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
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Sponsor:
Horizon Pharma USA, Inc.
Information provided by (Responsible Party):
Horizon Pharma USA, Inc.
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Study Type | Interventional |
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Study Design | Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Condition |
Mitochondrial Diseases |
Intervention |
Drug: Cysteamine Bitartrate |
Enrollment | 22 |
Participant Flow
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 |
Baseline Characteristics
Outcome Measures
Adverse Events
Limitations and Caveats
The study was closed prematurely due to lack of efficacy demonstrated in base study RP103-MITO-001 and no efficacy analyses were conducted.
More Information
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
Name/Title: | Evelyn Olson, Director |
Organization: | Horizon Pharma USA, Inc. |
Phone: | 224- 383-3000 |
EMail: | clinicaltrials@horizonpharma.com |
Publications:
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 |