The Efficacy of Low Dose Naltrexone Therapy in Children With Crohn's Disease (LDN-Ped)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Jill P. Smith, Penn State University
ClinicalTrials.gov Identifier:
NCT00715117
First received: July 14, 2008
Last updated: May 29, 2013
Last verified: May 2013
Results First Received: August 4, 2011  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Crohn's Disease
Interventions: Drug: Naltrexone
Other: Placebo, sugar pill

  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
14 subjects were enrolled in this pilot trial and 2 were screen failures and not randomized or treated

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
The 2 subjects who were screen failures had PCDAI scores less than 30.

Reporting Groups
  Description
A: Placebo Then Naltrexone Subjects will receive placebo for for the first 8weeks then be crossed over to active drug naltrexone for the last 8 weeks
B: Naltrexone Then Naltrexone Naltrexone 0.1 mg/kg (not to exceed 4.5mg) once a day for 8 weeks followed by the same treatment for an additional 8 weeks

Participant Flow:   Overall Study
    A: Placebo Then Naltrexone     B: Naltrexone Then Naltrexone  
STARTED     6     6  
Completion fo 8 Week Study     6     6  
COMPLETED     6     5 [1]
NOT COMPLETED     0     1  
Crohn's flare and rescue with steroids                 0                 1  
[1] One subject flared and was rescued with steroids



  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.
No text entered.

Reporting Groups
  Description
A: Placebo Control Group Subjects will receive placebo for for the first 8weeks then be crossed over to active drug for the last 8 weeks
B: Naltrexone, Active Drug Group Naltrexone 0.1 mg/kg (not to exceed 4.5mg) once a day for 16 weeks
Total Total of all reporting groups

Baseline Measures
    A: Placebo Control Group     B: Naltrexone, Active Drug Group     Total  
Number of Participants  
[units: participants]
  6     6     12  
Age  
[units: participants]
     
<=18 years     6     6     12  
Between 18 and 65 years     0     0     0  
>=65 years     0     0     0  
Age  
[units: years]
Mean ± Standard Deviation
  12.2  ± 3.31     13  ± 3.22     12.5  ± 3.2  
Gender  
[units: participants]
     
Female     2     5     7  
Male     4     1     5  
Gender  
[units: participants]
     
Female     2     5     7  
Male     4     1     5  
Region of Enrollment  
[units: participants]
     
United States     6     6     12  
Pediatric Crohn's Disease Activity Index (PCDAI) score [1]
[units: score]
Mean ( Full Range )
  45  
  ( 35 to 52.5 )  
  38.3  
  ( 30 to 57 )  
  41.7  
  ( 30 to 57 )  
[1] The PCDAI score is a number unit that is calculated from symptoms scores by the subject over a 7-day period prior to the visit, laboratory values, height & weight, and physical exam findings. A score of 10 and under denotes "remission". Mild disease (score of 11-30); moderate disease (score of 31-45), a severe disease (scores greater than 45. A decline of 10 points or more is considered "response to therapy". The score can range from 0 to >60 Patient must have a PCDAI score of equal or greater than 30 to qualify for this study (i.e., moderate to severe disease).



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Number of Patients Reporting Side Effects   [ Time Frame: 8 weeks or 16 weeks ]

2.  Secondary:   Pediatric Crohn's Disease Activity Index Score (PCDAI)   [ Time Frame: Pretreatment and 8 weeks ]

3.  Secondary:   Change in Quality of Life Scores From Baseline to After 8 Weeks of Naltrexone Therapy   [ Time Frame: 16 weeks ]


  Serious Adverse Events


  Other Adverse Events


  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.


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
This was a pseudo-crossover trial where 6 subjects received placebo for 8 wks then were crossed over to active drug for 8 wks to increase the N treated with active drug. A smaller cohort of subjects on placebo were for safety & toxicity comparison.  


Results Point of Contact:  
Name/Title: Jill P Smith, MD Professor Emeritus of medicine
Organization: Pennsylvania State University
phone: 717-531-3694
e-mail: jsmith2@psu.edu


Publications of Results:
Other Publications:

Responsible Party: Jill P. Smith, Penn State University
ClinicalTrials.gov Identifier: NCT00715117     History of Changes
Other Study ID Numbers: PSU-IRB-27793
Study First Received: July 14, 2008
Results First Received: August 4, 2011
Last Updated: May 29, 2013
Health Authority: United States: Food and Drug Administration