Collection of Blood Samples From Patients With Relapsing MS Who Developed ITP After Receiving Lemtrada (LEMTRADA-ITP)

• ClinicalTrials.gov Identifier: NCT03784898
• Recruitment Status: Completed
• First Posted: December 24, 2018
• Results First Posted: July 20, 2020
• Last Update Posted: July 20, 2020
• Sponsor: Genzyme, a Sanofi Company
• Information provided by (Responsible Party): Sanofi ( Genzyme, a Sanofi Company )

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Other
• Condition: Multiple Sclerosis
• Intervention: Other: Blood draw for future biomarker analysis
• Enrollment: 13

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.

Arm/Group Title	Participants Diagnosed With ITP
Arm/Group Description	Participants with Relapsing Forms of Multiple Sclerosis (RMS) who developed Immune thrombocytopenic purpura (ITP) after Lemtrada treatment were included in this study and provided blood samples for future genetic testing and biomarker analysis.
Period Title: Overall Study
Started	13
Completed	13
Not Completed	0

Baseline Characteristics

Arm/Group Title		Participants Diagnosed With ITP
Arm/Group Description		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.
Overall Number of Baseline Participants		13
Baseline Analysis Population Description		Analysis was performed on all enrolled participants.
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	13 participants
		48.2 (7.8)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	13 participants
	Female	10 76.9%
	Male	3 23.1%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	13 participants
	American Indian or Alaska Native	0 0.0%
	Asian	0 0.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%
	Black or African American	0 0.0%
	White	13 100.0%
	More than one race	0 0.0%
	Unknown or Not Reported	0 0.0%

Outcome Measures

1. Primary Outcome

Title	Number of Participants Who Provided Whole Blood Samples for DNA Extraction and Analysis
Description	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)

Outcome Measure Data

Analysis Population Description

Analysis was performed on all enrolled population.

Arm/Group Title	Participants Diagnosed With ITP
Arm/Group Description:	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.
Overall Number of Participants Analyzed	13
Measure Type: Count of Participants; Unit of Measure: Participants
DNA PAXgene (tube 1)	12 92.3%
DNA PAXgene (tube 2)	12 92.3%
RNA PAXgene (tube 1)	12 92.3%
RNA PAXgene (tube 2)	12 92.3%
EDTA for PBMCs (tube 1)	13 100.0%
EDTA for PBMCs (tube 2)	13 100.0%
EDTA for PBMCs (tube 3)	13 100.0%
EDTA for PBMCs (tube 4)	13 100.0%
EDTA for PBMCs (tube 5)	5 38.5%
SST tube	12 92.3%
K2EDTA tube	12 92.3%

Adverse Events

Time Frame	Adverse Event data was collected from the signature of the Informed consent form until the end of the study (i.e. up to 307 days)
Adverse Event Reporting Description	Analysis was performed on all enrolled participants.
Arm/Group Title	Participants Diagnosed With ITP
Arm/Group Description	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.
All-Cause Mortality
	Participants Diagnosed With ITP
	Affected / at Risk (%)
Total	0/13 (0.00%)
Serious Adverse Events
	Participants Diagnosed With ITP
	Affected / at Risk (%)
Total	0/13 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Participants Diagnosed With ITP
	Affected / at Risk (%)
Total	0/13 (0.00%)

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.

Results Point of Contact

• Name/Title: Trial Transparency Team
• Organization: Sanofi
• Phone: 800-633-1610 ext 1#
• EMail: Contact-US@sanofi.com

• Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
• ClinicalTrials.gov Identifier: NCT03784898
• Other Study ID Numbers: ASY15905; U1111-1225-1333
• First Submitted: December 20, 2018
• First Posted: December 24, 2018
• Results First Submitted: July 2, 2020
• Results First Posted: July 20, 2020
• Last Update Posted: July 20, 2020