Vinblastine and Methotrexate in Children With Pulmonary Vein Stenosis

NCT00215046 | Completed Phase 2 DMC

• 1 other identifier: CH 02-04-054 R
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 2

Brief Summary

To evaluate the efficacy of the chemotherapeutic agents vinblastine and methotrexate in the treatment of two groups of children with multivessel pulmonary vein stenosis. Group 1 will contain children with multivessel pulmonary vein stenosis who do not have structural heart disease, and Group 2 will consist of children with multivessel pulmonary vein stenosis and concomitant structural heart disease. The primary outcome variable for efficacy is patient status one year after the start of treatment, where status is classified as either failure or success. Failure is defined as death or evidence of progressive obstruction at any time over the course of treatment as defined in the protocol. Success constitutes complete or partial response to treatment or stability of disease. Secondary outcome variables for efficacy are survival, time from diagnosis of pulmonary vein stenosis until failure, and change in patient classification on a scale measuring the severity of the obstructive disease. 1.2 To assess the safety of vinblastine and methotrexate in the treatment of multivessel pulmonary vein stenosis. The primary outcome variable for safety is any occurrence of toxicity related to the administration of the chemotherapeutic agents over the treatment period.

Conditions

Intraluminal Pulmonary Vein Stenosis

Keywords

vinblastine; methotrexate

Outcome Measures

Primary Outcomes (1)

• The primary outcome variable for efficacy is patient status one year after the start of treatment, where status is classified as either failure or success. Failure is defined as death or evidence of progressive obstruction at any time over the course of

  Clinical status is assessed at one year for efficacy. Additionally, the toxicity are assessed at this time for the safety endpoint.

  1 year

Secondary Outcomes (1)

• Secondary outcome variables for efficacy are survival, time from diagnosis of pulmonary vein stenosis until failure, and change in patient classification on a scale measuring the severity of the obstructive disease.

  1 year

Study Arms (1)

Drug

EXPERIMENTAL

no randomization, all patients receive experimental drugs

Drug: Vinblastine; Drug: Methotrexate

Interventions

Vinblastine DRUG

drug - dosage and frequency is based on size of patient and response to treatment.

Also known as: antimicrotubule drug

Drug

Methotrexate DRUG

drug - dosage is based on size of patient and frequency and duration is based on response to treatment.

Also known as: Trexall

Drug

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis can be based on clinical and radiographic grounds or at the time of biopsy or prior surgical procedures. The diagnosis must be consistent with multivessel pulmonary stenosis.
• There must be evidence of severe pulmonary vein stenosis in at least two pulmonary veins.
• Evidence of myofibroblast neo-proliferation, if biopsies were obtained.
• Staging must include a complete cardiovascular evaluation including echocardiogram, and EKG.
• Accepted organ function includes:
• Creatinine \< 1.5 x normal for age.
• SGPT, Bilirubin \< 1.5 x normal for age.
• ANC ³ 1,500/mm3, Hemoglobin ³ 10g/dl, Platelets ³ 100,000/mm3
• Placement of a permanent central venous line. CVL access is necessary in all patients as vinblastine is a vesicant and will cause a tissue burn if infiltrated into the skin. Because a CVL may interfere with proper cardiac assessment in this patient population, the study physician will be made aware prior to the placement of a line.
• Patients may be listed for a lung transplant while enrolled on this study.
• All patients must have given written informed consent according to institutional guidelines.

Sponsors & Collaborators

• Boston Children's Hospital: lead
• Dana-Farber Cancer Institute: collaborator

Study Sites (2)

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Interventions

Vinblastine; Methotrexate

Intervention Hierarchy (Ancestors)

Vinca Alkaloids; Secologanin Tryptamine Alkaloids; Indole Alkaloids; Alkaloids; Heterocyclic Compounds; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Indolizidines; Indolizines; Aminopterin; Pterins; Pteridines

Study Officials

• Mark W Kieran, MD,PhD

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: September 15, 2005
• First Posted: September 22, 2005
• Study Start: March 1, 2000
• Primary Completion: October 1, 2006
• Study Completion: October 1, 2006
• Last Updated: June 27, 2011

Record last verified: 2011-06

Locations

US (2)