Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria (INHEPP)

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: NCT01550705
Recruitment Status : Terminated (Interim analysis demonstrated the treatment was not effective)
First Posted : March 12, 2012
Results First Posted : January 16, 2017
Last Update Posted : January 16, 2017
Sponsor:
Collaborators:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Icahn School of Medicine at Mount Sinai
University of Alabama at Birmingham
University of California, San Francisco
University of Texas
Information provided by (Responsible Party):
John Phillips, University of Utah

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Erythropoietic Protoporphyria (EPP)
X Linked Erythropoietic Protoporphyria
Intervention Drug: Isoniazid
Enrollment 11
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Isoniazid
Hide Arm/Group Description

Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check.

Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.

Period Title: Overall Study
Started 11
Completed 10
Not Completed 1
Arm/Group Title Isoniazid
Hide Arm/Group Description

Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check.

Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.

Overall Number of Baseline Participants 11
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
<=18 years
0
   0.0%
Between 18 and 65 years
11
 100.0%
>=65 years
0
   0.0%
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
Female
6
  54.5%
Male
5
  45.5%
Protoporphyria Type  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
Erythropoietic Protoporphyria (EPP)
9
  81.8%
X-linked Erythropoietic Protoporphyria (XLEPP)
2
  18.2%
Baseline Protoporphyrin IX level  
Mean (Standard Deviation)
Unit of measure:  Micrograms per deciliter (µg/dL)
Number Analyzed 11 participants
13.3  (14.4)
1.Primary Outcome
Title Change in Plasma Protoporphyrin IX Level
Hide Description Plasma Protoporphyrin IX will be measured at baseline and at 3 months
Time Frame Baseline and 3 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Isoniazid
Hide Arm/Group Description:

Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check.

Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.

Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: µg/dL
0.36  (9.1)
2.Secondary Outcome
Title Participants With Increased Sun Sensitivity
Hide Description Study participants were asked to report after 3 months if they had experienced an increase in subjective measures of sun sensitivity during the trial. Reported outcome is the number of study participants who reported increased sun sensitivity
Time Frame Baseline and 3 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Isoniazid
Hide Arm/Group Description:

Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check.

Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.

Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: participants
0
Time Frame 3 Months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Isoniazid
Hide Arm/Group Description

Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check.

Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.

All-Cause Mortality
Isoniazid
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Isoniazid
Affected / at Risk (%) # Events
Total   1/11 (9.09%)    
General disorders   
Flu-like Symptoms  1  1/11 (9.09%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE v4.03
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Isoniazid
Affected / at Risk (%) # Events
Total   5/11 (45.45%)    
Cardiac disorders   
Atrial Flutter  1  1/11 (9.09%)  1
Ear and labyrinth disorders   
Vertigo  1  1/11 (9.09%)  1
Gastrointestinal disorders   
Constipation  1  1/11 (9.09%)  1
Nervous system disorders   
Headache  1  1/11 (9.09%)  1
Renal and urinary disorders   
Urinary Tract Pain  1  1/11 (9.09%)  1
Reproductive system and breast disorders   
Dysmenorrhea  1  1/11 (9.09%)  1
Respiratory, thoracic and mediastinal disorders   
Productive Cough  1  1/11 (9.09%)  1
Nasal Congestion  1  1/11 (9.09%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE v4.03
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
Layout table for Results Point of Contact information
Name/Title: John D. Phillips, Ph.D.
Organization: University of Utah
Phone: 801-581-6650
EMail: john.phillips@hsc.utah.edu
Layout table for additonal information
Responsible Party: John Phillips, University of Utah
ClinicalTrials.gov Identifier: NCT01550705     History of Changes
Other Study ID Numbers: UTINH
U54DK083909 ( U.S. NIH Grant/Contract )
First Submitted: March 5, 2012
First Posted: March 12, 2012
Results First Submitted: November 19, 2016
Results First Posted: January 16, 2017
Last Update Posted: January 16, 2017