Clinical Trial on Sickle Cell Disease - Clinical Trials Registry

Study Overview

Status

Completed

Conditions

Sickle Cell Disease

Detailed Description

This prospective, observational study will take place, as follows:

Calculation of the PRESEV score in the ER will determine or not eligibility of the patient in the DREPADOM structure. With a low score, a patient is eligible for home monitoring and after duly provided information, can sign a patient consent for the use of personal medical data.

The coordinating nurse from DREPADOM can then set up a whole network, with the delivery of oxygen supply at the patient's house, dispatch of a medical prescription to a neighboring pharmacy and daily visits from homecare service providers. Both opioids and parenteral hydration and treatments can be prescribed and daily visits, up to three, are programmed. As such, the patient receives a minimum of 1 visit per day from a nurse for monitoring and is provided with a medical oxygen concentrator for home use (3L/min continuously or for at least 15h/day) and parenteral hydration. In addition, patients are called daily by general practitioners or SCD experts, for teleconsultations.

The DREPADOM coordinating nurse is in constant interaction with nurses/providers of homecare services/medical doctors and status and evolution of the patient's global condition is reported both through the constant contact with the coordinating nurse from DREPADOM and in a dedicated software (Link4Life), with monitoring of blood pressure, temperature, respiratory rate, pain, etc… Link4Life is a software tool developed especially for home monitoring. It is adaptable to specific disorders, fully complies with the French Law for Health Data management and allows data extraction. Link4Life has been adapted to SCD in full collaboration with SCD medical experts of the Henri Mondor hospital. It is to be noted that all medical doctors (general practitioners or SCD experts), as well as coordinating and visiting nurses have all access to this application. As such, Link4Life will gather and capture all patient data.

When reporting patients' clinical parameters daily in the dedicated app (controlled by the DREPADOM team of experts), alerts are generated in the software when these are above or below determined thresholds. The medical team of experts from DREPADOM then takes over for daily teleconsultations and medical decisions for pursuing home monitoring or hospital admittance.

Of important note, a patient can be included twice in the DREPADOM study, for two distinct VOCs. Indeed, the PRESEV1 study highlighted that each crisis is unique and should be treated accordingly.

Data capture will be done through the Link4Life software, as well as extraction for data analysis.

Open-label
Blind
Number of treatment arms:

Single arm

Multi arms specify the number of arms:

Not Controlled
Controlled, specify the comparator:

Versus placebo Versus reference product Versus other (specify) -Randomized: Parallel group Cross over Other (specify)

-Intervention (gas and/or drug, and/or device, and/or service /study arm):

Study Type

Observational

Enrollment (Actual)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

France
- - Creteil, France, 94000
    - Henri Mondor Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Sickle Cell Disease (SCD) is the most widespread genetic disease in the world. With an incidence rate of approximately 1/3000 newborns each year in France, International Standards consider SCD a rare disease. However, in the Ile-de-France region, that incidence drops to 1/435 newborns, rendering SCD a major territorial issue. It is estimated that more than 10 000 SCD patients live in this region, which represents approximately 30% of the French SCD population and 10% of the European SCD population.

Vaso-occlusive crisis (VOC) or acute bone pain vaso-occlusion is a characteristic manifestation of SCD and represents the first cause of patients 'hospitalizations. Moreover, 16% of VOCs can develop into a potentially severe complication: the acute chest syndrome (ACS).

Description

Inclusion Criteria:

SS or Sβ0
Adult patients with a low PRESEV score

Exclusion Criteria:

SC/Sβ+ patients
SCD adult patients with an intermediate or high PRESEV score
Pregnant or breastfeeding women
Homeless patient
Patients without Social Security
Patient deprived of liberty by judicial or administrative decision or patient under guardianship

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Observational Models: Cohort
Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of acute chest pain Time Frame: 30 months	The primary endpoint is the incidence of Acute chest pain complications developed by patients in the DREPADOM system ( Home Care Services and Hospitalizations for Sickle Cell Disease Patients) and comparison with the historical cohorts of( PRESEV1 and PRESEV2 studies).	30 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of clinical complications associated to Sickle cell disease Time Frame: 30 months	Transfusions and comparison with historical cohorts of PRESEV1 and PRESEV2 studies Patient admission in intensive care unit and comparison with historical cohorts of PRESEV1 and PRESEV2 studies Mortality and comparison with historical cohorts of PRESEV1 and PRESEV2 studies	30 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Soutien aux Actions contre les Maladies du Globule Rouge

Investigators

Principal Investigator: PABLO BARTOLUCCI, PROFESSOR, Soutien aux Actions contre les Maladies du Globule Rouge

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 1, 2021

Primary Completion (Actual)

February 1, 2022

Study Completion (Actual)

June 1, 2022

Study Registration Dates

First Submitted