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Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria

This study has been completed.
Sponsor:
Collaborator:
Schering-Plough
Information provided by (Responsible Party):
Mark Kieran, Boston Children's Hospital
ClinicalTrials.gov Identifier:
NCT00425607
First received: January 22, 2007
Last updated: April 14, 2017
Last verified: April 2017
Results First Received: September 25, 2015  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Progeria
Hutchinson-Gilford Syndrome
Intervention: Drug: Lonafarnib

  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
26 patients with classic HGPS from 16 countries were enrolled from May through October 2007 at Boston Children's Hospital. 2 additional patients had nonclassic mutations and are not included in the analyses.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
One additional patient signed consent but withdrew from the protocol before receiving therapy.

Reporting Groups
  Description
Lonafarnib Lonafarnib (Merck & Co., Inc.) dosing was initiated at 115 mg/m2 and was increased to 150 mg/m2 after an adjustment period of at least 4 mo. Dosage was reduced back to 115 mg/m2 for patients experiencing drug-related grade 3 or 4 toxicity and also not responding to supportive care. Once dosage was reduced, patients were permitted to increase the dose of lonafarnib. Patients received oral lonafarnib either by capsule or liquid suspension dispersed in Ora-Blend SF or Ora-Plus (Paddock Laboratories, Inc.) every 12 ± 2 h for a period of 24–29 mo.

Participant Flow:   Overall Study
    Lonafarnib
STARTED   29 
Received Treatment   28 
COMPLETED   27 
NOT COMPLETED   2 
Death                1 
Withdrawal by Subject                1 



  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.
The analysis population represents the 25 patients with classic HGPS who received the farnesyltransferase inhibitor lonafarnib for a minimum of 2 y. The analysis population does not include 1 patient (with prior history of strokes) who died of a stroke after 5 mo on study, or 2 additional patients who had nonclassic mutations.

Reporting Groups
  Description
Lonafarnib Lonafarnib (Merck & Co., Inc.) dosing was initiated at 115 mg/m2 and was increased to 150 mg/m2 after an adjustment period of at least 4 mo. Dosage was reduced back to 115 mg/m2 fo patients experiencing drug-related grade 3 or 4 toxicity and also not responding to supportive care. Once dosage was reduced, patients were permitted to increase the dose of lonafarnib. Patients received oral lonafarnib either by capsule or liquid suspension dispersed in Ora-Blend SF or Ora-Plus (Paddock Laboratories, Inc.) every 12 ± 2 h for a period of 24–29 mo.

Baseline Measures
   Lonafarnib 
Overall Participants Analyzed 
[Units: Participants]
 25 
Age 
[Units: Years]
Mean (Standard Deviation)
 7.0  (3) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      14  56.0% 
Male      11  44.0% 


  Outcome Measures

1.  Primary:   Proportion of Participants With Successful Rate of Weight Gain   [ Time Frame: Assessed at weeks 16, 32, 52, 68, 84 and 104 ]


  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
This was an open label clinical trial on a very rare disease population. Patient numbers are small, though 26 participants represents a significant portion of the world's population of children with HGPS


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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:  
Name/Title: Dr. Mark Kieran
Organization: Dana-Farber Cancer Institute
phone: 617-632-4907
e-mail: mark_kieran@dfci.harvard.edu


Publications of Results:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Mark Kieran, Boston Children's Hospital
ClinicalTrials.gov Identifier: NCT00425607     History of Changes
Obsolete Identifiers: NCT00426088
Other Study ID Numbers: 07-01-007
P05009 ( Other Identifier: Schering-Plough )
Study First Received: January 22, 2007
Results First Received: September 25, 2015
Last Updated: April 14, 2017