Study of Forodesine Hydrochloride in Patients With Relapsed/Refractory Precursor T-Lymphoblastic Leukemia/Lymphoma Who Have Failed Two or More Prior Treatment Regimens

• ClinicalTrials.gov Identifier: NCT00419081
• Recruitment Status: Terminated
• First Posted: January 8, 2007
• Last Update Posted: January 23, 2012
• Sponsor: BioCryst Pharmaceuticals
• Information provided by (Responsible Party): BioCryst Pharmaceuticals

Study Description

Brief Summary:

The purpose of this study is to determine whether Forodesine Hydrochloride is effective in treating patients with relapsed/refractory precursor T-Lymphoblastic Leukemia/Lymphoma who have failed two or more prior treatment regimens.

Condition or disease	Intervention/treatment	Phase
Leukemia; Lymphoma	Drug: Forodesine Hydrochloride Sterile Solution, 5 mg/mL; Drug: Forodesine Hydrochloride Capsules (100 mg)	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Enrollment: 100 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase IIb, Multicenter, Open-Label, Nonrandomized, Repeat-Dose Study of Forodesine Hydrochloride in Patients With Relapsed/Refractory Precursor T-Lymphoblastic Leukemia/Lymphoma Who Have Failed Two or More Prior Treatment Regimens.
• Study Start Date: July 2006
• Actual Primary Completion Date: March 2007
• Actual Study Completion Date: March 2007

Arms and Interventions

Outcome Measures

Primary Outcome Measures :

1. To determine the rate of complete remission for T lymphoblastic leukemia/lymphoma relapsed or refractory patients. The complete remission rate will be evaluated over the three-month Initial Treatment Period.

Secondary Outcome Measures :

1. To determine the rate of CR achieved based on prior HSCT status
2. Assess safety and tolerability
3. Assess survival end points
4. Evaluate the maintenance and duration of response
5. Evaluate the proportion of patients able to proceed to HSCT
6. Evaluate the effects of this forodesine regimen on plasma levels of dGuo
7. Determine the effects of this forodesine regimen on clinical end points
8. Explore potential predictive biomarkers

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Patients with an unequivocal histologic diagnosis of precursor T-lymphoblastic leukemia/lymphoma (World Health Organization [WHO] classification).
• Failure to have responded to or relapsed after two or more treatment regimens for their disease, one of which could be HSCT.
• Performance status of 2 by Eastern Cooperative Oncology Group (ECOG) criteria (see Appendix A).
• Eighteen years of age and older.
• Life expectancy of at least three months.
• Adequate liver function (aspartate aminotransferase [AST] and/or alanine aminotransferase [ALT] ≤3 times upper limit of normal), unless related to the underlying leukemia.
• Negative serum or urine pregnancy test within two to seven days prior to the start of study treatment in females of childbearing potential.
• Females of childbearing potential and males must be willing and able to use an adequate method of contraception to avoid pregnancy for the duration of the study in such a manner that the risk of pregnancy is minimized. Acceptable contraceptives include intra-uterine devices (IUDs), hormonal contraceptives (oral, depot, patch, or injectable), and double-barrier methods such as condoms or diaphragms with spermicidal gel or foam.
• Signed informed consent form (ICF) prior to start of any study-specific procedures.
• Willing and able to provide authorization for the use and disclosure of personal health information in accordance with Health Insurance Portability and Accountability Act (HIPAA) policy (U.S. patients only).

Exclusion Criteria:

• Patients with known human immunodeficiency virus (HIV) infection or human T lymphotrophic virus 1 (HTLV-1).
• Patients with active hepatitis B or C infection.
• Patients with clinical evidence of active central nervous system (CNS) leukemia.
• Active serious infection not controlled by oral or intravenous antibiotics.
• Patients with a calculated creatinine clearance of <50 mL/min.
• Prior treatment with any investigational antileukemic or chemotherapy agent within seven days prior to study entry or lack of full recovery from side effects due to prior therapy, independent of when that therapy was given.
• Rapidly progressive disease with compromised organ function judged to be life threatening by the Investigator.
• Concurrent treatment with other antileukemia agents (CNS prophylaxis [e.g., intrathecal methotrexate, cytarabine, or hydrocortisone] and corticosteroid use will not be excluded, but must first be approved by the Medical Monitor) (see Sections 9.2.1 and 9.2.2).
• Pregnant and/or lactating female.
• Patients who cannot swallow or who have chronic gastrointestinal disease or conditions that may hamper compliance and/or absorption of the product.
• Hypersensitive or intolerant to any component of the study drug formulations.
• Patients who have received prior forodesine treatment.

Contacts and Locations

Locations

United States, Illinois

University of Chicago Medical Center

Chicago, Illinois, United States, 60637

United States, Louisiana

Louisana State University Health Sciences Center

Shreveport, Louisiana, United States, 71103

United States, New York

New York Medical College Division of Oncology

Valhalla, New York, United States, 10595

United States, South Carolina

Liberty Hematology and Oncology

Columbia, South Carolina, United States, 29203

United States, Texas

University of Texas MD Anderson Cancer Center

Houston, Texas, United States, 77030

Sponsors and Collaborators

BioCryst Pharmaceuticals

Investigators

• Principal Investigator: Auro del Giglio, MD; Faculdade de Medicina do ABC
• Principal Investigator: Belinda Pinto Simoes, MD; Hospital das Clinicas de Faculdade de Medicina de Ribeirao Preto
• Principal Investigator: Carlos Sergio Chiattone, MD; Irmandade da Santa Casa de Misercordia de Sao Paulo
• Principal Investigator: Carmino Antonio de Souza, MD; Hospital de Clinicias da UICAMP - Centro de Hematologia e Hemoterapia
• Principal Investigator: David Barqueti Jendiroba, MD; Centro Goiano de Oncologia
• Principal Investigator: Ernesto de Meis, MD; INCA - Instituto Nacional de Cancer
• Principal Investigator: Fernando Antonio Sellos Ribeiro, MD; Instituto Estadual de Hematologia Artur de Siqueira Cavalcanti - HEMORIO
• Principal Investigator: Ines Guterres, MD; Hospital Nossa Senhora de Conceicao
• Principal Investigator: Johnny Francisco Cordeiro Camargo, MD; Hospital Erasto Gaertner - Liga Paranaense de Combate ao Cancer - CEPEP
• Principal Investigator: Jose Salvador Rodrigues Oliveira, MD; Hospital Santa Marcelina
• Principal Investigator: Lucia Mariano da Rocha Silla, MD; Hospital de Clinicas de Porto Alegre
• Principal Investigator: Mair Pedro de Souza, MD; Fundacao Hospital Amaral Carvalho
• Principal Investigator: Maria Aparecida Zanichelli, MD; Hospital Brigadeiro
• Principal Investigator: Maria Lucia de Martino Lee, MD; Instituto de Oncologia Pediatrica - GRAACC Grupo de Apoio as Adolescente e a Crianca com Cancer - UNIFESP
• Principal Investigator: Ricardo Pasquini, MD; Hospital de Clinicas da Universidade Federal do Parana
• Principal Investigator: Nicholas Novitzky, MD; Division of Haematology
• Principal Investigator: Vernon Louw, MD; Free State University
• Principal Investigator: Farhad Ravandi, MD; M.D. Anderson Cancer Center
• Principal Investigator: Karen Seiter, MD; New York Medical College, Division of Oncology
• Principal Investigator: Francesco Turturro, MD; Louisana State University Health Sciences Center
• Principal Investigator: Luis Isola, MD; Icahn School of Medicine at Mount Sinai
• Principal Investigator: Wendy Stock, MD; University of Chicago Hospital
• Principal Investigator: Francisco Gonzalez, MD; Liberty Hematology and Oncology
• Principal Investigator: James Foran, MD; University of Alabama at Birmingham
• Principal Investigator: Ellen Ritchie, MD; Weill Medical College of Cornell University
• Principal Investigator: Leonard Heffner, MD; The Emory Clinic
• Principal Investigator: Madan Jagasia, MD; The Vanderbilt University Medical Center
• Principal Investigator: Katarzyna Jamieson, MD; University of Florida
• Principal Investigator: Gary Schiller, MD; University of California, Los Angeles
• Principal Investigator: Anjali Advani, MD; The Cleveland Clinic
• Principal Investigator: Kellie Sprague, MD; Tufts Medical Center
• Principal Investigator: Tanya Trippett, MD; Memorial Sloan Kettering Cancer Center
• Principal Investigator: Meir Wetzler, MD; Roswell Park Cancer Institute
• Principal Investigator: Alexander Gaiger, MD; Medical University Hospital
• Principal Investigator: Werner Linkersch, MD; Department of Heamtology
• Principal Investigator: David Nachbaur, MD; Department of Heamatology
• Principal Investigator: Agnes Buzin, MD
• Principal Investigator: Stephane de Botton, MD
• Principal Investigator: Jacques Delaunnay, MD
• Principal Investigator: Herve Dombret, MD; Service Clinique des Maladies du Sang
• Principal Investigator: Francoise Huguet-Rigal, MD; Service d'Hematologie
• Principal Investigator: Norbert Ifrah, MD
• Principal Investigator: Arnaud Pigneux, MD; CHU du Hart Leveque
• Principal Investigator: Bruno Quesnel, MD
• Principal Investigator: Houmedaly Reman, MD
• Principal Investigator: Xavier Thomas, MD; Maladies du Sang
• Principal Investigator: Norbert Vey, MD
• Principal Investigator: Francis Witz, MD
• Principal Investigator: Genadi Iosava, MD; Institute for Heamatology & Transfusiology
• Principal Investigator: Mamia Zodelava, MD; Heamatology and Transfusiology Dept
• Principal Investigator: Reinhard Andreesen, MD; Universitat Regensburg
• Principal Investigator: Walter Aulitzky, MD; Innere Abt. II
• Principal Investigator: Ulrich Duehrsen, MD
• Principal Investigator: Gerhard Ehninger, MD; Klinikum Carl Gustav Carus der Technischen Universitat
• Principal Investigator: Arnold Ganser, MD; ABT Hamatologie und Onkologie
• Principal Investigator: R. Haas, MD; Universitatsklinik Dusseldorf
• Principal Investigator: Dieter Hoelzer, MD; Leiterin der Studienzentrale
• Principal Investigator: W-D Ludwig, MD; Charite Universitatsmedizin Berlin
• Principal Investigator: Deiter Niederwieser, MD; Abt. Haematologie / Onkologie
• Principal Investigator: Michael Pfreunschuh, MD; Medizinische Klinik I
• Principal Investigator: Mathias Schmid, MD; Innere Medizin III, Hamatologie und Onkologie
• Principal Investigator: Norbert Schmitz, MD; Hamatologsiche Abteilung
• Principal Investigator: Peter Staib, MD; Med. Klinik I / Hamatolgie
• Principal Investigator: Matthias Stelljes, MD; Innere Medizin A
• Principal Investigator: Michele Baccarani, MD; Istitutp di Ematologia ed Oncologia Medica
• Principal Investigator: Renato Bassan, MD; Dipartimento di Oncoloia ed Ematico
• Principal Investigator: Alberto Bosi, MD; Aziendo Osperdaliero Universitaria Careggi Oncologia
• Principal Investigator: Felicetto Ferrara, MD; Azienda Ospedaliera Antonio Cardarelli
• Principal Investigator: Roberto Foa, MD; Cattedra di Ematologie
• Principal Investigator: Eugenio Gallo, MD; Divisione di Ematologia
• Principal Investigator: Vincenzo Liso, MD; UO Universita degi Studi Policinico Ematologia
• Principal Investigator: Salvatore Mirto, MD; Azienda Osperdaliera Vincenzo Cervello Divisione
• Principal Investigator: Enrica Morra, MD; Complessa di Ematologia and Dir Departmento
• Principal Investigator: Enrico Pogliani, MD; Ematologia
• Principal Investigator: Bob Lowenberg, MD; Eramus University Medical Centre
• Principal Investigator: Jerzy Holowiecki, MD; Klinika Hematologii I
• Principal Investigator: Wieslaw Jedrzejczak, MD; Klinika Hematologii i Onkologii
• Principal Investigator: Kazimierz Kuliczkowski, MD; Department of Heamatology
• Principal Investigator: Tadeusz Robak, MD; Klinika Hematologii Akademii Meduycznej Wojewodzki
• Principal Investigator: Aleksander Skotnicki, MD; Katedra i Klinika Hematologii Collegium Medicum
• Principal Investigator: K. Abdulkadyrov, MD; Head of Hematology Clinic
• Principal Investigator: Anatoli Golenkov, MD; Head of Clinical Heam and Immunotherapy Dept.
• Principal Investigator: S. Moiseev, MD; Faculty Therapy Dept
• Principal Investigator: Valerie Savchenko, MD; Dir. Research Institute for BMT and Molecular Hematology
• Principal Investigator: J. Ribera, MD; Insitut Català d'Oncologia-Hospital Universitari Germans Trias i Pujol

More Information

• Responsible Party: BioCryst Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT00419081
• Other Study ID Numbers: BCX1777-Tio-05-202
• First Posted: January 8, 2007
• Last Update Posted: January 23, 2012
• Last Verified: January 2012

Keywords provided by BioCryst Pharmaceuticals:

leukemia; lymphoma

Additional relevant MeSH terms:

Lymphoma; Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Lymphoid; Precursor T-Cell Lymphoblastic Leukemia-Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases