Multiple Sclerosis
Conditions
Brief summary
Primary Objective: To collect blood samples in a new cohort of Relapsing Forms of Multiple Sclerosis (RMS) participants who had developed immune thrombocytopenic purpura (ITP) after LEMTRADA treatment, for future Deoxyribonucleic acid (DNA) analysis as part of a global biomarker project assessing pre-identified candidate single nucleotide polymorphisms (SNPs) associated to the development of ITP after LEMTRADA treatment in RMS participants.
Detailed description
One to 70 days, as screening and inclusion visit(s) were performed the same day or on separate days with a maximum timeframe of 30 days apart. One visit was required for blood draw. However, if a second visit occured, it took place within 40 days of the first visit.
Interventions
OTHERBlood draw for future biomarker analysis
Each participant was required to undergo blood draw for future genetic testing and biomarker analysis as mentioned in the informed consent form (ICF).
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
: * Male and female adult participants who had been treated with LEMTRADA, with development of ITP subsequent to treatment. Participants does not need to be currently on LEMTRADA treatment. * Participants who had signed the study ICF.
Exclusion criteria
\- Not applicable The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | Baseline (Day 1) | Each participant provided a maximum total of 69 milliliter of whole blood sample. Samples were collected in the following order: 1. Deoxyribonucleic acid (DNA) Paired box (PAX)gene tubes (2 tubes), 2. Ribonucleic acid (RNA) PAXgene tubes (2 tubes), 3. Ethylenediaminetetraacetic acid (EDTA) tubes for peripheral blood mononuclear cells (PBMCs) (5 tubes), 4. Serum Separator tube (SST) (1 tube), and 5. Dipotassium Ethylenediaminetetraacetic acid (K2EDTA) tube (1 tube); where DNA PAXgene tubes were used for DNA preparation, RNA PAXgene tubes for RNA preparation, EDTA for PBMC isolation, SST tubes for serum preparation and K2EDTA for plasma preparation. DNA was extracted from peripheral blood for future analysis. |
Countries
United States
Participant flow
Recruitment details
The study was conducted at a single site in the United States. A total of 14 participants were screened between 8 February 2019 to 11 December 2019, of which,1 participant was screen failure. Screen failures was mainly due to inclusion criteria not met.
Pre-assignment details
A total of 13 participants were enrolled in the study.
Participants by arm
| Arm | Count |
|---|---|
| Participants Diagnosed With ITP Participants with RMS who developed ITP after Lemtrada treatment were included in this study and provided blood samples for future genetic testing and biomarker analysis. | 13 |
| Total | 13 |
Baseline characteristics
| Characteristic | Participants Diagnosed With ITP |
|---|---|
| Age, Continuous | 48.2 years STANDARD_DEVIATION 7.8 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 13 Participants |
| Sex: Female, Male Female | 10 Participants |
| Sex: Female, Male Male | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 13 |
| other Total, other adverse events | 0 / 13 |
| serious Total, serious adverse events | 0 / 13 |
Outcome results
Primary
Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis
Each participant provided a maximum total of 69 milliliter of whole blood sample. Samples were collected in the following order: 1. Deoxyribonucleic acid (DNA) Paired box (PAX)gene tubes (2 tubes), 2. Ribonucleic acid (RNA) PAXgene tubes (2 tubes), 3. Ethylenediaminetetraacetic acid (EDTA) tubes for peripheral blood mononuclear cells (PBMCs) (5 tubes), 4. Serum Separator tube (SST) (1 tube), and 5. Dipotassium Ethylenediaminetetraacetic acid (K2EDTA) tube (1 tube); where DNA PAXgene tubes were used for DNA preparation, RNA PAXgene tubes for RNA preparation, EDTA for PBMC isolation, SST tubes for serum preparation and K2EDTA for plasma preparation. DNA was extracted from peripheral blood for future analysis.
Time frame: Baseline (Day 1)
Population: Analysis was performed on all enrolled population.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | DNA PAXgene (tube 1) | 12 Participants |
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | DNA PAXgene (tube 2) | 12 Participants |
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | RNA PAXgene (tube 1) | 12 Participants |
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | RNA PAXgene (tube 2) | 12 Participants |
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | EDTA for PBMCs (tube 1) | 13 Participants |
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | EDTA for PBMCs (tube 2) | 13 Participants |
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | EDTA for PBMCs (tube 3) | 13 Participants |
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | EDTA for PBMCs (tube 4) | 13 Participants |
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | EDTA for PBMCs (tube 5) | 5 Participants |
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | SST tube | 12 Participants |
| Participants Diagnosed With ITP | Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis | K2EDTA tube | 12 Participants |