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Rimeporide in Patients With Duchenne Muscular Dystrophy (RIM4DMD)

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: NCT02710591
Recruitment Status : Completed
First Posted : March 17, 2016
Results First Posted : July 18, 2019
Last Update Posted : July 18, 2019
Sponsor:
Information provided by (Responsible Party):
EspeRare Foundation

Study Type Interventional
Study Design Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Muscular Dystrophy, Duchenne
Intervention Drug: Rimeporide
Enrollment 20
Recruitment Details The recruitment period varied depending on the recruitment speed ; started in March 2016 to November 2017. it was competitive among the 4 sites: France, Spain, Italy and UK. A time interval of at least 1 week was maintained between adminstration of first dose in the first 3 patients of each cohort. It was extended to all patients for cohort 4.
Pre-assignment Details

Screening details:

Screening was carried out within 4 week prior to first administration of Rimeporide (SD1) to enable confirmation of patient eligibility and following the signature of the Informed Consent Form.

Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description 5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 50 mg TID; patients with a body weight more than 30kg at baseline were administered 75 mg TID. Each patient received Rimeporide during 4 weeks. 5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 100 mg TID; patients with a body weight more than 30kg at baseline were administered 150 mg TID. Each patient received Rimeporide during 4 weeks. 5 patients in total: patients with a with a body weight less than or equal to 30kg at baseline were administered 150 mg TID; patients with a body weight more than 30kg at baseline were administered 200 mg TID. Each patient received rimeporide during 4 weeks. 5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 200 mg TID; patients with a body weight more than 30kg at baseline were administered 300 mg TID. Each patient received rimeporide during 4 weeks
Period Title: Overall Study
Started 5 5 5 5
Completed 5 5 5 5
Not Completed 0 0 0 0
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4 Total
Hide Arm/Group Description 5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 50 mg TID; patients with a body weight more than 30kg at baseline were administered 75 mg TID. Each patient received Rimeporide during 4 weeks. 5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 100 mg TID; patients with a body weight more than 30kg at baseline were administered 150 mg TID. Each patient received Rimeporide during 4 weeks. 5 patients in total: patients with a with a body weight less than or equal to 30kg at baseline were administered 150 mg TID; patients with a body weight more than 30kg at baseline were administered 200 mg TID. Each patient received rimeporide during 4 weeks. 5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 200 mg TID; patients with a body weight more than 30kg at baseline were administered 300 mg TID. Each patient received rimeporide during 4 weeks Total of all reporting groups
Overall Number of Baseline Participants 5 5 5 5 20
Hide Baseline Analysis Population Description
Safety population (all patients receiving at least one dose of Rimeporide)
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 5 participants 5 participants 5 participants 20 participants
<=18 years
5
 100.0%
5
 100.0%
5
 100.0%
5
 100.0%
20
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 5 participants 5 participants 5 participants 5 participants 20 participants
8.4  (1.7) 8.2  (1.5) 8.8  (1.6) 9.2  (0.4) 8.7  (1.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 5 participants 5 participants 5 participants 20 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
5
 100.0%
5
 100.0%
5
 100.0%
5
 100.0%
20
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 5 participants 5 participants 5 participants 20 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
5
 100.0%
5
 100.0%
5
 100.0%
5
 100.0%
20
 100.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 5 participants 5 participants 5 participants 5 participants 20 participants
United Kingdom 0 1 3 1 5
Italy 0 1 2 3 6
France 5 0 0 1 6
Spain 0 3 0 0 3
1.Primary Outcome
Title Number of Participants With Adverse Events
Hide Description

Observations are given for the safety population (all patients who received at least one dose of study drug). Categorical data are presented with the number of subjects with at least one event for the following selections:

  • treatment-emergent AEs (TEAEs)
  • study drug-related TEAEs (ADRs)
  • serious TEAEs
  • study drug-related serious TEAEs (serious ADRs)
  • TEAEs leading to withdrawal
  • study drug-related TEAEs (ADRs) leading to withdrawal
  • serious TEAEs leading to withdrawal
  • TEAEs leading to death as outcome
Time Frame up to 6 weeks from first administration
Hide Outcome Measure Data
Hide Analysis Population Description
Observations are given for the safety population (all patients who received at least one dose of study drug).
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description:
5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 50 mg TID; patients with a body weight more than 30kg at baseline were administered 75 mg TID. Each patient received Rimeporide during 4 weeks.
5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 100 mg TID; patients with a body weight more than 30kg at baseline were administered 150 mg TID. Each patient received Rimeporide during 4 weeks.
5 patients in total: patients with a with a body weight less than or equal to 30kg at baseline were administered 150 mg TID; patients with a body weight more than 30kg at baseline were administered 200 mg TID. Each patient received rimeporide during 4 weeks.
5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 200 mg TID; patients with a body weight more than 30kg at baseline were administered 300 mg TID. Each patient received rimeporide during 4 weeks
Overall Number of Participants Analyzed 5 5 5 5
Measure Type: Count of Participants
Unit of Measure: Participants
Treatment emergent adverse events (TEAEs)
2
  40.0%
1
  20.0%
5
 100.0%
4
  80.0%
Treatment emergent adverse drug reactions (ADRs)
0
   0.0%
0
   0.0%
0
   0.0%
2
  40.0%
SAEs
0
   0.0%
0
   0.0%
1
  20.0%
0
   0.0%
Serious ADRs
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
TEAEs leading to withdrawal
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
TEAEs leading to death
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
2.Other Pre-specified Outcome
Title PK Profile of Rimeporide - Cmax
Hide Description

PK samples were collected according to the following schedule:

  • At Day 1: for half of the patients: just before first administration, and one sample in each of the following time frames after the first dose:

    • 0.5 to 1h after dosing,
    • 1 to 2h after dosing,
    • 2.5 to 3.5h after dosing,
    • 6h after dosing
  • At Day 1: for the other half of the patients: just before first administration, and one sample in each of the following time frames after the second dose:

    • 0.5 to 1h after dosing,
    • 1 to 2h after dosing,
    • 2.5 to 3.5h after dosing,
    • 6h after dosing

Finally, at week 4 (Day 28) after the last dose:

  • 0.5 to 1h after dosing,
  • 6h after dosing
Time Frame 4 week study treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description:
5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 50 mg TID; patients with a body weight more than 30kg at baseline were administered 75 mg TID. Each patient received Rimeporide during 4 weeks.
5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 100 mg TID; patients with a body weight more than 30kg at baseline were administered 150 mg TID. Each patient received Rimeporide during 4 weeks.
5 patients in total: patients with a with a body weight less than or equal to 30kg at baseline were administered 150 mg TID; patients with a body weight more than 30kg at baseline were administered 200 mg TID. Each patient received rimeporide during 4 weeks.
5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 200 mg TID; patients with a body weight more than 30kg at baseline were administered 300 mg TID. Each patient received rimeporide during 4 weeks
Overall Number of Participants Analyzed 5 5 5 5
Mean (Standard Deviation)
Unit of Measure: ng/mL
Cmax at Day 1 1286  (568) 1987  (650) 3013  (730) 3819  (743)
Cmax at Day 28 1361  (431) 2077  (974) 2817  (675) 3909  (846)
Time Frame Treatment emergent adverse events were collected from first administration of the study drug up to 6 weeks.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Cohort 4
Hide Arm/Group Description 5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 50 mg TID; patients with a body weight more than 30kg at baseline were administered 75 mg TID. Each patient received Rimeporide during 4 weeks. 5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 100 mg TID; patients with a body weight more than 30kg at baseline were administered 150 mg TID. Each patient received Rimeporide during 4 weeks. 5 patients in total: patients with a with a body weight less than or equal to 30kg at baseline were administered 150 mg TID; patients with a body weight more than 30kg at baseline were administered 200 mg TID. Each patient received rimeporide during 4 weeks. 5 patients in total: patients with a body weight less than or equal to 30kg at baseline were administered 200 mg TID; patients with a body weight more than 30kg at baseline were administered 300 mg TID. Each patient received rimeporide during 4 weeks
All-Cause Mortality
Cohort 1 Cohort 2 Cohort 3 Cohort 4
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/5 (0.00%)      0/5 (0.00%)      0/5 (0.00%)      0/5 (0.00%)    
Hide Serious Adverse Events
Cohort 1 Cohort 2 Cohort 3 Cohort 4
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/5 (0.00%)      0/5 (0.00%)      1/5 (20.00%)      0/5 (0.00%)    
Gastrointestinal disorders         
Diarrhoea  1  0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0
Vomitting  1  0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0
1
Term from vocabulary, MedDRA (18.1)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cohort 1 Cohort 2 Cohort 3 Cohort 4
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/5 (40.00%)      1/5 (20.00%)      5/5 (100.00%)      4/5 (80.00%)    
Ear and labyrinth disorders         
Tympanic membrane perforation  1  0/5 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1
Gastrointestinal disorders         
Diarrhoea  1  0/5 (0.00%)  0 0/5 (0.00%)  0 2/5 (40.00%)  3 0/5 (0.00%)  0
Abdominal pain upper  1  0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  2 1/5 (20.00%)  1
Vomiting  1  0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 1/5 (20.00%)  1
Mouth ulceration  1  0/5 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0
General disorders         
Chills  1  1/5 (20.00%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0
Pyrexia  1  0/5 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1
Infections and infestations         
Nasopharyngitis  1  0/5 (0.00%)  0 1/5 (20.00%)  1 2/5 (40.00%)  2 1/5 (20.00%)  1
Injury, poisoning and procedural complications         
Fall  1  1/5 (20.00%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0
Musculoskeletal and connective tissue disorders         
Pain in extremity  1  1/5 (20.00%)  2 0/5 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0
Nervous system disorders         
Headache  1  1/5 (20.00%)  1 0/5 (0.00%)  0 2/5 (40.00%)  2 2/5 (40.00%)  3
Presyncope  1  0/5 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1
Dizziness  1  0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0
Respiratory, thoracic and mediastinal disorders         
Cough  1  0/5 (0.00%)  0 1/5 (20.00%)  1 1/5 (20.00%)  1 1/5 (20.00%)  1
Oropharyngeal pain  1  0/5 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1
Skin and subcutaneous tissue disorders         
Urticaria  1  0/5 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1
Vascular disorders         
Flushing  1  0/5 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 1/5 (20.00%)  2
1
Term from vocabulary, MedDRA (18.1)
Indicates events were collected by systematic assessment
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Florence Porte-Thomé
Organization: EspeRare Fondation
Phone: 22 794 4004 ext +41
EMail: porte.florence@esperare.org
Layout table for additonal information
Responsible Party: EspeRare Foundation
ClinicalTrials.gov Identifier: NCT02710591    
Other Study ID Numbers: EspeRare_RIM_001
2015-002530-50 ( EudraCT Number )
First Submitted: January 26, 2016
First Posted: March 17, 2016
Results First Submitted: December 21, 2018
Results First Posted: July 18, 2019
Last Update Posted: July 18, 2019