A Safety and Efficacy Study of Oral Cladribine in Subjects With Relapsing-remitting Multiple Sclerosis (RRMS)

NCT ID: NCT00213135

Last Updated: 2014-02-07

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

1326 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-04-30

Study Completion Date

2008-11-30

Brief Summary

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The purpose of the study is to determine if cladribine tablets are a safe and effective treatment for relapsing-remitting multiple sclerosis (RRMS).

Detailed Description

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This is a randomized, double-blind, three-arm, placebo-controlled, multi-center study. The study includes a pre-study evaluation period (up to 28 days prior to the start of treatment); an initial treatment period from Week 1 to 48; and a re-treatment period during Week 49 to 96.

During the initial treatment period (Week 1 to 48), eligible subjects are equally randomized by a central randomization system to receive either a) cladribine at a low dose (0.875 milligram per kilogram per course \[mg/kg/course\] for two courses plus placebo for two courses); b) cladribine at a high dose (0.875 mg/kg/course for four courses); or c) placebo (four courses). During the re-treatment period (Weeks 49 to 96), subjects received either a) cladribine at a low dose (0.875 mg/kg/course for two courses); or b) placebo (two courses).

For all randomized subjects, there is a rescue option of treatment with Rebif® (interferon beta-1a 44 microgram (mcg) given subcutaneously three times a week), if the subject experienced more than one qualifying relapse, and/or experienced a sustained increase in their EDSS score of greater than or equal to (\>=) 1 point, or \>=1.5 points if baseline EDSS score is 0, (over a period of three months or greater), during a calendar year beginning at Week 24.

To maintain the blind, there is a treating physician who view clinical laboratory results and assess adverse events and safety information, and an independent blinded evaluating physician who will perform neurological exams. A central neuroradiology center, also blinded to treatment, will assess magnetic resonance imaging (MRI) evaluations.

Conditions

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Multiple Sclerosis, Relapsing-Remitting

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Cladribine 5.25 mg/kg

Group Type EXPERIMENTAL

Cladribine 5.25 mg/kg

Intervention Type DRUG

Cladribine tablet will be administered as cumulative dose of 0.875 milligram per kilogram (mg/kg) over a course of 4 or 5 consecutive days of 28-day period at Week 1, 5, 9, 13, 48, and 52 resulting in total cladribine dose of 5.25 mg/kg during the treatment period of 96 weeks.

Cladribine 3.5 mg/kg

Group Type EXPERIMENTAL

Cladribine 3.5 mg/kg

Intervention Type DRUG

Cladribine tablet will be administered as cumulative dose of 0.875 mg/kg over a course of 4 or 5 consecutive days of 28-day period at Weeks 1, 5, 48, and 52 and placebo matched to cladribine tablet will be administered at Week 9 and 13 resulting in total cladribine dose of 3.5 mg/kg during the treatment period of 96 weeks.

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Placebo matched to cladribine tablet will be administered over a course of 4 or 5 consecutive days of 28-day period at Weeks 1, 5, 9, 13, 48 and 52 during the treatment period of 96 weeks.

Interventions

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Cladribine 5.25 mg/kg

Cladribine tablet will be administered as cumulative dose of 0.875 milligram per kilogram (mg/kg) over a course of 4 or 5 consecutive days of 28-day period at Week 1, 5, 9, 13, 48, and 52 resulting in total cladribine dose of 5.25 mg/kg during the treatment period of 96 weeks.

Intervention Type DRUG

Cladribine 3.5 mg/kg

Cladribine tablet will be administered as cumulative dose of 0.875 mg/kg over a course of 4 or 5 consecutive days of 28-day period at Weeks 1, 5, 48, and 52 and placebo matched to cladribine tablet will be administered at Week 9 and 13 resulting in total cladribine dose of 3.5 mg/kg during the treatment period of 96 weeks.

Intervention Type DRUG

Placebo

Placebo matched to cladribine tablet will be administered over a course of 4 or 5 consecutive days of 28-day period at Weeks 1, 5, 9, 13, 48 and 52 during the treatment period of 96 weeks.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Male or female, between 18 and 65 years of age (inclusive, at time of informed consent)
* Has definite MS according to the McDonald criteria
* Has relapsing-remitting disease with 1 or more relapses within 12 months prior to Study Day 1
* Must have been clinically stable and not has a relapse within 28 days prior to Study Day 1
* Has MRI consistent with MS at the pre-study evaluation according to the Fazekas criteria
* Has a EDSS score from 0 to 5.5, inclusive
* Weighed between 40-120 kilogram (kg), inclusive
* If female, she must:

1. be post-menopausal or surgically sterilized; or
2. uses a hormonal contraceptive, intra uterine device, diaphragm with spermicide, or condom with spermicide, for the duration of the study; and
3. be neither pregnant nor breast-feeding
* If male, he must be willing to use contraception to avoid pregnancies
* Be willing and able to comply with study procedures for the duration of the study
* Voluntarily provides written informed consent, and for United states of America (USA) sites only, a subject authorization under Health Insurance Portability and Accountability Act (HIPAA)

Exclusion Criteria

* Has secondary progressive MS (SPMS) or primary progressive MS (PPMS)
* Prior use of disease modifying drugs (DMDs) within the last 3 months, or 2 or more prior treatment failures with DMDs on the basis of efficacy
* Has significant leukopenia (white blood cell count less than 0.5 times the lower limit of normal of the central laboratory) within 28 days prior to Study Day 1
* Has received cladribine, mitoxantrone, total lymphoid irradiation, myelosuppressive therapy, campath-1h, cyclophosphamide, azathioprine, methotrexate or natalizumab
* Has received oral or systemic corticosteroids or adrenocorticotropic hormone within 28 days prior to Study Day 1
* Has compromised immune function or infection
* Has received oral or systemic corticosteroids or adrenocorticotropic hormone within 28 days prior to Study Day 1
* Has received cytokine-based therapy, intravenous immunoglobulin therapy, or plasmapheresis within 3 months prior to Study Day 1
* Has platelet and absolute neutrophil counts below the lower limit of normal range within 28 days prior to Study Day 1
* Has prior or current history of malignancy
* Has a history of persistent anemia, leukopenia, neutropenia, or thrombocytopenia after immunosuppressive therapy
* Has systemic disease that, in the opinion of the Investigator, might interfere with subject safety, compliance or evaluation of the condition under Study (for example, insulin-dependent diabetes, Lyme disease, clinically significant cardiac, hepatic, or renal disease, Human Immunodeficiency Virus, or Human T-Cell Lymphotrophic Virus Type-1)
* Has a psychiatric disorder that, in the opinion of the Investigator, was unstable or would preclude safe participation in the study
* Has allergy or hypersensitivity to gadolinium, to cladribine or any of its excipients
* Has used any investigational drug or experimental procedure within 6 months prior to Study Day 1
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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EMD Serono

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Steven J. Greenberg, M.D.

Role: STUDY_DIRECTOR

EMD Serono

References

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Giovannoni G, Comi G, Cook S, Rammohan K, Rieckmann P, Soelberg Sorensen P, Vermersch P, Chang P, Hamlett A, Musch B, Greenberg SJ; CLARITY Study Group. A placebo-controlled trial of oral cladribine for relapsing multiple sclerosis. N Engl J Med. 2010 Feb 4;362(5):416-26. doi: 10.1056/NEJMoa0902533. Epub 2010 Jan 20.

Reference Type RESULT
PMID: 20089960 (View on PubMed)

Stefano N, Sormani MP, Giovannoni G, Rammohan K, Leist TP, Coyle PK, Dangond F, Alexandri N, Galazka A. Relapses in people with multiple sclerosis treated with cladribine tablets followed for up to 5 years: a plain language summary. Neurodegener Dis Manag. 2022 Dec;12(6):303-310. doi: 10.2217/nmt-2022-0019. Epub 2022 Aug 26.

Reference Type DERIVED
PMID: 36017797 (View on PubMed)

Giovannoni G, Comi G, Rammohan K, Rieckmann P, Dangond F, Jack D, Vermersch P. Disease stability over five years in people with multiple sclerosis treated with cladribine tablets: a plain language summary. Neurodegener Dis Manag. 2022 Dec;12(6):295-301. doi: 10.2217/nmt-2022-0018. Epub 2022 Aug 26.

Reference Type DERIVED
PMID: 36017780 (View on PubMed)

Vermersch P, Galazka A, Dangond F, Damian D, Wong SL, Jack D, Harty G. The effect of cladribine tablets in people with more active multiple sclerosis: a plain language summary. Neurodegener Dis Manag. 2022 Dec;12(6):285-293. doi: 10.2217/nmt-2022-0009. Epub 2022 Aug 3.

Reference Type DERIVED
PMID: 35920065 (View on PubMed)

Oh J, Walker B, Giovannoni G, Jack D, Dangond F, Nolting A, Aldridge J, Lebson LA, Leist TP. Side effects that occurred early in people with multiple sclerosis during the first year of treatment with cladribine tablets: a plain language summary. Neurodegener Dis Manag. 2022 Feb 1;12(1):1-7. doi: 10.2217/nmt-2021-0041. Epub 2022 Jan 12.

Reference Type DERIVED
PMID: 35019731 (View on PubMed)

Giovannoni G, Coyle PK, Vermersch P, Walker B, Aldridge J, Nolting A, Galazka A, Lemieux C, Leist TP. Integrated Lymphopenia Analysis in Younger and Older Patients With Multiple Sclerosis Treated With Cladribine Tablets. Front Immunol. 2021 Dec 24;12:763433. doi: 10.3389/fimmu.2021.763433. eCollection 2021.

Reference Type DERIVED
PMID: 35003076 (View on PubMed)

Giovannoni G, Comi G, Rammohan K, Rieckmann P, Dangond F, Keller B, Jack D, Vermersch P. Long-Term Disease Stability Assessed by the Expanded Disability Status Scale in Patients Treated with Cladribine Tablets 3.5 mg/kg for Relapsing Multiple Sclerosis: An Exploratory Post Hoc Analysis of the CLARITY and CLARITY Extension Studies. Adv Ther. 2021 Sep;38(9):4975-4985. doi: 10.1007/s12325-021-01865-w. Epub 2021 Aug 9.

Reference Type DERIVED
PMID: 34370275 (View on PubMed)

De Stefano N, Sormani MP, Giovannoni G, Rammohan K, Leist T, Coyle PK, Dangond F, Keller B, Alexandri N, Galazka A. Analysis of frequency and severity of relapses in multiple sclerosis patients treated with cladribine tablets or placebo: The CLARITY and CLARITY Extension studies. Mult Scler. 2022 Jan;28(1):111-120. doi: 10.1177/13524585211010294. Epub 2021 May 10.

Reference Type DERIVED
PMID: 33969750 (View on PubMed)

Giovannoni G, Galazka A, Schick R, Leist T, Comi G, Montalban X, Damian D, Dangond F, Cook S. Pregnancy Outcomes During the Clinical Development Program of Cladribine in Multiple Sclerosis: An Integrated Analysis of Safety. Drug Saf. 2020 Jul;43(7):635-643. doi: 10.1007/s40264-020-00948-x.

Reference Type DERIVED
PMID: 32447743 (View on PubMed)

Terranova N, Hicking C, Dangond F, Munafo A. Effects of Postponing Treatment in the Second Year of Cladribine Administration: Clinical Trial Simulation Analysis of Absolute Lymphocyte Counts and Relapse Rate in Patients with Relapsing-Remitting Multiple Sclerosis. Clin Pharmacokinet. 2019 Mar;58(3):325-333. doi: 10.1007/s40262-018-0693-y.

Reference Type DERIVED
PMID: 29992396 (View on PubMed)

Afolabi D, Albor C, Zalewski L, Altmann DR, Baker D, Schmierer K. Positive impact of cladribine on quality of life in people with relapsing multiple sclerosis. Mult Scler. 2018 Oct;24(11):1461-1468. doi: 10.1177/1352458517726380. Epub 2017 Aug 17.

Reference Type DERIVED
PMID: 28817997 (View on PubMed)

Savic RM, Novakovic AM, Ekblom M, Munafo A, Karlsson MO. Population Pharmacokinetics of Cladribine in Patients with Multiple Sclerosis. Clin Pharmacokinet. 2017 Oct;56(10):1245-1253. doi: 10.1007/s40262-017-0516-6.

Reference Type DERIVED
PMID: 28255849 (View on PubMed)

De Stefano N, Giorgio A, Battaglini M, De Leucio A, Hicking C, Dangond F, Giovannoni G, Sormani MP. Reduced brain atrophy rates are associated with lower risk of disability progression in patients with relapsing multiple sclerosis treated with cladribine tablets. Mult Scler. 2018 Feb;24(2):222-226. doi: 10.1177/1352458517690269. Epub 2017 Jan 31.

Reference Type DERIVED
PMID: 28140753 (View on PubMed)

Ali S, Paracha N, Cook S, Giovannoni G, Comi G, Rammohan K, Rieckmann P, Sorensen PS, Vermersch P, Greenberg S, Scott DA, Joyeux A; CLARITY (CLAdRIbine Tablets treating multiple sclerosis orallY) Study Group. Reduction in healthcare and societal resource utilization associated with cladribine tablets in patients with relapsing-remitting multiple sclerosis: analysis of economic data from the CLARITY Study. Clin Drug Investig. 2012 Jan 1;32(1):15-27. doi: 10.2165/11593310-000000000-00000.

Reference Type DERIVED
PMID: 22017519 (View on PubMed)

Other Identifiers

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25643

Identifier Type: -

Identifier Source: org_study_id

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