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Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Raptor Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT01000961
First received: October 22, 2009
Last updated: November 18, 2014
Last verified: November 2014
Results First Received: November 2, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Cystinosis
Interventions: Drug: Cystagon® (Cysteamine Bitartrate)
Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)

  Participant Flow
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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
Participants randomized to each per sequence Arm are expected to remain in the same Arm throughout all intervention periods.

Reporting Groups
  Description
Cystagon First, Then Cystagon, Then RP103 Cystagon® dose in 50 mg and 150 mg capsule formulations administered every 6 hours in first intervention (after Run-in period) and RP103 dose in 25 mg and 75 mg capsule formulations administered every 12 hours in second intervention period.
Cystagon First, Then RP103, Then Cystagon RP103 dose in 25 mg and 75 mg capsule formulations administered every 12 hours in first intervention (after Run-in period) and Cystagon® dose in 50 mg and 150 mg capsule formulations administered every 6 hours in second intervention period.

Participant Flow for 3 periods

Period 1:   Run-in Period of 2-3 Weeks on Cystagon
    Cystagon First, Then Cystagon, Then RP103     Cystagon First, Then RP103, Then Cystagon  
STARTED     21 [1]   22 [1]
COMPLETED     21     22  
NOT COMPLETED     0     0  
[1] Part of the Safety analysis set

Period 2:   First Intervention (3 Weeks)
    Cystagon First, Then Cystagon, Then RP103     Cystagon First, Then RP103, Then Cystagon  
STARTED     21     22  
COMPLETED     21     20  
NOT COMPLETED     0     2  
Withdrawal by Subject                 0                 1  
Infection after pre-planned surgery                 0                 1  

Period 3:   Second Intervention (3 Weeks)
    Cystagon First, Then Cystagon, Then RP103     Cystagon First, Then RP103, Then Cystagon  
STARTED     21     20  
COMPLETED     21 [1]   20 [1]
NOT COMPLETED     0     0  
[1] Part of Efficacy analysis set



  Baseline Characteristics
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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.
There were 39 participants analyzed for Cystagon®; and there were 37 participants analyzed for RP103.

Reporting Groups
  Description
RP103 and Cystagon® Crossover Per Protocol Population

Baseline Measures
    RP103 and Cystagon® Crossover  
Number of Participants  
[units: participants]
  39  
Age  
[units: participants]
 
<=18 years     36  
Between 18 and 65 years     3  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  11.9  ± 4.33  
Gender  
[units: participants]
 
Female     16  
Male     23  
Region of Enrollment  
[units: participants]
 
France     13  
United States     22  
Netherlands     4  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon®   [ Time Frame: 4 weeks after the last subject has completed the study ]

2.  Secondary:   Comparison of Cysteamine PK Profiles, Steady State Cmax, Between RP103 and Cystagon®.   [ Time Frame: 4 weeks after the last subject has completed the study ]

3.  Secondary:   Comparison of Cysteamine PK Profiles, Steady State Tmax, Between RP103 and Cystagon®.   [ Time Frame: 4 weeks after the last subject has completed the study ]

4.  Secondary:   Comparison of Cysteamine PK Profiles, AUC(0-t), Between RP103 and Cystagon®.   [ Time Frame: 6 hours post dosing for Cystagon®; 12 hours post dosing for RP103. ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
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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: Mary Jo Bagger
Organization: Raptor Pharmaceuticals Inc.
phone: 415-408-6200 ext 6210
e-mail: mbagger@raptorpharma.com


Publications:

Responsible Party: Raptor Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT01000961     History of Changes
Other Study ID Numbers: RP103-03
Study First Received: October 22, 2009
Results First Received: November 2, 2012
Last Updated: November 18, 2014
Health Authority: United States: Food and Drug Administration
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Netherlands: Ministry of Health, Welfare and Sport