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PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma

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ClinicalTrials.gov Identifier: NCT00686842
Recruitment Status : Terminated (Drug supply unavailable.)
First Posted : May 30, 2008
Results First Posted : March 5, 2012
Last Update Posted : June 5, 2018
Sponsor:
Collaborators:
National Cancer Institute (NCI)
PTC Therapeutics
The EMMES Corporation
Information provided by (Responsible Party):
AIDS Malignancy Consortium

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (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
Enrollment 17
Recruitment Details  
Pre-assignment Details  
Arm/Group Title VEGF Inhibitor PTC299
Hide Arm/Group Description Single arm study - all subjects received PTC299
Period Title: Overall Study
Started 17
Completed 17
Not Completed 0
Arm/Group Title VEGF Inhibitor PTC299
Hide Arm/Group Description Single arm study - all subjects received PTC299
Overall Number of Baseline Participants 17
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
<=18 years
0
   0.0%
Between 18 and 65 years
17
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 17 participants
44.7  (6.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
Female
0
   0.0%
Male
17
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 17 participants
17
1.Primary Outcome
Title Safety and Toxicity of Anti-VEGF Small Molecule PTC299
Hide Description Patients who experienced an adverse event of grade 3 or greater
Time Frame All study visits
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title VEGF Inhibitor PTC299
Hide Arm/Group Description:
Single arm study - all subjects received PTC299
Overall Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: participants
7
2.Primary Outcome
Title Maximum Tolerated Dose
Hide Description [Not Specified]
Time Frame After each group of 3 subjects completes cycle 1 of treatment
Outcome Measure Data Not Reported
3.Primary Outcome
Title Response to Treatment
Hide Description [Not Specified]
Time Frame After each 28-day cycle of treatment and at discontinuation of therapy
Outcome Measure Data Not Reported
4.Secondary Outcome
Title Pharmacokinetics
Hide Description [Not Specified]
Time Frame Days 1, 15, 28, 57
Outcome Measure Data Not Reported
5.Secondary Outcome
Title Effects of Study Drug on Serum and Plasma VEGF, VEGFR, and Cytokine Profiles
Hide Description [Not Specified]
Time Frame On the first day of every 28-day cycle of treatment, Day 15, and treatment discontinuation
Outcome Measure Data Not Reported
6.Secondary Outcome
Title Effects of Study Drug on HIV and KSHV Viral Loads
Hide Description [Not Specified]
Time Frame Screening, end of cycle 1, end of every third cycle thereafter, and treatment discontinuation
Outcome Measure Data Not Reported
7.Secondary Outcome
Title Effects of Study Drug on T-lymphocyte Subsets (i.e., CD4 and CD8)
Hide Description [Not Specified]
Time Frame Screening, day 29, every 3 cycles thereafter, and at treatment discontinuation
Outcome Measure Data Not Reported
8.Secondary Outcome
Title 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
Hide Description [Not Specified]
Time Frame Screening and day 28
Outcome Measure Data Not Reported
9.Secondary Outcome
Title 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
Hide Description [Not Specified]
Time Frame Screening and day 28
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title VEGF Inhibitor PTC299
Hide Arm/Group Description Single arm study - all subjects received PTC299
All-Cause Mortality
VEGF Inhibitor PTC299
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
VEGF Inhibitor PTC299
Affected / at Risk (%) # Events
Total   3/17 (17.65%)    
General disorders   
Sudden death  1/17 (5.88%)  1
Musculoskeletal and connective tissue disorders   
Myalgia  1/17 (5.88%)  1
Renal and urinary disorders   
Renal and urinary disorder  1/17 (5.88%)  1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
VEGF Inhibitor PTC299
Affected / at Risk (%) # Events
Total   17/17 (100.00%)    
Blood and lymphatic system disorders   
Anemia  2/17 (11.76%)  2
Neutrophil count decreases  2/17 (11.76%)  2
Gastrointestinal disorders   
Abdominal pain  2/17 (11.76%)  2
Constipation  2/17 (11.76%)  2
Diarrhea  4/17 (23.53%)  5
Nausea  7/17 (41.18%)  10
Vomiting  3/17 (17.65%)  5
General disorders   
Acidosis  2/17 (11.76%)  2
Edema face  2/17 (11.76%)  2
Edema limbs  3/17 (17.65%)  3
Fatigue  5/17 (29.41%)  6
Immune system disorders   
Arthralgia  2/17 (11.76%)  2
Infections and infestations   
Cholesterol high  4/17 (23.53%)  6
Skin infection  3/17 (17.65%)  4
Investigations   
Blood bilirubin increased  3/17 (17.65%)  3
Creatinine increased  3/17 (17.65%)  3
Metabolism and nutrition disorders   
alanine aminotransferase increases  2/17 (11.76%)  4
Hyperglycemia  5/17 (29.41%)  8
Hypertriglyceridemia  5/17 (29.41%)  6
Hypoalbuminemia  3/17 (17.65%)  4
Hypocalcemia  2/17 (11.76%)  5
Hyponatremia  2/17 (11.76%)  2
Hypophosphatemia  2/17 (11.76%)  3
Musculoskeletal and connective tissue disorders   
Pain  5/17 (29.41%)  6
Nervous system disorders   
Headache  2/17 (11.76%)  2
Peripheral sensory neuropathy  2/17 (11.76%)  2
Renal and urinary disorders   
Proteinuria  3/17 (17.65%)  4
Respiratory, thoracic and mediastinal disorders   
Cough  2/17 (11.76%)  2
Dyspnea  2/17 (11.76%)  2
Skin and subcutaneous tissue disorders   
alopecia  2/17 (11.76%)  2
Skin and subcutaneous tissue disorder  2/17 (11.76%)  2
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: Susan Krown, MD, Protocol Chair
Organization: AMC
Phone: 301-251-1161
Responsible Party: AIDS Malignancy Consortium
ClinicalTrials.gov Identifier: NCT00686842     History of Changes
Other Study ID Numbers: AMC-059
U01CA121947 ( U.S. NIH Grant/Contract )
CDR0000596565 ( Other Identifier: NCI )
PTC299-ONC-005-KS ( Other Identifier: PTC Therapeutics )
First Submitted: May 29, 2008
First Posted: May 30, 2008
Results First Submitted: January 30, 2012
Results First Posted: March 5, 2012
Last Update Posted: June 5, 2018