Hematological MalignanciesPhase 1/2NCT01063647
Dose-range Finding Treosulfan-based Conditioning
A Phase 1/2 study testing Treosulfan in people with Hematological Malignancies, run by medac GmbH at 7 sites in 4 countries.
Trial summary
Evaluation of the safety and efficacy of 3 x 10, 3 x 12 or 3 x 14 g/m² Treosulfan resp., combined with 5 x 30 mg/m² fludarabine prior to allogeneic, hematopoietic stem cell transplantation of patients with hematological malignancies, but non-eligible to standard conditioning treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematological Malignancies
- What is being tested?
- Treosulfan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 7 sitesin 4 countries
- Study dates
- Nov 2001to Jun 2006
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with a haematological chemosensitive malignancy indicated for an allogeneic transplantation, but presenting an increased toxicity risk for classical (high-dose busulfan or standard-dose total body irradiation) conditioning therapies (remission criteria ref. to Appendix L):
- CML in first or subsequent chronic phase
- NHL in 2nd CR/PR, chemosensitive PR after autologous transplantation ; CLL in 2nd or subsequent CR/PR
- Relapsed Morbus Hodgkin (MH) after autologous transplantation
- Multiple Myeloma (MM) stage II and III according to Durie and Salmon
- AML in 2nd CR/PR or high-risk AML in 1st CR/PR
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- High-risk defined for example by the following:
- Cytogenetics: -5/5q, -7/7q, del(5q), abnormalities of 3q, complex karyotype (> 3 abnormalities), or
- PR after 1 cycle of induction therapy
- ALL in 2nd CR/PR or high-risk ALL in 1st CR/PR
- High-risk defined as follows:
- Leukocytes > 3000/µl (B-Linage) or > 100000/µl (T-Linage);
- Pro-B-ALL, pre-T-ALL
- Cytogenetics: t(9;22)/BCR-ABL; t(4;11)/ALL1-AF
- MDS (patients without prior chemotherapy may be included)
- Availability of an HLA-identical sibling donor (MRD) or HLA-identical unrelated donor (MUD) or one mismatch (out of the 6 standard markers) sibling donor (1 misMRD):
- HLA-identity defined by the following markers: A, B, DRB1. DQB1 must be recorded.
- Age > 18 years
- Karnofsky Index > 80 %
- Adequate contraception in female patients of child-bearing potential
- Co-operative behavior of individual patients
- Written informed consent
Who cannot join
- Completely chemotherapy-resistant disease
- Severe cardiac insufficiency, severe cardio-vascular or other severe concomitant diseases
- Symptomatic malignant involvement of the CNS
- Active infectious disease
- HIV-positive or active hepatitis infection
- Impaired liver function (Bilirubin > 1.5 x upper normal limit; Transaminases > 3.0 x upper normal limit)
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- 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 fludarabine and/or treosulfan
- Parallel participation in another experimental drug trial
Trial locations and contacts
7 sites in 4 countries.
7 sites in 4 countries
Germany4 sites
Charité University Hospital Berlin
Berlin, 12203
University Hospital Hamburg Eppendorf
Hamburg, 20246
5th Medical Clinic, Clinic North
Nuremberg, 90340
University Hospital Rostock
Rostock, 18057
Sweden1 site
Karolinska University Hospital & Karolinska Institute
Stockholm, 141 86
About this study
Official title: Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Advanced Hematological Malignancies After Treosulfan-based Conditioning Therapy - A Clinical Phase II Study
Evaluation of the safety and efficacy of 3 x 10, 3 x 12 or 3 x 14 g/m² Treosulfan resp., combined with 5 x 30 mg/m² fludarabine prior to allogeneic, hematopoietic stem cell transplantation of patients with hematological malignancies, but non-eligible to standard conditioning treatment.
- Conditions
- Hematological Malignancies
- Being tested
- Treosulfan
- Keywords
- Treosulfan, Allogeneic stem cell transplantation, Conditioning therapy
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TreosulfanDrug
Also called Ovastat
10 g/m² i.v. infusion, day -6, -5, -4
TreosulfanDrug
Also called Ovastat
12 g/m² i.v. infusion, day -6, -5, -4
TreosulfanDrug
Also called Ovastat
14 g/m² i.v. infusion, day -6, -5, -4
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Treosulfan: 10 g/m² i.v. on 3 consecutive days (day -6 to -4) Receives: Drug: Treosulfan |
| 2 | Experimental | Treosulfan:12 g/m² i.v. on 3 consecutive days (day -6 to -4) Receives: Drug: Treosulfan |
| 3 | Experimental | Treosulfan: 14 g/m² i.v. on 3 consecutive days (day -6 to -4) Receives: Drug: Treosulfan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety - Evaluation of feasibility and tolerability of 3 x 10, 12 or 14 g/m² Treosulfan combined with 5 x 30 mg/m² fludarabine prior to allogeneic stem cell transplantation • frequency and severity of TRM until 6 months after transplantation
Measured: 6 months
Secondary outcomes (1)
Efficacy - Evaluation of the proportion of relapse- and/or progression free patients six months after transplantation (using standard remission criteria)
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2010 (estimated)
- Study startNov 2001
- Primary completionJun 2005
- Study completionJun 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematological MalignanciesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- medac GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mathias Freund, MD, Principal investigator, University Hospital Rostock
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01063647
- Sponsor's protocol ID
- MC-FludT.6/L
- Status last verified
- Nov 2023
- First submitted
- 3 Feb 2010
- Last updated
- 12 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Advanced Hematological Malignancies After Treosulfan-based Conditioning Therapy - A Clinical Phase II Study
- ClinicalTrials.gov ID
- NCT01063647
- Acronym / sponsor ID
- MC-FludT.6/L
- Conditions
- Hematological Malignancies
- Keywords
- Treosulfan, Allogeneic stem cell transplantation, Conditioning therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- medac GmbHIndustry (pharma, biotech, devices)
- Study start
- Nov 2001
- Primary completion
- Jun 2005 (actual)
- Study completion
- Jun 2006 (actual)
- First posted
- 5 Feb 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Evaluation of the safety and efficacy of 3 x 10, 3 x 12 or 3 x 14 g/m² Treosulfan resp., combined with 5 x 30 mg/m² fludarabine prior to allogeneic, hematopoietic stem cell transplantation of patients with hematological malignancies, but non-eligible to standard conditioning treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: TreosulfanTreosulfan: 10 g/m² i.v. on 3 consecutive days (day -6 to -4) |
| 2 | Experimental | Drug: TreosulfanTreosulfan:12 g/m² i.v. on 3 consecutive days (day -6 to -4) |
| 3 | Experimental | Drug: TreosulfanTreosulfan: 14 g/m² i.v. on 3 consecutive days (day -6 to -4) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- Patients with a haematological chemosensitive malignancy indicated for an allogeneic transplantation, but presenting an increased toxicity risk for classical (high-dose busulfan or standard-dose total body irradiation) conditioning therapies (remission criteria ref. to Appendix L):
- Availability of an HLA-identical sibling donor (MRD) or HLA-identical unrelated donor (MUD) or one mismatch (out of the 6 standard markers) sibling donor (1 misMRD):
- Age > 18 years
- Karnofsky Index > 80 %
- Adequate contraception in female patients of child-bearing potential
Key exclusion criteria (11 listed)
- Completely chemotherapy-resistant disease
- Severe cardiac insufficiency, severe cardio-vascular or other severe concomitant diseases
- Symptomatic malignant involvement of the CNS
- Active infectious disease
- HIV-positive or active hepatitis infection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TreosulfanAlso: Ovastat10 g/m² i.v. infusion, day -6, -5, -4 | Drug | 1 |
| TreosulfanAlso: Ovastat12 g/m² i.v. infusion, day -6, -5, -4 | Drug | 2 |
| TreosulfanAlso: Ovastat14 g/m² i.v. infusion, day -6, -5, -4 | Drug | 3 |
Primary outcome
Safety - Evaluation of feasibility and tolerability of 3 x 10, 12 or 14 g/m² Treosulfan combined with 5 x 30 mg/m² fludarabine prior to allogeneic stem cell transplantation • frequency and severity of TRM until 6 months after transplantation
Time frame: 6 months
1 secondary outcome
Efficacy - Evaluation of the proportion of relapse- and/or progression free patients six months after transplantation (using standard remission criteria)
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mathias Freund, MD principal investigator
University Hospital Rostock
7 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Treosulfan in people with Hematological Malignancies, run by medac GmbH at 7 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 22 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Germany, Finland, Poland, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety - Evaluation of feasibility and tolerability of 3 x 10, 12 or 14 g/m² Treosulfan combined with 5 x 30 mg/m² fludarabine prior to allogeneic stem cell transplantation • frequency and severity of TRM until 6 months after transplantation (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2026.