PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma

This study has been terminated.
(Drug supply unavailable.)
Sponsor:
Collaborators:
PTC Therapeutics
Information provided by (Responsible Party):
AIDS Malignancy Clinical Trials Consortium
ClinicalTrials.gov Identifier:
NCT00686842
First received: May 29, 2008
Last updated: June 19, 2012
Last verified: June 2012
Results First Received: January 30, 2012  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Kaposi's Sarcoma
Interventions: Drug: VEGF inhibitor PTC299
Genetic: gene expression analysis
Genetic: polymerase chain reaction
Genetic: protein expression analysis
Other: immunohistochemistry staining method
Other: laboratory biomarker analysis
Other: pharmacological study
Procedure: biopsy

  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
No text entered.

Reporting Groups
  Description
VEGF Inhibitor PTC299 Single arm study - all subjects received PTC299

Participant Flow:   Overall Study
    VEGF Inhibitor PTC299  
STARTED     17  
COMPLETED     17  
NOT COMPLETED     0  



  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
VEGF Inhibitor PTC299 Single arm study - all subjects received PTC299

Baseline Measures
    VEGF Inhibitor PTC299  
Number of Participants  
[units: participants]
  17  
Age  
[units: participants]
 
<=18 years     0  
Between 18 and 65 years     17  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  44.7  ± 6.5  
Gender  
[units: participants]
 
Female     0  
Male     17  
Region of Enrollment  
[units: participants]
 
United States     17  



  Outcome Measures

1.  Primary:   Safety and Toxicity of Anti-VEGF Small Molecule PTC299   [ Time Frame: All study visits ]

2.  Primary:   Maximum Tolerated Dose   [ Time Frame: After each group of 3 subjects completes cycle 1 of treatment ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   Yes

3.  Primary:   Response to Treatment   [ Time Frame: After each 28-day cycle of treatment and at discontinuation of therapy ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

4.  Secondary:   Pharmacokinetics   [ Time Frame: Days 1, 15, 28, 57 ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

5.  Secondary:   Effects of Study Drug on Serum and Plasma VEGF, VEGFR, and Cytokine Profiles   [ Time Frame: On the first day of every 28-day cycle of treatment, Day 15, and treatment discontinuation ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

6.  Secondary:   Effects of Study Drug on HIV and KSHV Viral Loads   [ Time Frame: Screening, end of cycle 1, end of every third cycle thereafter, and treatment discontinuation ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

7.  Secondary:   Effects of Study Drug on T-lymphocyte Subsets (i.e., CD4 and CD8)   [ Time Frame: Screening, day 29, every 3 cycles thereafter, and at treatment discontinuation ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

8.  Secondary:   Effects of Study Drug on VEGF, VEGFR-2 and -3, Phospho-Akt, p53, and HIF-1α Expression and Tumor Cell Proliferation, as Measured by Ki-67 Staining, in Tumor Biopsy Samples   [ Time Frame: Screening and day 28 ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

9.  Secondary:   Effects of Study Drug on Viral Gene Expression and Cellular Gene Transcription, as Measured by Real-time Quantitative PCR-based Profiling, in Tumor Biopsy Samples   [ Time Frame: Screening and day 28 ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events
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Time Frame No text entered.
Additional Description No text entered.

Frequency Threshold
Threshold above which other adverse events are reported   5%  

Reporting Groups
  Description
VEGF Inhibitor PTC299 Single arm study - all subjects received PTC299

Other Adverse Events
    VEGF Inhibitor PTC299  
Total, other (not including serious) adverse events    
# participants affected / at risk     17/17  
Blood and lymphatic system disorders    
Anemia    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Neutrophil count decreases    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Gastrointestinal disorders    
Abdominal pain    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Constipation    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Diarrhea    
# participants affected / at risk     4/17 (23.53%)  
# events     5  
Nausea    
# participants affected / at risk     7/17 (41.18%)  
# events     10  
Vomiting    
# participants affected / at risk     3/17 (17.65%)  
# events     5  
General disorders    
Acidosis    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Edema face    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Edema limbs    
# participants affected / at risk     3/17 (17.65%)  
# events     3  
Fatigue    
# participants affected / at risk     5/17 (29.41%)  
# events     6  
Immune system disorders    
Arthralgia    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Infections and infestations    
Cholesterol high    
# participants affected / at risk     4/17 (23.53%)  
# events     6  
Skin infection    
# participants affected / at risk     3/17 (17.65%)  
# events     4  
Investigations    
Blood bilirubin increased    
# participants affected / at risk     3/17 (17.65%)  
# events     3  
Creatinine increased    
# participants affected / at risk     3/17 (17.65%)  
# events     3  
Metabolism and nutrition disorders    
alanine aminotransferase increases    
# participants affected / at risk     2/17 (11.76%)  
# events     4  
Hyperglycemia    
# participants affected / at risk     5/17 (29.41%)  
# events     8  
Hypertriglyceridemia    
# participants affected / at risk     5/17 (29.41%)  
# events     6  
Hypoalbuminemia    
# participants affected / at risk     3/17 (17.65%)  
# events     4  
Hypocalcemia    
# participants affected / at risk     2/17 (11.76%)  
# events     5  
Hyponatremia    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Hypophosphatemia    
# participants affected / at risk     2/17 (11.76%)  
# events     3  
Musculoskeletal and connective tissue disorders    
Pain    
# participants affected / at risk     5/17 (29.41%)  
# events     6  
Nervous system disorders    
Headache    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Peripheral sensory neuropathy    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Renal and urinary disorders    
Proteinuria    
# participants affected / at risk     3/17 (17.65%)  
# events     4  
Respiratory, thoracic and mediastinal disorders    
Cough    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Dyspnea    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Skin and subcutaneous tissue disorders    
alopecia    
# participants affected / at risk     2/17 (11.76%)  
# events     2  
Skin and subcutaneous tissue disorder    
# participants affected / at risk     2/17 (11.76%)  
# events     2  



  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.


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
No text entered.  


Results Point of Contact:  
Name/Title: Susan Krown, MD, Protocol Chair
Organization: AMC
phone: 301-251-1161
e-mail: amcpm@emmes.com


No publications provided


Responsible Party: AIDS Malignancy Clinical Trials Consortium
ClinicalTrials.gov Identifier: NCT00686842     History of Changes
Other Study ID Numbers: CDR0000596565, U01CA121947, AMC-059, PTC299-ONC-005-KS
Study First Received: May 29, 2008
Results First Received: January 30, 2012
Last Updated: June 19, 2012
Health Authority: United States: Federal Government
United States: Food and Drug Administration
United States: Institutional Review Board