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Trial record 96 of 399 for:    sodium phosphate

Clinical Trial of Vincristine vs. Prednisolone for Treatment of Complicated Hemangiomas

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ClinicalTrials.gov Identifier: NCT00555464
Recruitment Status : Terminated (The introduction of oral propranolol as a highly efficacious agent for infantile hemangiomas)
First Posted : November 8, 2007
Results First Posted : June 11, 2013
Last Update Posted : June 11, 2013
Sponsor:
Collaborator:
FDA Office of Orphan Products Development
Information provided by (Responsible Party):
Beth Drolet, Medical College of Wisconsin

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hemangioma
Interventions Drug: Vincristine
Drug: Prednisone
Enrollment 8
Recruitment Details Study participants were recruited from the Pediatric Dermatology clinic at Children's Hospital of Wisconsin between. Study enrollment period extended from 2008-2009.
Pre-assignment Details Subjects meeting eligibility criteria including diagnosis and lesional size criteria were enrolled in the study. Subjects who had received topical or intralesional corticosteroids were eligible following a 1 week wash-out period.
Arm/Group Title Vincristine Treatment Group Oral Steroid Treatment Group
Hide Arm/Group Description

Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas.

Vincristine : Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.

The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment.

Prednisone : Prednisolone given at 3 mg/kg/day by mouth for 12 week

Period Title: Overall Study
Started 4 4
Completed 4 1
Not Completed 0 3
Arm/Group Title Vincristine Treatment Group Oral Steroid Treatment Group Total
Hide Arm/Group Description

Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas.

Vincristine : Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.

The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment.

Prednisone : Prednisolone given at 3 mg/kg/day by mouth for 12 week

Total of all reporting groups
Overall Number of Baseline Participants 4 4 8
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 4 participants 8 participants
<=18 years
4
 100.0%
4
 100.0%
8
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 4 participants 8 participants
0.18  (0.05) 0.11  (0.05) 0.15  (0.06)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 4 participants 8 participants
Female
3
  75.0%
2
  50.0%
5
  62.5%
Male
1
  25.0%
2
  50.0%
3
  37.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 4 participants 4 participants 8 participants
4 4 8
1.Primary Outcome
Title Response of Hemangioma (IH) to Treatment
Hide Description

Response of IH not confined to the dermis will be coded using the following criteria: Progressive disease: >40% increase in volume by MRI, Partial response: >65% reduction in volume by MRI, Complete response: no visual or radiographic evidence of disease, Stable disease: none of the above or <40% increase or <65% decrease in volume by MRI.

Response of superficial IH will be coded using the following criteria (based on RECIST): Progressive disease: >30% increase in IH size, Partial response: >30% reduction in size, Complete response: no evidence of disease, Stable disease: none of the above.

Our first 3 patients showed limits to using MRI volume to measure IH size/response to therapy. Unlike other solid tumors, the superficial distribution of some IH made getting volume by MRI difficult, resulting in smaller tumor estimation compared to clinical assessment. Based on these observations, we amended the protocol to report response based on RECIST criteria instead of change in IH volume.

Time Frame 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Per protocol, all participants were analyzed, no matter the treatment arm or treatment success.
Arm/Group Title Vincristine Treatment Group Oral Steroid Treatment Group
Hide Arm/Group Description:

Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas.

Vincristine : Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.

The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment.

Prednisone : Prednisolone given at 3 mg/kg/day by mouth for 12 week

Overall Number of Participants Analyzed 4 3
Measure Type: Number
Unit of Measure: participants
Progressive Disease 0 1
Partial Response 2 0
Complete Response 0 0
Stable Disease 2 2
2.Secondary Outcome
Title Toxicity to Medications
Hide Description

Adverse events were closely monitored and recorded at weekly visits during treatment period and for two years after treatment ceased. Laboratory values were taken every other week during the treatment period.

Please see Adverse Events module for more details.

Time Frame Initial visit, 2, 4, 6, 10 and 12 weeks of therapy
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Vincristine Treatment Group Oral Steroid Treatment Group
Hide Arm/Group Description:

Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas.

Vincristine : Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.

The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment.

Prednisone : Prednisolone given at 3 mg/kg/day by mouth for 12 week

Overall Number of Participants Analyzed 4 4
Measure Type: Number
Unit of Measure: participants
Patients with Serious Adverse Events 0 0
Patients with Other Adverse Events 3 0
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Vincristine Treatment Group Oral Steroid Treatment Group
Hide Arm/Group Description

Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas.

Vincristine : Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.

The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment.

Prednisone : Prednisolone given at 3 mg/kg/day by mouth for 12 week

All-Cause Mortality
Vincristine Treatment Group Oral Steroid Treatment Group
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Vincristine Treatment Group Oral Steroid Treatment Group
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/4 (0.00%)      0/4 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
Vincristine Treatment Group Oral Steroid Treatment Group
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/4 (75.00%)      0/4 (0.00%)    
Blood and lymphatic system disorders     
Anemia  3/4 (75.00%)  5 0/4 (0.00%)  0
Gastrointestinal disorders     
Gastrointestinal: weight loss  1/4 (25.00%)  1 0/4 (0.00%)  0
Infections and infestations     
Infection with normal ANC  1/4 (25.00%)  1 0/4 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
obstruction/stenosis of airway  1/4 (25.00%)  1 0/4 (0.00%)  0
Skin and subcutaneous tissue disorders     
Rash/desquamation  1/4 (25.00%)  1 0/4 (0.00%)  0
1
Term from vocabulary, MedDRA (6.1)
The limitations of this trial include early termination of enrollment leading to small numbers of subjects analyzed.
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: Beth Drolet, MD
Organization: Medical College of Wisconsin
Phone: 414-955-2817
Publications:
1.Drolet BA, Esterly NB, Frieden IJ. Hemangiomas in Children. NEJM 1999;341:173-180. 2.Haggstrom AN, Drolet BA, Baselga E, Chamlin S, Esterly NB, Garzon M, Horii K, Lucky A, Metry DW, Mancini AJ, Nopper A, Frieden IJ. Prospective study of infantile hemangiomas, part II:clinical characteristics predicting complications and treatment. Pediatrics 2006;118: 882-887. 3.Haggstrom A, Drolet BA, Baselga E, Chamlin SL, Esterly NB, Garzon MC,. Prospective study of infantile hemangiomas, Part I: Demographic, prenatal and perinatal characteristics. J Pediatr 2007; 150(3):291-4. 4.Frieden IJ, Reese V, Cohen D. PHACE syndrome. The association of posterior fossa brain malformations, hemangiomas, arterial anomalies, coarctation of the aorta and cardiac defects, and eye abnormalities. Archives of Dermatology 1996 132(3):307-11.
Responsible Party: Beth Drolet, Medical College of Wisconsin
ClinicalTrials.gov Identifier: NCT00555464     History of Changes
Other Study ID Numbers: 3429
#FDA-R-003429-01
First Submitted: November 7, 2007
First Posted: November 8, 2007
Results First Submitted: February 19, 2013
Results First Posted: June 11, 2013
Last Update Posted: June 11, 2013