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Lonafarnib for Chronic Hepatitis D

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01495585
Recruitment Status : Completed
First Posted : December 20, 2011
Results First Posted : August 31, 2016
Last Update Posted : August 31, 2016
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Care Provider);   Primary Purpose: Treatment
Condition Hepatitis D
Interventions Drug: Lonafarnib
Other: Placebo
Enrollment 14
Recruitment Details  
Pre-assignment Details In Group 1, participants were randomized either placebo or lonafarnib 100 mg. In Group2, participants were randomized into either placebo or lonafarnib 200 mg.
Arm/Group Title Placebo Lonafarnib 100 mg Lonafarnib 200 mg
Hide Arm/Group Description Two placebo participants in Group1 and two placebo participants in Group 2. The two placebo participants in Group 1 received open label lonafarnib 200 mg. 6 participants were randomized to Lonafarnib 100 mg. 4 participants were randomized to Lonafarnib 200 mg.
Period Title: Overall Study
Started 4 6 4
Open Label Lonafarnib 200 mg 2 0 0
Completed 4 6 4
Not Completed 0 0 0
Arm/Group Title Placebo Lonafarnib 100 mg Lonafarnib 200 mg Total
Hide Arm/Group Description placebo control. 6 participants were randomized to Lonafarnib 100 mg. 4 participants were randomized to Lonafarnib 200 mg and two "Placebo" participants in Lonafarnib 100 mg arm received open label lonafarnib 200 mg . Total of all reporting groups
Overall Number of Baseline Participants 4 6 6 16
Hide Baseline Analysis Population Description
Enrolled patients were sequentially assigned into one of two dosing groups which consisted of lonafarnib 100mg in group 1 and lonafarnib 200mg in group 2, with placebo controls in each group. Group1 placebo patients received open-label lonafarnib as group 2 participants.
Age, Continuous  
Median (Inter-Quartile Range)
Unit of measure:  Years
Number Analyzed 4 participants 6 participants 6 participants 16 participants
34
(33 to 39)
36
(30 to 42)
45
(38 to 55)
38
(33 to 48)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 6 participants 6 participants 16 participants
Female
2
  50.0%
1
  16.7%
2
  33.3%
5
  31.3%
Male
2
  50.0%
5
  83.3%
4
  66.7%
11
  68.8%
Body mass index  
Median (Inter-Quartile Range)
Unit of measure:  Kg/m^2
Number Analyzed 4 participants 6 participants 6 participants 16 participants
26.5
(24.1 to 28.9)
22.8
(21.9 to 23.5)
26.1
(25.5 to 27.3)
24.4
(21.9 to 27.3)
Pre-treatment mucleoside analogues  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 4 participants 6 participants 6 participants 16 participants
yes 1 2 2 5
no 3 4 4 11
Ethnic origin  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 4 participants 6 participants 6 participants 16 participants
Asian 2 3 3 8
White 1 3 3 7
African 1 0 0 1
1.Primary Outcome
Title Change in Quantitative Serum HDV RNA Levels After 28 Days of Lonafarnib Therapy.
Hide Description [Not Specified]
Time Frame 28 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Group 1 Group 2
Hide Arm/Group Description:

placebo control.

Group 1 placebo participants received open-label lonafarnib as group 2 participants.

Each group consisted of 8 participants (6 lonafarnib ands 2 placebo).

lonafarnib 100 mg
lonafarnib 200 mg
Overall Number of Participants Analyzed 4 6 6
Mean (Standard Deviation)
Unit of Measure: log(IU/ml)
-0.13  (0.14) -0.73  (0.54) -1.54  (0.36)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Group 1
Comments Student t-test was used on the change in serum log HDV RNA after 28 days of therapy with lonafarnib.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.03
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Group 2
Comments Student t-test was used on the change in serum log HDV RNA after 28 days of therapy with lonafarnib.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
2.Secondary Outcome
Title ALT Levels
Hide Description [Not Specified]
Time Frame 7 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Group 1 Group 2
Hide Arm/Group Description:

placebo control.

Group 1 placebo participants received open-label lonafarnib as group 2 participants.

Each group consisted of 8 participants (6 lonafarnib ands 2 placebo).

lonafarnib 100 mg
lonafarnib 200 mg
Overall Number of Participants Analyzed 4 6 6
Mean (Standard Deviation)
Unit of Measure: U/L
18  (13) 4  (93) 29  (85)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Group 1
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.96
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Group 2
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Time Frame 7 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Group 1 Group 2
Hide Arm/Group Description

placebo control.

Group 1 placebo participants received open-label lonafarnib as group 2 participants.

Each group consisted of 8 participants (6 lonafarnib ands 2 placebo).

lonafarnib 100 mg lonafarnib 200 mg
All-Cause Mortality
Placebo Group 1 Group 2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Hide Serious Adverse Events
Placebo Group 1 Group 2
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/4 (0.00%)      0/6 (0.00%)      0/6 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
Placebo Group 1 Group 2
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/4 (75.00%)      6/6 (100.00%)      6/6 (100.00%)    
Gastrointestinal disorders       
Nausea * 1  1/4 (25.00%)  1 1/6 (16.67%)  2 5/6 (83.33%)  6
Diarrhoea * 1  0/4 (0.00%)  0 3/6 (50.00%)  3 6/6 (100.00%)  6
Decreased appetite * 1  0/4 (0.00%)  0 1/6 (16.67%)  1 5/6 (83.33%)  5
Abdominal bloating/dyspepsia * 1  1/4 (25.00%)  1 1/6 (16.67%)  1 6/6 (100.00%)  6
Vomitting * 1  0/4 (0.00%)  0 0/6 (0.00%)  0 3/6 (50.00%)  3
General disorders       
Fatigue * 1  0/4 (0.00%)  0 1/6 (16.67%)  1 1/6 (16.67%)  1
Headache * 1  1/4 (25.00%)  1 1/6 (16.67%)  1 1/6 (16.67%)  1
Lightheadedness * 1  1/4 (25.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0
Metabolism and nutrition disorders       
Weight loss > 2kg * 1  0/4 (0.00%)  0 1/6 (16.67%)  1 6/6 (100.00%)  6
Reproductive system and breast disorders       
Testicular pain * 1  0/4 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Theo Heller
Organization: National Insitute of DIabetes and Digestive and Kidney Diseases
Phone: 301-402-7147
EMail: hellert@mail.nih.gov
Layout table for additonal information
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) )
ClinicalTrials.gov Identifier: NCT01495585    
Other Study ID Numbers: 120046
12-DK-0046 ( Other Identifier: NIH Clinical Center )
First Submitted: December 16, 2011
First Posted: December 20, 2011
Results First Submitted: April 28, 2016
Results First Posted: August 31, 2016
Last Update Posted: August 31, 2016