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A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

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ClinicalTrials.gov Identifier: NCT02473445
Recruitment Status : Terminated (Sponsor decision to end development of RP103 for mitochondrial disease due to lack of efficacy demonstrated in base study RP103-MITO-001.)
First Posted : June 16, 2015
Results First Posted : May 11, 2018
Last Update Posted : May 11, 2018
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma USA, Inc.

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Mitochondrial Diseases
Intervention Drug: Cysteamine Bitartrate
Enrollment 22
Recruitment Details Participants who completed study RP103-MITO-001 (NCT02023866) study were eligible for enrollment into this extension study. The study was conducted at 5 sites in the United States.
Pre-assignment Details  
Arm/Group Title Cysteamine Bitartrate Delayed-release
Hide Arm/Group Description Participants received cysteamine bitartrate delayed-release capsules (RP103) twice daily for up to 2 years. The starting dose was the same as the last dose received in study RP103-MITO-001, the maximum dose was 1.3 g/m²/day.
Period Title: Overall Study
Started 22
Completed 0
Not Completed 22
Reason Not Completed
Withdrawal by Subject             2
Sponsor Decision             20
Arm/Group Title Cysteamine Bitartrate Delayed-release
Hide Arm/Group Description Participants received cysteamine bitartrate delayed-release capsules (RP103) twice daily for up to 2 years. The starting dose was the same as the last dose received in study RP103-MITO-001, the maximum dose was 1.3 g/m²/day.
Overall Number of Baseline Participants 22
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 22 participants
10.8  (4.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
Female
10
  45.5%
Male
12
  54.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
22
 100.0%
Unknown or Not Reported
0
   0.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
   9.1%
Black of African American
0
   0.0%
Native Hawaiian or other Pacific Islander
0
   0.0%
White
18
  81.8%
Other
0
   0.0%
Multiple
2
   9.1%
1.Primary Outcome
Title Change in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Score
Hide Description

The NPMDS evaluates the progression of mitochondrial disease in pediatric patients in 4 domains:

I - Current Function (vision, hearing, communication, feeding, and mobility) with scores ranging from 0 to 21;

II - System Specific Involvement (seizures, encephalopathy, bleeding diathesis or coagulation defects, gastrointestinal, endocrine, respiratory, cardiovascular, renal, liver, and blood) with scores ranging from 0 to 30.

III - Current Clinical Assessment (growth and development over past 6 months, vision, strabismus and eye movement, myopathy, ataxia, pyramidal, extrapyramidal, and neuropathy) with scores ranging from 0 to 28;

IV - Quality of Life with scores ranging from 0 to 25. For sections I-III, higher scores reflect more severe disease. For Section IV, a higher score reflects a lower quality of life.

Time Frame Baseline, every 3 months and Study Exit (up to 24 Months)
Hide Outcome Measure Data
Hide Analysis Population Description
The study was closed prematurely due to lack of efficacy demonstrated in base study RP103-MITO-001. As a result, only a limited amount of data was collected for patients that were enrolled prior to termination. The decision was made that the data were not complete enough, and no analyses were conducted.
Arm/Group Title Cysteamine Bitartrate Delayed-release
Hide Arm/Group Description:
Participants received cysteamine bitartrate delayed-release capsules (RP103) twice daily for up to 2 years. The starting dose was the same as the last dose received in study RP103-MITO-001, the maximum dose was 1.3 g/m²/day.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Change Over Time in Two of the Most Pre-eminent Symptoms
Hide Description The two pre-eminent symptoms previously identified in study RP103-MITO-001 were to be continued to be assessed during the extension study. Symptoms included myopathy, dystonia, ataxia, retarded motor development, reduced activities of daily living, and vision.
Time Frame Baseline, every 3 months and Study Exit (up to 24 Months)
Hide Outcome Measure Data
Hide Analysis Population Description
The study was closed prematurely due to lack of efficacy demonstrated in base study RP103-MITO-001. As a result, only a limited amount of data was collected for patients that were enrolled prior to termination. The decision was made that the data were not complete enough, and no analyses were conducted.
Arm/Group Title Cysteamine Bitartrate Delayed-release
Hide Arm/Group Description:
Participants received cysteamine bitartrate delayed-release capsules (RP103) twice daily for up to 2 years. The starting dose was the same as the last dose received in study RP103-MITO-001, the maximum dose was 1.3 g/m²/day.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Change Over Time in Pharmacodynamic Biomarkers
Hide Description Change from baseline in glutathione, glutathione disulfide, and lactate analyses were not performed as the study was prematurely terminated.
Time Frame Baseline, every 3 months and Study Exit (up to 24 Months)
Hide Outcome Measure Data
Hide Analysis Population Description
The study was closed prematurely due to lack of efficacy demonstrated in base study RP103-MITO-001. As a result, only a limited amount of data was collected for patients that were enrolled prior to termination. The decision was made that the data were not complete enough, and no analyses were conducted.
Arm/Group Title Cysteamine Bitartrate Delayed-release
Hide Arm/Group Description:
Participants received cysteamine bitartrate delayed-release capsules (RP103) twice daily for up to 2 years. The starting dose was the same as the last dose received in study RP103-MITO-001, the maximum dose was 1.3 g/m²/day.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame From first dose of study drug until the last dose; median duration of treatment was 238 days.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cysteamine Bitartrate Delayed-release
Hide Arm/Group Description Participants received cysteamine bitartrate delayed-release capsules (RP103) twice daily for up to 2 years. The starting dose was the same as the last dose received in study RP103-MITO-001, the maximum dose was 1.3 g/m²/day.
All-Cause Mortality
Cysteamine Bitartrate Delayed-release
Affected / at Risk (%)
Total   0/22 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Cysteamine Bitartrate Delayed-release
Affected / at Risk (%)
Total   9/22 (40.91%) 
Eye disorders   
Blepharospasm  1  1/22 (4.55%) 
Gastrointestinal disorders   
Diarrhoea  1  1/22 (4.55%) 
Vomiting  1  2/22 (9.09%) 
Infections and infestations   
Clostridium difficile infection  1  1/22 (4.55%) 
Device related infection  1  1/22 (4.55%) 
Influenza  1  1/22 (4.55%) 
Parainfluenzae virus infection  1  1/22 (4.55%) 
Respiratory syncytial virus bronchiolitis  1  1/22 (4.55%) 
Rhinovirus infection  1  1/22 (4.55%) 
Urosepsis  1  1/22 (4.55%) 
Metabolism and nutrition disorders   
Dehydration  1  1/22 (4.55%) 
Musculoskeletal and connective tissue disorders   
Mobility decreased  1  1/22 (4.55%) 
Muscle twitching  1  1/22 (4.55%) 
Nervous system disorders   
Ataxia  1  1/22 (4.55%) 
Convulsion  1  2/22 (9.09%) 
Muscle spasticity  1  1/22 (4.55%) 
Status epilepticus  1  1/22 (4.55%) 
Psychiatric disorders   
Confusional state  1  1/22 (4.55%) 
Respiratory, thoracic and mediastinal disorders   
Acute respiratory failure  1  1/22 (4.55%) 
Cough  1  1/22 (4.55%) 
Hypercapnia  1  1/22 (4.55%) 
Hypoxia  1  1/22 (4.55%) 
Respiratory distress  1  1/22 (4.55%) 
Rhinorrhoea  1  1/22 (4.55%) 
1
Term from vocabulary, MedDRA 17.0
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cysteamine Bitartrate Delayed-release
Affected / at Risk (%)
Total   18/22 (81.82%) 
Gastrointestinal disorders   
Abdominal pain  1  2/22 (9.09%) 
Nausea  1  2/22 (9.09%) 
Vomiting  1  11/22 (50.00%) 
General disorders   
Asthenia  1  2/22 (9.09%) 
Pyrexia  1  5/22 (22.73%) 
Nervous system disorders   
Ataxia  1  2/22 (9.09%) 
Convulsion  1  2/22 (9.09%) 
Headache  1  2/22 (9.09%) 
Lethargy  1  2/22 (9.09%) 
Myoclonus  1  2/22 (9.09%) 
Respiratory, thoracic and mediastinal disorders   
Nasal congestion  1  2/22 (9.09%) 
1
Term from vocabulary, MedDRA 17.0
Indicates events were collected by systematic assessment
The study was closed prematurely due to lack of efficacy demonstrated in base study RP103-MITO-001 and no efficacy analyses were conducted.
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.
Horizon requests that any Investigator/institution that plans on presenting or publishing results provide written notification of their request a minimum of 60 days prior to presentation or publication. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsors’ Intellectual Property rights .
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Evelyn Olson, Director
Organization: Horizon Pharma USA, Inc.
Phone: 224- 383-3000
EMail: clinicaltrials@horizonpharma.com
Layout table for additonal information
Responsible Party: Horizon Pharma USA, Inc.
ClinicalTrials.gov Identifier: NCT02473445     History of Changes
Other Study ID Numbers: RP103-MITO-002
First Submitted: June 10, 2015
First Posted: June 16, 2015
Results First Submitted: March 6, 2018
Results First Posted: May 11, 2018
Last Update Posted: May 11, 2018