Interest of Mycophenolate for CIDP Weaning (MYCOPID)

• Sponsor: Assistance Publique - Hôpitaux de Paris
• Information provided by (Responsible Party): Assistance Publique - Hôpitaux de Paris

Study Description

Brief Summary:

The main objective is to study if the mycophenolate could decrease the proportion of patients who relapse during the IVIG tapering period and after the IVIG withdrawal.

Condition or disease	Intervention/treatment	Phase
Chronic Inflammatory Demyelinating Polyradiculopathy	Drug: Mycophenolate Mofetil; Drug: placebo	Phase 3

Detailed Description:

The secondary objectives are :

• Study if the mycophenolate could improve the proportion of withdrew patients.
• Study if the mycophenolate could improve the reduction of IVIG dose or could prolong the interval between two courses of IVIG compared to the baseline interval at month 12 and month 24 (= sparing treatment criteria).
• Study if mycophenolate could short the delay to perform the IVIG withdrawal.
• Study if mycophenolate could improve the clinical scores (ONLS, R-ODS MRC, INCAT sensory, 10 meters test) or pain score at month 12 and month 24.
• Study if mycophenolate could improve the quality of life at month12 and month 24.
• Identify clinical, biological and electrophysiological factors associated with withdrawal.
• To assess the pharmacokinetics factors (Area under the curve measuring the exposure to mycophenolate) and the pharmacogenetic factors (cytochrome and carrier, FcgammaR) associated with withdrawal.
• Evaluate the tolerance of Mycophenolate in this new indication.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 40 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Does the Mycophenolate Improve the Ability of Weaning Patients Off the Treatment in Chronic Inflammatory Demyelinating Polyradiculopathy (CIDP)
• Actual Study Start Date: November 18, 2013
• Actual Primary Completion Date: May 9, 2018
• Actual Study Completion Date: May 9, 2018

Arms and Interventions

Arm	Intervention/treatment
Experimental: mycophenolate mofetil	Drug: Mycophenolate Mofetil; 2g/day per os
Placebo Comparator: placebo; placebo pills	Drug: placebo

Outcome Measures

Primary Outcome Measures :

1. occurrence of a relapse during the tapering off period [ Time Frame: up to 18 months ]

   occurrence of a relapse during the tapering off period (up to 18 months after baseline) or after the withdrawal during the monitoring period.

   (the withdrawal is defined by the ability to reach the last day of IVIG treatment)

Secondary Outcome Measures :

1. Proportion of withdrew patients [ Time Frame: 6 months after the withdrawal ]
2. Proportion of withdrew patients at the end of the study [ Time Frame: 24 months ]
3. Sparing treatment (composite criteria) [ Time Frame: 24 months ]
   extension of the mean interval between IVIG courses at month 12 and month 24 compared to baseline, reduction of the total cumulative dose of IVIG at month 12 and month 24 in the mycophenolate group
4. Time to reach the withdrawal [ Time Frame: 24 months ]
5. EVA pain score [ Time Frame: 12 months ]
6. EVA pain score [ Time Frame: 24 months ]
7. ONLS scale [ Time Frame: 12 months ]
8. ONLS scale [ Time Frame: 24 months ]
9. R-ODS scale [ Time Frame: 12 months ]
10. R-ODS scale [ Time Frame: 24 months ]
11. MRC scale [ Time Frame: 12 months ]
12. MRC scale [ Time Frame: 24 months ]
13. INCAT sensory test [ Time Frame: 12 months ]
14. INCAT sensory test [ Time Frame: 24 months ]
15. 10 meters test [ Time Frame: 12 months ]
16. 10 meters test [ Time Frame: 24 months ]
17. SF-36 [ Time Frame: 12 months ]
    Quality of life scale
18. SF-36 [ Time Frame: 24 months ]
    Quality of life scale
19. Nottingham scale [ Time Frame: 12 months ]
    Quality of life scale
20. Nottingham scale [ Time Frame: 24 months ]
    Quality of life scale
21. global cost [ Time Frame: 24 months ]
    Comparison of the global cost in each group

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion criteria :

• Patient older than eighteen
• Written informed consent for study participation
• Definite or probable CIDP according to EFNS/PNS criteria or atypical CIDP (need to meet clinical EFNS/PNS criteria and at least 2 criteria among the EFNS/PNS supplementary criteria)
• Being responder (= decrease of at least 1 point on the ONLS score after IVIG) and dependent to IVIG (= increase of at least 1 point on the ONLS score after IVIG withdrawal or during the tapering period)
• Having received at least 3 courses of IVIG
• Negative pregnancy test for women of child-bearing age

Exclusion criteria :

• No social security benefit
• Pregnancy or intention to become pregnant
• Nursing mother
• Recent or active VIH or hepatitis B or C , or lyme infections
• Monoclonal IgM gammapathy with anti MAG antibodies or CANOMAD syndrome
• Neutropenia < 1G/L
• Malignancy during the 10 years before the inclusion
• Patients having received Mycophenolate
• History of allergy to mycophenolate or placebo excipient
• Patients having received immunosuppressive drugs during the 3 months period before the inclusion
• Patients receiving : plasma exchange, magnesium hydroxide, aluminium hydroxide, cholestyramine

Contacts and Locations

Locations

France

Neurology - pitié salpetrière hospital

Paris, France, 75013

Sponsors and Collaborators

Assistance Publique - Hôpitaux de Paris

Investigators

• Principal Investigator: Karine Viala, MD, PhD; Assistance Publique - Hôpitaux de Paris

More Information

• Responsible Party: Assistance Publique - Hôpitaux de Paris
• ClinicalTrials.gov Identifier: NCT02494505 History of Changes
• Other Study ID Numbers: P110148
• First Posted: July 10, 2015
• Last Update Posted: March 15, 2019
• Last Verified: March 2019

Keywords provided by Assistance Publique - Hôpitaux de Paris:

chronic inflammatory demyelinating polyradiculopathy; intravenous immunoglobulin (IVIG); withdrawal; quality of life; Clinical trial; cost

Additional relevant MeSH terms:

Polyradiculopathy; Polyradiculoneuropathy; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Mycophenolic Acid; Antibiotics, Antineoplastic; Antineoplastic Agents; Antibiotics, Antitubercular; Antitubercular Agents; Anti-Bacterial Agents; Anti-Infective Agents; Enzyme Inhibitors; Molecular Mechanisms of Pharmacological Action