ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT01997229
Previous Study | Return to List | Next Study

Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01997229
Recruitment Status : Completed
First Posted : November 28, 2013
Results First Posted : February 7, 2018
Last Update Posted : February 7, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Refractory Generalized Myasthenia Gravis
Interventions: Biological: Eculizumab
Drug: Placebo

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Eculizumab

Induction phase: 3 vials of study drug (equivalent to 900 mg of eculizumab) weekly for 4 doses (every 7 days ± 2 days) followed by 4 vials of study drug (equivalent to 1200 mg of eculizumab) 1 week later for the fifth dose (Week 4).

Maintenance phase: 4 vials of study drug (equivalent to 1200 mg of eculizumab) every 2 weeks (14 days ± 2 days) from the fifth dose onwards (Week 6 through Week 26).

Placebo

Induction phase: 3 vials of study drug (placebo) weekly for 4 doses (every 7 days ± 2 days) followed by 4 vials of study drug (placebo) 1 week later for the fifth dose (Week 4).

Maintenance phase: 4 vials of study drug (placebo) every 2 weeks (14 days ± 2 days) from the fifth dose onwards (Week 6 through Week 26).


Participant Flow:   Overall Study
    Eculizumab   Placebo
STARTED   62   63 
COMPLETED   57   61 
NOT COMPLETED   5   2 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Eculizumab

Induction phase: 3 vials of study drug (equivalent to 900 mg of eculizumab) weekly for 4 doses (every 7 days ± 2 days) followed by 4 vials of study drug (equivalent to 1200 mg of eculizumab) 1 week later for the fifth dose (Week 4).

Maintenance phase: 4 vials of study drug (equivalent to 1200 mg of eculizumab) every 2 weeks (14 days ± 2 days) from the fifth dose onwards (Week 6 through Week 26).

Placebo

Induction phase: 3 vials of study drug (placebo) weekly for 4 doses (every 7 days ± 2 days) followed by 4 vials of study drug (placebo) 1 week later for the fifth dose (Week 4).

Maintenance phase: 4 vials of study drug (placebo) every 2 weeks (14 days ± 2 days) from the fifth dose onwards (Week 6 through Week 26).

Total Total of all reporting groups

Baseline Measures
   Eculizumab   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 62   63   125 
Age 
[Units: Years]
Mean (Standard Deviation)
 47.5  (15.66)   46.9  (17.98)   47.2  (16.80) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      41  66.1%      41  65.1%      82  65.6% 
Male      21  33.9%      22  34.9%      43  34.4% 


  Outcome Measures

1.  Primary:   Myasthenia Gravis Activities of Daily Living Profile (MG-ADL): Change From Baseline in MG-ADL Total Score at Week 26 by Worst-Rank Analysis of Covariance (ANCOVA)   [ Time Frame: End of study (Week 26) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  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 agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Jing-Jing Wang, MD
Organization: Alexion Pharmaceuticals, Inc.
phone: +1 (475) 230-2596
e-mail: JingJing.Wang@alexion.com


Publications of Results:
Other Publications:

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01997229     History of Changes
Other Study ID Numbers: ECU-MG-301
2013-003589-15 ( EudraCT Number )
First Submitted: November 18, 2013
First Posted: November 28, 2013
Results First Submitted: November 21, 2017
Results First Posted: February 7, 2018
Last Update Posted: February 7, 2018