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

Sponsor

BioCryst Pharmaceuticals (Industry)

Overall Status

Terminated

CT.gov ID

NCT00419081

Collaborator

(none)

100

Enrollment

Locations

Duration (Months)

Patients Per Site

2.5

Patients Per Site Per Month

Study Details

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

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 :

Jul 1, 2006

Actual Primary Completion Date :

Mar 1, 2007

Actual Study Completion Date :

Mar 1, 2007

Outcome Measures

Primary Outcome Measures

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

To determine the rate of CR achieved based on prior HSCT status []
Assess safety and tolerability []
Assess survival end points []
Evaluate the maintenance and duration of response []
Evaluate the proportion of patients able to proceed to HSCT []
Evaluate the effects of this forodesine regimen on plasma levels of dGuo []
Determine the effects of this forodesine regimen on clinical end points []
Explore potential predictive biomarkers []

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

	Site	City	State	Country	Postal Code
1	University of Chicago Medical Center	Chicago	Illinois	United States	60637
2	Louisana State University Health Sciences Center	Shreveport	Louisiana	United States	71103
3	New York Medical College Division of Oncology	Valhalla	New York	United States	10595
4	Liberty Hematology and Oncology	Columbia	South Carolina	United States	29203
5	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