NCT00598624

Brief Summary

This is a multicentric, non-randomized, non-controlled open-label phase II trial to evaluate the safety and efficacy of treosulfan in a combination regimen with fludarabine as conditioning therapy prior to allogeneic stem cell transplantation (SCT) in patients with haematological malignancies. The aim is to demonstrate a clinical benefit compared with historical data on intravenous busulfan (BusulfexTM, BusilvexTM), the only drug so far registered in the indication conditioning before allogeneic stem cell transplantation.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
175

participants targeted

Target at P75+ for phase_2 leukemia

Timeline
Completed

Started Sep 2005

Geographic Reach
1 country

12 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2005

Completed
2.4 years until next milestone

First Submitted

Initial submission to the registry

January 10, 2008

Completed
12 days until next milestone

First Posted

Study publicly available on registry

January 22, 2008

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2009

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2010

Completed
Last Updated

August 11, 2009

Status Verified

August 1, 2009

Enrollment Period

4.3 years

First QC Date

January 10, 2008

Last Update Submit

August 10, 2009

Conditions

Outcome Measures

Primary Outcomes (2)

  • Efficacy: Evaluation of engraftment

    28 days

  • Safety: Evaluation of the incidence of CTC grade 3 and 4 adverse events

    between day -6 and day +28

Secondary Outcomes (8)

  • Efficacy: Evaluation of disease free survival (DFS)

    1 year

  • Efficacy: Evaluation of overall survival (OS)

    1 year

  • Efficacy: Evaluation of relapse incidence (RI)

    1 year

  • Efficacy: Documentation of donor chimerism

    on day +28, +56 and +100

  • Safety: Evaluation of incidence of non-relapse mortality (NRM)

    on day +28 and day +100

  • +3 more secondary outcomes

Study Arms (1)

A

EXPERIMENTAL
Drug: Treosulfan IV

Interventions

Treosulfan i.v.: 14 g/m²/d from day -6 to day -4

A

Eligibility Criteria

Age18 Years - 69 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with haematological malignancies, according to WHO classification, such as:
  • acute myeloid leukaemia -AML- in CR1 except "low-risk cases" defined by t(15;17), t(8;21), inv 16 or normal cytogenetics at diagnosis with FLT3-ITD negative and NPM-1 positive, with no high risk clinical criteria
  • any AML beyond CR1
  • acute lymphoblast leukaemia -ALL- in CR1 only if at "high risk" defined by cytogenetics as t(9;22), t(4;11) or for persistence of minimal residual disease (MRD)
  • any ALL beyond CR1
  • chronic myeloid leukaemia -CML- in chronic phase (CP) or accelerated phase (AP) intolerant/not responsive to TK-inhibitors
  • myeloproliferative disorders -MPD-
  • myelodysplastic syndrome -MDS- with intermediate or high risk International Prognostic Scoring System (IPSS)
  • diffuse large cell lymphoma -DLCL- with a chemosensitive relapse or beyond CR1
  • lymphoblastic and Burkitt lymphoma with a chemosensitive relapse or beyond CR1
  • mantle cell lymphoma -MCL- with a chemosensitive relapse or beyond CR1
  • follicular lymphoma -FCL- with a chemosensitive relapse or beyond CR2
  • Hodgkin lymphoma -HD- with a chemosensitive relapse or beyond CR1
  • chronic lymphocytic leukaemia -CLL- at "poor risk" in CR1 or with a chemosensitive relapse
  • CLL relapsing after high dose chemotherapy
  • +15 more criteria

You may not qualify if:

  • Secondary malignancies
  • Previous allogeneic transplantation
  • Hematopoietic cell transplantation-specific comorbidity index \> 4 (HCT-CI Sorror et al, Appendix M)
  • Known and manifested malignant involvement of the CNS
  • Active infectious disease
  • HIV- positivity or active hepatitis infection
  • Impaired liver function (Bilirubin \> upper normal limit; Transaminases \> 3.0 x upper normal limit)
  • Impaired renal function (Creatinine-clearance \< 60 ml/min; Serum Creatinine \> 1.5 x upper normal limit).
  • Pleural effusion or ascites \> 1.0 L
  • Pregnancy or lactation
  • Known hypersensitivity to treosulfan and/or fludarabine
  • Participation in another experimental drug trial within 4 weeks before day -6
  • Non-co-operative behaviour or non-compliance
  • Psychiatric diseases or conditions that might impair the ability to give informed consent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

Ematologia, Ospedale Casa Sollievo della Sofferenza

San Giovanni Rotondo, Foggia, Italy

RECRUITING

IRCCS San Raffaele; Unità Operativa di Ematologia

Milan, MI, 20100, Italy

RECRUITING

Dipartimento Biotecnologie Cellulari ed Ematologia; Azienda Policlinico Umberto I

Roma, Roma, 00100, Italy

RECRUITING

USC Ematologia, Ospedali Riuniti

Bergamo, Italy

RECRUITING

Ospedale centrale di Bolzano - Reparto di Ematologia

Bolzano, Italy

RECRUITING

PO "R.Binaghi" - CTMO

Cagliari, Italy

RECRUITING

AO "Santa Croce" e Carle - Reparto di Ematologia

Cuneo, Italy

RECRUITING

Istituto Europeo di Oncologia - Divisione di Ematologia

Milan, Italy

RECRUITING

Ospedale Civile - UTI ematologia per il trapianto emopoietico

Pescara, Italy

RECRUITING

Arcispedale Santa Maria Nuova - SC di Ematologia

Reggio Emilia, Italy

RECRUITING

AO San Camillo Forlanini - UOC ematologia e trapianto

Roma, Italy

RECRUITING

AOU Santa Maria della Misericordia - Clinica Ematologica

Udine, Italy

RECRUITING

Related Publications (1)

  • Lazzari L, Ruggeri A, Lupo Stanghellini MT, Mastaglio S, Messina C, Giglio F, Lorusso A, Perini T, Piemontese S, Marcatti M, Lorentino F, Xue E, Clerici D, Corti C, Bernardi M, Assanelli A, Greco R, Ciceri F, Peccatori J. Treosulfan-Based Conditioning Regimen Prior to Allogeneic Stem Cell Transplantation: Long-Term Results From a Phase 2 Clinical Trial. Front Oncol. 2021 Sep 10;11:731478. doi: 10.3389/fonc.2021.731478. eCollection 2021.

MeSH Terms

Conditions

LeukemiaLeukemia, Myelogenous, Chronic, BCR-ABL PositiveMyelodysplastic SyndromesLymphoma, Large B-Cell, DiffuseHodgkin DiseaseLeukemia, Lymphocytic, Chronic, B-CellMultiple Myeloma

Condition Hierarchy (Ancestors)

Neoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLeukemia, MyeloidMyeloproliferative DisordersBone Marrow DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsLymphoma, B-CellLymphoma, Non-HodgkinLymphomaLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesLeukemia, B-CellLeukemia, LymphoidNeoplasms, Plasma CellHemostatic DisordersVascular DiseasesCardiovascular DiseasesParaproteinemiasBlood Protein DisordersHemorrhagic Disorders

Study Officials

  • Fabio FC Ciceri, MD

    STUDY DIRECTOR

Central Study Contacts

Luciano LC Callegaro, Monitor

CONTACT

Stefania ST Trinca, Data Manager

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

January 10, 2008

First Posted

January 22, 2008

Study Start

September 1, 2005

Primary Completion

December 1, 2009

Study Completion

December 1, 2010

Last Updated

August 11, 2009

Record last verified: 2009-08

Locations