Hematologic Diseases

Blood Platelet Disorders

Cytopenia

Bone Marrow Failure Disorders

Pancytopenia

Aplastic Anemia

Chronic Myeloproliferative Disorders

Coagulants

All Drugs and Chemicals

Hemostatics

Human platelet derived lyophilized hemostatic

Thrombosomes

Leukocyte reduced apheresis platelets or whole blood derived pooled platelet concentrate equivalent (4-6 units)

Liquid Stored Platelets (LSP)

Terry Gernsheimer, MD

Mike Fitzpatrick, PhD

This prospective, multicenter, randomized, open-label, Phase 2, parallel, dose ranging, multidose trial will enroll patients into 3 Thrombosomes dose groups and 1 control liquid stored platelets (LSP) group in order to evaluate, in a dose-escalation manner, the safety, and impact on bleeding, and the preliminary effect on coagulation measures of increasing doses of allogeneic Thrombosomes in comparison to standard of care, LSP.

Thrombosomes® in Bleeding Thrombocytopenic Patients Study

A Prospective, Multicenter, Randomized, Open-Label Phase 2, Parallel, Dose Ranging Multidose Study of Thrombosomes® vs Liquid Stored Platelets (LSP) in Bleeding Thrombocytopenic Patients

Unanticipated problems involving risk to human subjects

Cessation or decrease in bleeding at primary bleeding site, based upon the most severe bleeding location at Day 1 baseline taken with in 12 hours prior to infusion, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score evaluated at 24 hours post initial infusion.

Number of days alive and without WHO (World Health Organization) Grade 2 or greater bleeding through initial 7 days after first Thrombosomes or LSP Infusion

30-day mortality post first infusion of Thrombosomes or post first infusion of LSP as control

Cessation or decrease in primary bleeding site, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score at 24, 48, 72 hours, Day 4, Day 5, Day 6 and Day 7 after first infusion of Thrombosomes or LSP infusion as control.

Cessation or decrease in each additional bleeding site (other than primary bleeding site), as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score at 24, 48, 72 hours, Day 4, Day 5, Day 6 and Day 7 after first infusion of Thrombosomes or LSP infusion as control.

Number, timing, type and reason for administration of all blood products including platelets and Thrombosomes during the initial 7 days after first Thrombosomes or LSP infusion

Platelet count measured at 24, 48, 72 hours and Day 7 of first infusion of Thrombosomes or LSP. Also evaluate at Day 4-6 if patient is hospitalized at that time.

Measures of hematology including: Prothrombin Fragment 1+2; thrombin generation assay (TGA); Thrombopoietin; activated Protein C, tissue plasminogen activator (TPA), and plasminogen activator inhibitor (PAI) per schedule of assessments

Measures of coagulation including: prothrombin time (PT); international normalized ratio (INR); fibrinogen; D-dimer; activated partial thromboplastin time (aPTT); and thromboelastography (TEG) or rotational thromboelastometry (ROTEM) per schedule of assessments

Changes in markers of endothelial cell injury/repair from preinfusion baseline through 72 hours after first infusion, including: Syndecan-1, hyaluronan, thrombomodulin, vascular endothelial growth factor (VEGF), interleukin 6, sVE cadherin per schedule of assessments.

Department of Health and Human Services

Cellphire Therapeutics, Inc.

Immune System Diseases

Diseases and Abnormalities at or Before Birth

Nutritional and Metabolic Diseases

Leukemia

Immunologic Deficiency Syndromes

Hemoglobinopathies

Severe Combined Immunodeficiency

Genetic Diseases, Inborn

Primary Immunodeficiency Diseases

Infant, Newborn, Diseases

DNA Repair-Deficiency Disorders

Metabolic Diseases

Acute Graft Versus Host Disease

The intervention consists of an adapted physical activity (APA) program defined at baseline, incorporating supervised sessions with an APA teacher, as well as autonomous sessions. The program is individualized according to age, aerobic capacity, and PA preferences.

The total duration of the intervention is a maximum of 3 months (during the period of hospitalization in a sterile room).

Supervised sessions of at least 15 minutes 4 times a Week combined with unsupervised sessions.

The supervised activities include moderate-intensity aerobic activities (exercise bike), muscle strengthening (dumbbells, elastic bands, medicine balls) and playful situations (ball games, cooperative games, body expression games).

Each patient can also do unsupervised sessions, autonomously, on the bike thanks to a smartphone application developed by the Kiplin company, with challenges to be carried out by teams of 2 to 5 people (team including several patients, possibility to include relatives) over periods of 1 to 3 weeks per challenge.

Physical activity program

Carine HALFON-DOMENECH, MD

To date, allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes, haemoglobinopathies, bone marrow aplasia, severe combined immunodeficiency). Despite the major therapeutic progress made over the last 50 years, particularly in terms of supportive care, post-transplant morbidity and mortality remains high. Infectious complications, whose incidence varies between 30 and 60%, are the first cause of mortality in the immediate post-transplant period.

In order to protect the patient from the occurrence of severe infectious episodes, aHSCTmust be performed in a highly protected environment (positive pressure chambers).

This has implications for the experience and impact of hospitalization on the patient and family. This is particularly true in paediatrics, whether in children, adolescents or young adults, where it is not only the patient's quality of life that is at stake, but also their emotional and psychomotor development. In these patients, prolonged hospitalization (at least 6 weeks) in a sterile room will be responsible for physical deconditioning accompanied by a decrease in muscle mass, itself concomitant with undernutrition, and an increase in sedentary lifestyle. This prolonged hospitalisation in a sterile room, associated with myeloablative treatments, is therefore the cause of social isolation of patients, but it is also often synonymous with physical inactivity leading to a rapid decrease in physical condition, quality of life and an increase in fatigue.

Today, the benefits of physical activity (PA) during and after cancer treatment have been widely demonstrated.

The objective is to evaluate the feasibility of an adapted physical activity program during the isolation phase for achieving aHSCT in children, adolescents and young adults. This is a prospective, interventional, monocentric cohort study conducted at the Institute of Paediatric Haematology and Oncology in Lyon. The intervention will take place during the isolation phase and consists of an adapted physical activity (APA) program defined at inclusion, integrating supervised sessions with an APA teacher, as well as autonomous sessions. The program is individualized according to age, aerobic capacity, and PA preferences. Sessions are also tailored to the biological, psychological, and social parameters of patients.

The total duration of the intervention is 3 months. To date, no PA studies have been performed in patients under 21 years of age requiring aGCSH during the sterile isolation phase. EVAADE will therefore be the first study in this population to offer an innovative procedure with a connected device.

A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts

A Feasibility Study to Assess Connected Bikes as a Means of Adapted Physical Activity in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts

achieved/non-achieved (achived meaning the patient attended at least one session of 15 minutes weekly during the whole program

Centre Leon Berard

The intervention will take place during the whole isolation phase and will consist of an APA program defined at inclusion, integrating supervised sessions with an APA teacher, as well as unsupervised sessions. The program will be individualized according to the patient's age, physical condition and PA preferences. The sessions will also be adapted to the biological, psychological and social parameters of the patients.

The intervention will be carried out during the entire hospitalization period and for a maximum of 3 months. An connected bike will be installed in the patient's room for the duration of the hospitalization.

The supervised sessions will be offered 4 times a week in the participants' rooms by the APA teachers and will include moderate intensity aerobic activities (exercise bike), muscle strengthening (dumbbells, elastic bands, medicine balls) and playful situations (ball games, cooperative games, body expression games).

Each patient will also be able to perform unsupervised sessions (on their own), following the advice given by the APA teacher. These unsupervised sessions can be done independently on the bike.

In order to encourage patients to perform these unsupervised sessions, patients and their relatives will be given a connected device and will have access to a mobile application developed by the Kiplin company, which offers connected physical activity games to be performed in teams (from 2 to 4 participants). These games, lasting from 1 to 3 weeks, use the participants' daily PA level as a unit of animation and are punctuated by challenges.

Rodolf MONGONDRY, MSc

Olivia PEROL, MSc

Cécile RENARD, MD

Allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes haemoglobinopathies, bone narrow aplasia, severe combined immunodeficiency). Despite the major therapeutic progress made over the last 50 years, particularly in terms of supportive care, post-transplant morbidity and mortality remain high. Infectious complications, whose incidence varies between 30 and 60%, are the first cause of mortality in the immediate post-transplant period.

In order to protect the patient from the occurence of severe infectious episodes, aHSCT must be performed in a highly protected environment (positive pressure chambers).

This has consequences for the experience and impact of hospitalization on the patient and family. This is particularly true in pediatrics, with children, adolescents or young adults, where it is not only the patient's quality of life that is at stake, but also his emotional and psychomotor development. In this specific population, prolonged hospitalization (at least 6 weeks) in a sterile room will be responsible for physical deconditioning accompanied by a decrease in muscle mass. Patients often experience an deteriorated quality of life.

Today, the benefits of physical activity (PA) during and after cancer treatment have been widely demonstrated.

The objective of the study is to assess the feasibility of an adapted physical activity program during the isolation phase for achieving aHSCT in children, adolescents and young adults. This is a prospective, interventional, monocentric cohort study conducted at the institute of Paediatric Haematology and Oncology in Lyon. The intervention will take place during the isolation phase and will be based on an adapted physical activity (APA) program defined at inclusion, integrating supervised sessions with an APA teacher, as well as autonomous sessions performed by means of a connected bike in the sterile room. The program will be individualized according to age, aerobic capacities, and PA preferences. Sessions will also be tailored to the biological, psychological, and social parameters of patients.

The total duration of the intervention is 3 months. To date, no PA studies have been performed in patients under 21 years old requiring aHSCT during the sterile isolation phase. EVAADE will therefore be the first study in this population to offer an innovative procedure with a connected device.

Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation

A Feasibility Study Evaluating Connected Bikes as a Means of Adapted Physical Activity in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation

achieved/non achieved (achieved meaning the patient attended at least one session of 15 minutes weekly during the whole program, considering both supervised and unsupervised sessions)

Rate of unsupervised sessions/total of performed sessions

PA practice of patient / PA practice of relatives

Number of enrolled patients/number of eligible patients

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Thrombosomes® in Bleeding Thrombocytopenic Patients Study	NCT04631211	Thrombocytopeni... Hematologic Mal... Bone Marrow Apl... Myeloproliferat... Myelodysplastic... Platelet Refrac...	Thrombosomes Liquid Stored P...	18 Years -	Cellphire Therapeutics, Inc.
A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts	NCT04331483	Haematological ... Acute Leukemia Myelodysplastic... Hemoglobinopath... Bone Marrow Apl... Severe Combined...	Physical activi...	8 Years - 21 Years	Centre Leon Berard
A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts	NCT04331483	Haematological ... Acute Leukemia Myelodysplastic... Hemoglobinopath... Bone Marrow Apl... Severe Combined...	Physical activi...	8 Years - 21 Years	Centre Leon Berard
Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation	NCT05298930	Haematological ... Acute Leukemia Myelodysplastic... Hemoglobinopath... Bone Marrow Apl... Severe Combined...	Physical activi...	8 Years - 21 Years	Centre Leon Berard

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