Trial Outcomes & Findings for Long-Term Outcomes and Durability of Effect Following Treatment With Cladribine Tablets for MS (CLASSIC-MS) (NCT NCT03961204)

NCT ID: NCT03961204

Last Updated: 2023-10-31

Results Overview

EDSS is a scale based on standardized neurological examination which comprised of optic, brain stem, pyramidal, cerebellar, sensory \& cerebral functions, as well as walking ability. EDDS is a scale from 0-10 that evaluates a person with Multiple Sclerosis (MS) disability/neurologic function level where 0=normal and 10=death due to MS. Score of 7.0 is defined as unable to walk beyond approximately 5 meters even with aid, essentially restricted to wheelchair; wheels self in standard wheelchair and transfers alone; up and about in wheelchair some 12 hours a day. Score of 8.0 is defined as Essentially restricted to bed or chair or perambulated in wheelchair, but may be out of bed itself much of the day; retains many self-care functions; generally has effective use of arms.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

662 participants

Primary outcome timeframe

3 months prior to study visit 1. Retrospectively from end of parent study (NCT00213135, NCT00641537 and NCT00725985) to study visit 1 (study visit 1 occurred up to 3 months from screening)

Results posted on

2023-10-31

Participant Flow

A total of 662 subjects were enrolled in this trial at different sites in United States and Europe.

Participant milestones

Participant milestones
Measure
Cohort A
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Overall Study
STARTED
662
Overall Study
COMPLETED
655
Overall Study
NOT COMPLETED
7

Reasons for withdrawal

Reasons for withdrawal
Measure
Cohort A
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Overall Study
Withdrawal by Subject
2
Overall Study
Lost to Follow-up
3
Overall Study
Other
1
Overall Study
Missing
1

Baseline Characteristics

Long-Term Outcomes and Durability of Effect Following Treatment With Cladribine Tablets for MS (CLASSIC-MS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Cohort A
n=662 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Age, Continuous
49.3 Years
STANDARD_DEVIATION 10.32 • n=5 Participants
Sex: Female, Male
Female
444 Participants
n=5 Participants
Sex: Female, Male
Male
218 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
8 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
3 Participants
n=5 Participants
Race (NIH/OMB)
White
645 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
6 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 3 months prior to study visit 1. Retrospectively from end of parent study (NCT00213135, NCT00641537 and NCT00725985) to study visit 1 (study visit 1 occurred up to 3 months from screening)

Population: FAS included all participants participating in CLASSIC study \[randomized in CLARITY and have received ≥ 1 course of investigational medicinal product (IMP) (Cladribine Tablets or placebo) or participants randomized in the ORACLE study and have received ≥ 1 course of IMP\]. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure.

EDSS is a scale based on standardized neurological examination which comprised of optic, brain stem, pyramidal, cerebellar, sensory \& cerebral functions, as well as walking ability. EDDS is a scale from 0-10 that evaluates a person with Multiple Sclerosis (MS) disability/neurologic function level where 0=normal and 10=death due to MS. Score of 7.0 is defined as unable to walk beyond approximately 5 meters even with aid, essentially restricted to wheelchair; wheels self in standard wheelchair and transfers alone; up and about in wheelchair some 12 hours a day. Score of 8.0 is defined as Essentially restricted to bed or chair or perambulated in wheelchair, but may be out of bed itself much of the day; retains many self-care functions; generally has effective use of arms.

Outcome measures

Outcome measures
Measure
Cohort A
n=636 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Percentage of Participants Using Wheelchair or Being Bedridden Assessed by Expanded Disability Status Scale (EDSS) Score 7.0 or Higher
8.2 percentage of participant
Interval 6.2 to 10.6

SECONDARY outcome

Timeframe: At study visit 1. Retrospectively after last IMP administration from parent study (NCT00213135, NCT00641537 and NCT00725985) to study visit 1 (study visit 1 occurred up to 3 months from screening)

Population: FAS included all participants participating in CLASSIC study \[randomized in CLARITY and have received ≥ 1 course of IMP (Cladribine Tablets or placebo) or participants randomized in the ORACLE study and have received ≥ 1 course of IMP\].

EDSS is a scale based on standardized neurological examination which comprised of optic, brain stem, pyramidal, cerebellar, sensory \& cerebral functions, as well as walking ability. EDDS is a scale from 0-10 that evaluates a person with MS disability/neurologic function level where 0= normal and 10= death due to MS. Score of 6.0 is defined as "intermittent or unilateral constant assistance (cane, crutch and brace) required to walk about 100 meters with or without resting".

Outcome measures

Outcome measures
Measure
Cohort A
n=662 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Percentage of Participants With Expanded Disability Status Scale (EDSS) Score 6.0 or Higher
13.9 percentage of participant
Interval 11.4 to 16.8

SECONDARY outcome

Timeframe: At study visit 1, occurred up to 3 months from screening (Retrospective analysis of medical record of parent study-NCT00213135, NCT00641537 and NCT00725985)

Population: FAS included all participants participating in CLASSIC study \[randomized in CLARITY and have received ≥ 1 course of IMP (Cladribine Tablets or placebo) or participants randomized in the ORACLE study and have received ≥ 1 course of IMP\]. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and and "Number analyzed" refers to number of participants evaluable for specified categories.

Clinical and demographic characteristics including age and disease duration is reported in the form of long term responders and non-responder. Here long term responder is defined as study participants not requiring disease modifying drug (DMD) 4 years or later following their last dose of IMP in parent study. Non-responder is defined as study participants requiring DMD less than 4 years following their last dose of IMP in parent study.

Outcome measures

Outcome measures
Measure
Cohort A
n=627 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Clinical and Demographic Characteristic: Age, Disease Duration
Long-term responders: Age at study visit 1
50.5 years
Standard Deviation 10.65
Clinical and Demographic Characteristic: Age, Disease Duration
Non-responders: Age at study visit 1
47.1 years
Standard Deviation 9.47
Clinical and Demographic Characteristic: Age, Disease Duration
Long term responder: Disease duration
19.82 years
Standard Deviation 9.161
Clinical and Demographic Characteristic: Age, Disease Duration
Non-responder: Disease duration
16.82 years
Standard Deviation 8.321

SECONDARY outcome

Timeframe: At study visit 1, occurred up to 3 months from screening (Retrospective analysis of medical record of parent study-NCT00213135, NCT00641537 and NCT00725985)

Population: FAS included all participants participating in CLASSIC study \[randomized in CLARITY and have received ≥ 1 course of IMP (Cladribine Tablets or placebo) or participants randomized in the ORACLE study and have received ≥ 1 course of IMP\]. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number analyzed" refers to number of participants evaluable for specified categories.

Clinical and demographic characteristics included gender, race, disease classification (relapsing remitting multiple sclerosis \[RRMS\], Secondary Progressive Multiple Sclerosis \[SPMS\], unknown \& no MS disease), Prior use of DMDs \& high-disease activity (HAD) status, education level and employment status. Number of participants in each category of clinical and demographic characteristics were reported in form of long term responders \& non-responder. Here long term responder is defined as participants not requiring DMD 4 years or later following their last dose of IMP in parent study. Non-responder is defined as study participants requiring DMD \< 4 years following their last dose of IMP in parent study.

Outcome measures

Outcome measures
Measure
Cohort A
n=627 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Prior Use of DMDs
57 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non- responder: Education level (8 to 10 Years)
43 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Sex (Female)
251 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Sex (Female)
171 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long-term responder: Sex (Male)
127 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Sex (Male)
78 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Race (White)
368 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Race (White)
244 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Race (Black or African American)
1 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Race (Black or African American)
0 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Race (Asian)
5 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Race (Asian)
3 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Race (Other)
4 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Race (Other)
2 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Type of MS-RRMS
259 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Type of MS-RRMS
173 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Type of MS-SPMS
71 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Type of MS-SPMS
41 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Type of MS-Unknown
0 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Type of MS -Unknown
14 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Type of MS-No MS disease
48 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Type of MS-No MS disease
21 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Prior Use of DMDs
33 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: HDA participants
83 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: HDA participants
35 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Education level (Below 8 Years)
19 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Education level (Below 8 Years)
16 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Education level (8 to 10 Years)
73 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Education level (10 to 15 Years)
190 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Education level (10 to 15 Years)
130 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Education level (Over 15 Years)
91 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Education level (Over 15 Years)
56 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Employment Status (Employed for wages)
160 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Employment Status (Employed for wages)
122 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Employment Status (Self-Employed)
38 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Employment Status (Self-Employed)
17 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Employment Status (Out of Work for more than 1 year)
12 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Employment Status (Out of Work for more than 1 year)
13 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Employment Status (Out of Work for less than 1 year)
4 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Employment Status (Out of Work for less than 1 year)
2 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Employment Status (A Homemaker)
22 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Employment Status (A Homemaker)
24 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Employment Status (Retired)
67 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Employment Status (Retired)
19 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Employment Status (Unable to work)
48 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Employment Status (Unable to work)
26 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Employment Status (Not collected at the site)
3 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Employment Status (Not collected at the site)
6 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Long term responder: Employment Status (Unknown/Not reported)
24 Participants
Number of Participants in Each Category of Clinical and Demographic Characteristics
Non-responder: Employment Status (Unknown/Not reported)
20 Participants

SECONDARY outcome

Timeframe: At study visit 1, occurred up to 3 months from screening (Retrospective analysis of medical record of parent study-NCT00213135, NCT00641537 and NCT00725985)

Population: FAS included all participants participating in CLASSIC study \[randomized in CLARITY and have received ≥ 1 course of IMP (Cladribine Tablets or placebo) or participants randomized in the ORACLE study and have received ≥ 1 course of IMP\]. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number analyzed" refers to number of participants evaluable for specified categories.

EDSS is a scale based on standardized neurological examination which comprised of optic, brain stem, pyramidal, cerebellar, sensory \& cerebral functions, as well as walking ability. EDSS scores range from 0.0 (normal) to 10.0 (dead). Clinical characteristics of EDSS score in form of long-term responders \& non-responder was reported for at parent study baseline (based on retrospective data collection \[based on chart review\] at study visit 1) \& study visit 1. Here long term responder is defined as study participants not requiring DMD 4 years or later following their last dose of IMP in parent study. Non-responder is defined as study participants requiring DMD \< 4 years following their last dose of IMP in parent study.

Outcome measures

Outcome measures
Measure
Cohort A
n=627 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Clinical Characteristic: Expanded Disability Status Scale (EDSS) Score
Long-term responders: EDSS score at parent study baseline
2.44 score on a scale
Standard Deviation 1.292
Clinical Characteristic: Expanded Disability Status Scale (EDSS) Score
Non-responders: EDSS score at parent study baseline
2.38 score on a scale
Standard Deviation 1.251
Clinical Characteristic: Expanded Disability Status Scale (EDSS) Score
Long term responder: EDSS score at study visit 1
3.23 score on a scale
Standard Deviation 2.121
Clinical Characteristic: Expanded Disability Status Scale (EDSS) Score
Non-responder: EDSS score at study visit 1
3.32 score on a scale
Standard Deviation 2.102

SECONDARY outcome

Timeframe: At study visit 1, occurred up to 3 months from screening (Retrospective analysis of medical record of parent study-NCT00213135, NCT00641537 and NCT00725985)

Population: FAS included all participants participating in CLASSIC study \[randomized in CLARITY and have received ≥ 1 course of IMP (Cladribine Tablets or placebo) or participants randomized in the ORACLE study and have received ≥ 1 course of IMP\]. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number analyzed" refers to number of participants evaluable for specified categories.

Relapse was defined as participant-reported symptoms \& objectively observed signs typical of an acute inflammatory demyelinating event in CNS, developing acutely or sub-acutely with duration of at least 24 hours, in absence of fever or infection. Clinical characteristics of number of relapses during last year before enrollment of parent study (it is reported based on retrospective data collection \[based on chart review\] at study visit 1) in the form of long-term responders \& non-responder was reported. Here long term responder is defined as study participants not requiring DMD 4 years or later following their last dose of IMP in parent study. Non-responder is defined as study participants requiring DMD less than 4 years following their last dose of IMP in parent study.

Outcome measures

Outcome measures
Measure
Cohort A
n=408 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Clinical Characteristic: Number of Relapses
Long-term responders: number of relapses during last year before enrollment of parent study
1.3 Relapses
Standard Deviation 0.64
Clinical Characteristic: Number of Relapses
Non-responders: number of relapses during last year before enrollment of parent study
1.3 Relapses
Standard Deviation 0.56

SECONDARY outcome

Timeframe: At study visit 2, within 6 months from screening (Retrospective analysis of medical record of parent study-NCT00213135, NCT00641537 and NCT00725985)

Population: The MRI analysis population includes all FAS participants who signed the MRI sub- study informed consent. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number analyzed" refers to number of participants evaluable for specified categories.

Total number of T1-W lesion were measured by Using magnetic resonance imaging (MRI) Scans. Here long term responder is defined as study participants not requiring DMD 4 years or later following their last dose of IMP in parent study. Non-responder is defined as study participants requiring DMD less than 4 years following their last dose of IMP in parent study.

Outcome measures

Outcome measures
Measure
Cohort A
n=39 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Number of Total T1-weighted (T1-W) Lesions
Long-term responder
12.7 lesions
Standard Deviation 8.97
Number of Total T1-weighted (T1-W) Lesions
Non-responder
16.1 lesions
Standard Deviation 10.90

SECONDARY outcome

Timeframe: At study visit 2, within 6 months from screening (Retrospective analysis of medical record of parent study-NCT00213135, NCT00641537 and NCT00725985)

Population: The MRI analysis population includes all FAS participants who signed the MRI sub- study informed consent. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number analyzed" refers to number of participants evaluable for specified categories.

Total number of T2-W lesion were measured by Using magnetic resonance imaging (MRI) Scans. Here long term responder is defined as study participants not requiring DMD 4 years or later following their last dose of IMP in parent study. Non-responder is defined as study participants requiring DMD less than 4 years following their last dose of IMP in parent study.

Outcome measures

Outcome measures
Measure
Cohort A
n=41 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Number of Total T2-weighted (T2-W) Lesions
Long-term responder
20.2 lesions
Standard Deviation 18.67
Number of Total T2-weighted (T2-W) Lesions
Non-responder
25.1 lesions
Standard Deviation 18.17

SECONDARY outcome

Timeframe: At study visit 2, within 6 months from screening (Retrospective analysis of medical record of parent study-NCT00213135, NCT00641537 and NCT00725985)

Population: The MRI analysis population includes all FAS participants who signed the MRI sub- study informed consent. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number analyzed" refers to number of participants evaluable for specified categories.

T1-W lesion volume were measured by Using magnetic resonance imaging (MRI) Scans. Here long term responder is defined as study participants not requiring DMD 4 years or later following their last dose of IMP in parent study. Non-responder is defined as study participants requiring DMD less than 4 years following their last dose of IMP in parent study.

Outcome measures

Outcome measures
Measure
Cohort A
n=38 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
T1-weighted (T1-W) Lesion Volume
Long-term responder
1.655 cubic centimeter (cm^3)
Standard Deviation 1.3953
T1-weighted (T1-W) Lesion Volume
Non-responder
6.773 cubic centimeter (cm^3)
Standard Deviation 6.4788

SECONDARY outcome

Timeframe: At study visit 2, within 6 months from screening (Retrospective analysis of medical record of parent study-NCT00213135, NCT00641537 and NCT00725985)

Population: The MRI analysis population includes all FAS participants who signed the MRI sub- study informed consent. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number analyzed" refers to number of participants evaluable for specified categories.

T2-W lesion volume were measured by Using magnetic resonance imaging (MRI) Scans. Here long term responder is defined as study participants not requiring DMD 4 years or later following their last dose of IMP in parent study. Non-responder is defined as study participants requiring DMD less than 4 years following their last dose of IMP in parent study.

Outcome measures

Outcome measures
Measure
Cohort A
n=41 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
T2-weighted (T2-W) Lesion Volume
Long-term responder
4.920 cubic centimeter (cm^3)
Standard Deviation 6.7665
T2-weighted (T2-W) Lesion Volume
Non-responder
14.664 cubic centimeter (cm^3)
Standard Deviation 13.8109

SECONDARY outcome

Timeframe: At study visit 2, within 6 months from screening (Retrospective analysis of medical record of parent study-NCT00213135, NCT00641537 and NCT00725985)

Population: The MRI analysis population includes all FAS participants who signed the MRI sub- study informed consent. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number analyzed" refers to number of participants evaluable for specified categories.

Brain volume were measured by Using magnetic resonance imaging (MRI) Scans. Here long term responder is defined as study participants not requiring DMD 4 years or later following their last dose of IMP in parent study. Non-responder is defined as study participants requiring DMD less than 4 years following their last dose of IMP in parent study.

Outcome measures

Outcome measures
Measure
Cohort A
n=39 Participants
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Total Brain Volume
Long-term responder
1472.559 cubic centimeter (cm^3)
Standard Deviation 59.9500
Total Brain Volume
Non-responder
1417.431 cubic centimeter (cm^3)
Standard Deviation 109.8668

Adverse Events

Cohort A

Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Cohort A
n=662 participants at risk
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Prostate cancer
0.15%
1/662 • Up to study visit 2 (Up to approximately 6 months and Retrospective AEs data collection based on chart review of participants from end of parent studies; NCT00213135, NCT00641537 and NCT00725985)

Other adverse events

Other adverse events
Measure
Cohort A
n=662 participants at risk
Participants previously enrolled in parent studies CLARITY (NCT00213135), CLARITY-EXT (NCT00641537), ORACLE (NCT00725985) and had received Cladribine tablet and Placebo were invited up to 2 visit for follow-up/data collection.
Blood and lymphatic system disorders
Lymphopenia
0.30%
2/662 • Up to study visit 2 (Up to approximately 6 months and Retrospective AEs data collection based on chart review of participants from end of parent studies; NCT00213135, NCT00641537 and NCT00725985)
Blood and lymphatic system disorders
Leukopenia
0.30%
2/662 • Up to study visit 2 (Up to approximately 6 months and Retrospective AEs data collection based on chart review of participants from end of parent studies; NCT00213135, NCT00641537 and NCT00725985)
Blood and lymphatic system disorders
Neutropenia
0.15%
1/662 • Up to study visit 2 (Up to approximately 6 months and Retrospective AEs data collection based on chart review of participants from end of parent studies; NCT00213135, NCT00641537 and NCT00725985)
Infections and infestations
Nasopharyngitis
0.15%
1/662 • Up to study visit 2 (Up to approximately 6 months and Retrospective AEs data collection based on chart review of participants from end of parent studies; NCT00213135, NCT00641537 and NCT00725985)
Endocrine disorders
Autoimmune thyroid disorder
0.15%
1/662 • Up to study visit 2 (Up to approximately 6 months and Retrospective AEs data collection based on chart review of participants from end of parent studies; NCT00213135, NCT00641537 and NCT00725985)
Investigations
Hepatic enzyme increased
0.15%
1/662 • Up to study visit 2 (Up to approximately 6 months and Retrospective AEs data collection based on chart review of participants from end of parent studies; NCT00213135, NCT00641537 and NCT00725985)
Vascular disorders
Flushing
0.15%
1/662 • Up to study visit 2 (Up to approximately 6 months and Retrospective AEs data collection based on chart review of participants from end of parent studies; NCT00213135, NCT00641537 and NCT00725985)

Additional Information

Communication Center

Merck Healthcare KGaA, Darmstadt, Germany

Phone: +49 6151-72-5200

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place