Acute Lymphoblastic Leukaemias (ALL)Phase 2NCT02333058
Treosulfan-based Conditioning in Paediatric Patients With Haematological Malignancies
A Phase 2 study testing Treosulfan in people with Acute Lymphoblastic Leukaemias (ALL) and Acute Myeloid Leukaemias (AML), run by medac GmbH at 24 sites in 6 countries.
Trial summary
The primary goal of this study is to evaluate an alternative myeloablative, but reduced toxicity conditioning regimen in children, to describe the safety and efficacy of intravenous (i.v.) Treosulfan administered as part of a standardised Fludarabine-containing conditioning and to contribute to the current… Read more about this study
- Who is it for?
- Ages 28 days to 17 years
- Conditions the study is for
- Acute Lymphoblastic Leukaemias (ALL), Acute Myeloid Leukaemias (AML), Myelodysplastic Syndromes (MDS)
- 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
- 70 participants
- Where
- 24 sitesin 6 countries
- Study dates
- 21 Nov 2014to 30 Sep 2019
- Phase
- Phase 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 28 days to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Haematological malignant disease i.e. ALL, AML, MDS or JMML, indicated for allo-HSCT.
- Indication for first allo-HSCT or second allo-HSCT due to disease relapse, graft failure, or secondary malignancy after previous HSCT.
- Available matched sibling donor (MSD), matched family donor (MFD) or matched unrelated donor (MUD). For bone marrow (BM) and peripheral blood (PB) match is defined as 9/10 or 10/10 allele match after four digit typing in human leucocyte antigens (HLA)-A, B, C, DRB1 and DQB1.
- Patients with ALL or AML in complete morphologic remission (blast counts <5 % in BM) and patients with MDS or JMML with blast counts < 20 % in BM at study entry.
- Age at time of registration from 28 days to less than 18 years of age.
- Lansky (patients aged <16 years) or Karnofsky (patients aged ≥ 16 years) performance score of at least 70 %.
Show all 9Show fewer
- Written informed consent of the parents/ legal guardians and patient's assent/consent according to national regulations.
- Females of child-bearing potential or male patients' partners with child-bearing potential must use a highly effective method of contraception (pearl index < 1 %) such as complete sexual abstinence, combined oral contraceptive, hormone intrauterine contraceptive device (IUCD), vaginal hormone ring, transdermal contraceptive patch, contraceptive implant or depot contraceptive injection in combination with a second method of contraception like a condom or a cervical cap / diaphragm with spermicide or surgical sterilisation (vasectomy) in male patients or male partners during the study and at least 6 months thereafter.
- Negative pregnancy test for females of child-bearing potential.
Who cannot join
- Third or later allo-HSCT.
- HSCT from haploidentical or umbilical cord blood donor.
- Symptomatic involvement of central nervous system (CNS) at study entry.
- Treatment with cytotoxic drugs within 10 days prior to day 7.
- Obese paediatric patients with body mass index: weight (kg)/[height (m)]² > 30 kg/m².
- Solid tumours (e.g. neuroblastoma, peripheral neuroectodermal tumour [PNET], Ewing sarcoma).
Show all 15Show fewer
- Fanconi anaemia and other deoxyribonucleic acid (DNA) breakage repair disorders.
- Impaired liver function indicated by Bilirubin > three times the upper limit of normal (ULN) or aspartate aminotransferase/alanine aminotransferase (AST/ALT) > five times ULN, or active infectious hepatitis.
- Impaired renal function indicated by estimated glomerular filtration rate ([GFR], according to the Schwartz formula) < 60 mL/min/1,73m2.
- Impaired cardiac function: severe cardiac insufficiency indicated by left ventricle ejection fraction (LVEF) < 35 %.
- Requirement for supplementary continuous oxygen.
- Severe active infection requiring deferral of conditioning.
- Human immunodeficiency virus (HIV) positivity.
- Known pregnancy, breast feeding.
- Known hypersensitivity to Treosulfan and/or Fludarabine.
Trial locations and contacts
24 sites in 6 countries.
24 sites in 6 countries
Germany13 sites
University Clinic Düsseldorf
Düsseldorf, 40225
University Clinic Erlangen-Nürnberg
Erlangen, 91054
Universitätsklinikum Essen
Essen, 45147
University Hospital Johann Wolfgang Goethe
Frankfurt, 60590
University Clinic Hamburg-Eppendorf
Hamburg, 20246
Medical University Hannover
Hanover, 30625
University Clinic Heidelberg
Heidelberg, 69120
University Clinic Jena
Jena, 07740
University Clinic München
München, 80804
University Clinic Münster
Münster, 48129
University Clinic Regensburg
Regensburg, 93053
University Clinic Ulm
Ulm, 89075
University Clinic Würzburg
Würzburg, 97080
Poland4 sites
Bydgoszcz Medical University
Bydgoszcz, 85-094
Kraków Medical University
Krakow, 30-663
Lublin Medical University
Lublin, 20-093
Wroclaw Medical University
Wroclaw, 50-368
United Kingdom3 sites
Birmingham Children's Hospital
Birmingham, B4 6NH
Central Manchester University Hospital
Manchester, M13 9WL
Sheffield Children's Hospital
Sheffield, S10 2TH
Italy2 sites
Ospedale Bambino Gesu Roma
Rome, 00165
Ospedale Infantile Regina Margherita Torino
Turin, 10126
Czechia1 site
University Hospital Motol, Charles University, Prague
Prague, 150-06
About this study
Official title: Clinical Phase II Trial to Describe the Safety and Efficacy of Treosulfan-based Conditioning Therapy Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Paediatric Patients With Haematological Malignancies
The primary goal of this study is to evaluate an alternative myeloablative, but reduced toxicity conditioning regimen in children, to describe the safety and efficacy of intravenous (i.v.) Treosulfan administered as part of a standardised Fludarabine-containing conditioning and to contribute to the current pharmacokinetic model to be able to finally give age (or body surface area) dependent dose recommendations. The treatment regimens given in the protocol MC-FludT.17/M are based on sufficient clinical safety and efficacy data. Considering the vital indication for allogeneic haematopoietic stem cell transplantation of the selected patient population, the risk-benefit assessment is therefore reasonably in favour of the study conduct.
Read the detailed description
The protocol MC-FludT.17/M is a clinical phase II trial to describe the safety and efficacy of Treosulfan-based conditioning therapy prior to allogeneic haematopoietic stem cell transplantation (allo-HSCT) in at least 70 paediatric patients with haematological malignancies (male and female children with haematological malignant diseases as acute lymphoblastic leukaemias (ALL), acute myeloid leukaemias (AML), myelodysplastic syndromes (MDS) and juvenile myelomonocytic leukaemias (JMML), requiring myeloablative conditioning treatment with following allo-HSCT).
Treosulfan dose per day is to be calculated by using body surface area (BSA). Two background conditioning regimens with Treosulfan are allowed: One regimen consists of a standardised Fludarabine-containing regimen and the other consists of an intensified regimen with Fludarabine and ThioTEPA.
Freedom from transplant (treatment)-related mortality (TRM), defined as death from any transplant-related cause from the day of first administration of study medication until day +100 after HSCT is the primary objective of the trial.
Moreover, the current pharmacokinetic (PK) model should be contributed to be able to finally give age (or BSA) dependent dose recommendations.
- Conditions
- Acute Lymphoblastic Leukaemias (ALL), Acute Myeloid Leukaemias (AML), Myelodysplastic Syndromes (MDS), Juvenile Myelomonocytic Leukaemias (JMML)
- Being tested
- Treosulfan
- Keywords
- ALL, AML, MDS, JMML
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TreosulfanDrug
Also called Treograft®, Ovastat®
Treosulfan dose per day is to be calculated by using BSA:
One dose of Treosulfan per day on three consecutive days (day -6, day -5 and day -4) as intravenous (i.v.) infusion, given over 2 hours.
Two background conditioning regimens with Treosulfan are allowed: One regimen consists of a standardised Fludarabine-containing regimen (regimen A) and the other consists of an intensified regimen with Fludarabine and ThioTEPA (regimen B). The investigator decides for each individual patient whether to treat the patient with regimen A or with regimen B.
Treosulfan: i.v., BSA adapted: 10, 12 or 14 g/m²/day within 120 min to be administered prior to Fludarabine; Fludarabine: i.v., 30 mg/m2/day on days from -7 to -3 prior to HSCT; ThioTEPA (Regimen B): i.v., 2 x 5mg/kg/day on day -2.
| Study group | Role | What they receive |
|---|---|---|
| Treosulfan | Experimental | Treosulfan dose per day is to be calculated by using BSA. One dose of Treosulfan per day on three consecutive days (day -6, day -5 and day -4) as intravenous (i.v.) infusion, given over 2 hours.
Two background conditioning regimens with Treosulfan are allowed: One regimen consists of a standardised Fludarabine-containing regimen (regimen A) and the other consists of an intensified regimen with Fludarabine and ThioTEPA (regimen B). The investigator decides for each individual patient whether to treat the patient with regimen A or with regimen B.
Treosulfan: i.v., BSA adapted: 10, 12 or 14 g/m²/day within 120 min to be administered prior to Fludarabine; Fludarabine: i.v., 30 mg/m2/day on days from -7 to -3 prior to HSCT; ThioTEPA (Regimen B): i.v., 2 x 5mg/kg/day on day -2. Receives: Drug: Treosulfan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Freedom from transplant (treatment)-related mortality (TRM)
TRM is defined as death from any transplant-related cause
Measured: from the day of first administration of study medication until day +100 after HSCT
Secondary outcomes (8)
Engraftment after HSCT
Engraftment is defined as first of three consecutive days for each of the following four criteria:
- a leukocyte count of more than 1 x 109/L
- an absolute neutrophil count (ANC) of more than 0.5 x 109/L
- a platelet count of at least 20 x 109/L in the absence of platelet transfusion
- a platelet count of at least 50 x 109/L in the absence of platelet transfusion
Measured: until engraftment
Safety including early toxicity until day +100 after HSCT, serious adverse reactions (SARs) until the end of the longer-term follow-up phase
based on Common Terminology Criteria for Adverse Events (CTCAE) version 4.03
Measured: until 12 months after HSCT
Hepatic sinusoidal obstruction syndrome (HSOS), lung toxicity (CTCAE term pulmonary fibrosis), hepatic toxicity and infections of any CTCAE grade (non-serious and serious)
Measured: until day +100 after HSCT
Donor-type chimerism
The incidences of complete donor-type chimerism will be estimated as the number of patients with complete chimerism divided by the total number of patients at risk.
Measured: on day +28, day +100 and 12 months after HSCT
Non relapse mortality (NRM), transplant related mortality (TRM), graft failure rate, incidence of relapse/progression, relapse-free/progression-free survival (RFS/PFS) and overall survival (OS)
Non-relapse mortality will be defined as the probability of dying in the absence of persisting disease or previous occurrence of relapse/progression or graft failures.
TRM is defined as the probability of dying from a transplant-related cause. The associated time span is defined as the interval from day 0 to death due to a transplant-related cause.
The incidence of relapse/progression is defined as the probability of having relapse/progression of the underlying disease.
Relapse-free/progression-free survival is defined as the time length between day 0 and the date of relapse/progression of the underlying disease or death due to any cause.
OS after HSCT is defined as the probability of surviving. Survival time is defined as the time period between day 0 and the day of death due to any cause.
Kaplan-Meier methods will be applied for estimating the probability of these parameters over time.
Measured: after 12 months after HSCT and until the end of the longer-term follow-up phase
Incidence and severity of acute (until day +100) and chronic (until 12 months after HSCT) graft versus host disease (aGvHD/cGvHD)
The probability of grade I-IV and grade III-IV aGvHD will be estimated by cumulative incidence rates and summarised for selected time points together with their approximate 90 % confidence intervals.
As for aGvHD, the probability of cGvHD will be estimated by cumulative incidence rates.
Measured: until 12 months after HSCT
Use of rescue therapies including donor-lymphocyte infusions (DLIs) and further conditioning regimens
Measured: until 12 months after HSCT
PK parameters of Treosulfan and its epoxides
The following PK parameters of Treosulfan and its epoxides will be measured: Clearance (CL); volume of distribution (Vss); terminal elimination rate constant (λz); terminal elimination half-life (t1/2); area under the concentration time curve from time zero to infinity (AUC∞); maximum observed concentration (Cmax, i.e. C end of infusion).
Measured: day -6 prior to HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2015 (estimated)
- Study start21 Nov 2014
- Primary completion24 Dec 2016
- Study completion30 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Lymphoblastic Leukaemias (ALL)Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- medac GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Celerion, Syneos Health
- Responsible party
- Sponsor
- Investigators
- Ajay Vora, MD, Prof., Principal investigator, Great Ormond Street Hospital NHS Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02333058
- Sponsor's protocol ID
- MC-FludT.17/M
- Status last verified
- Apr 2020
- First submitted
- 22 Dec 2014
- Last updated
- 4 May 2020
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Kalwak K, Mielcarek M, Patrick K, Styczynski J, Bader P, Corbacioglu S, Burkhardt B, Sykora KW, Drabko K, Gozdzik J, Fagioli F, Greil J, Gruhn B, Beier R, Locatelli F, Muller I, Schlegel PG, Sedlacek P, Stachel KD, Hemmelmann C, Moller AK, Baumgart J, Vora A. Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies. Bone Marrow Transplant. 2020 Oct;55(10):1996-2007. doi: 10.1038/s41409-020-0869-6. Epub 2020 Mar 20.
PubMed 32203268
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Phase II Trial to Describe the Safety and Efficacy of Treosulfan-based Conditioning Therapy Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Paediatric Patients With Haematological Malignancies
- ClinicalTrials.gov ID
- NCT02333058
- Acronym / sponsor ID
- MC-FludT.17/M
- Conditions
- Acute Lymphoblastic Leukaemias (ALL), Acute Myeloid Leukaemias (AML), Myelodysplastic Syndromes (MDS), Juvenile Myelomonocytic Leukaemias (JMML)
- Keywords
- ALL, AML, MDS, JMML
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- medac GmbHIndustry (pharma, biotech, devices)
- Collaborators
- Celerion, Syneos Health
- Study start
- 21 Nov 2014 (actual)
- Primary completion
- 24 Dec 2016 (actual)
- Study completion
- 30 Sep 2019 (actual)
- First posted
- 7 Jan 2015 (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.
The primary goal of this study is to evaluate an alternative myeloablative, but reduced toxicity conditioning regimen in children, to describe the safety and efficacy of intravenous (i.v.) Treosulfan administered as part of a standardised Fludarabine-containing conditioning and to contribute to the current pharmacokinetic model to be able to finally give age (or body surface area) dependent dose recommendations. The treatment regimens given in the protocol MC-FludT.17/M are based on sufficient clinical safety and efficacy data. Considering the vital indication for allogeneic haematopoietic stem cell transplantation of the selected patient population, the risk-benefit assessment is therefore reasonably in favour of the study conduct.
Detailed description
The protocol MC-FludT.17/M is a clinical phase II trial to describe the safety and efficacy of Treosulfan-based conditioning therapy prior to allogeneic haematopoietic stem cell transplantation (allo-HSCT) in at least 70 paediatric patients with haematological malignancies (male and female children with haematological malignant diseases as acute lymphoblastic leukaemias (ALL), acute myeloid leukaemias (AML), myelodysplastic syndromes (MDS) and juvenile myelomonocytic leukaemias (JMML), requiring myeloablative conditioning treatment with following allo-HSCT).
Treosulfan dose per day is to be calculated by using body surface area (BSA). Two background conditioning regimens with Treosulfan are allowed: One regimen consists of a standardised Fludarabine-containing regimen and the other consists of an intensified regimen with Fludarabine and ThioTEPA.
Freedom from transplant (treatment)-related mortality (TRM), defined as death from any transplant-related cause from the day of first administration of study medication until day +100 after HSCT is the primary objective of the trial.
Moreover, the current pharmacokinetic (PK) model should be contributed to be able to finally give age (or BSA) dependent dose recommendations.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treosulfan | Experimental | Drug: TreosulfanTreosulfan dose per day is to be calculated by using BSA. One dose of Treosulfan per day on three consecutive days (day -6, day -5 and day -4) as intravenous (i.v.) infusion, given over 2 hours. Two background conditioning regimens with Treosulfan are allowed: One regimen consists of a standardised Fludarabine-containing regimen (regimen A) and the other consists of an intensified regimen with Fludarabine and ThioTEPA (regimen B). The investigator decides for each individual patient whether to treat the patient with regimen A or with regimen B. Treosulfan: i.v., BSA adapted: 10, 12 or 14 g/m²/day within 120 min to be administered prior to Fludarabine; Fludarabine: i.v., 30 mg/m2/day on days from -7 to -3 prior to HSCT; ThioTEPA (Regimen B): i.v., 2 x 5mg/kg/day on day -2. |
Who may qualify
- Ages
- 28 days to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Haematological malignant disease i.e. ALL, AML, MDS or JMML, indicated for allo-HSCT.
- Indication for first allo-HSCT or second allo-HSCT due to disease relapse, graft failure, or secondary malignancy after previous HSCT.
- Available matched sibling donor (MSD), matched family donor (MFD) or matched unrelated donor (MUD). For bone marrow (BM) and peripheral blood (PB) match is defined as 9/10 or 10/10 allele match after four digit typing in human leucocyte antigens (HLA)-A, B, C, DRB1 and DQB1.
- Patients with ALL or AML in complete morphologic remission (blast counts <5 % in BM) and patients with MDS or JMML with blast counts < 20 % in BM at study entry.
- Age at time of registration from 28 days to less than 18 years of age.
Key exclusion criteria (15 listed)
- Third or later allo-HSCT.
- HSCT from haploidentical or umbilical cord blood donor.
- Symptomatic involvement of central nervous system (CNS) at study entry.
- Treatment with cytotoxic drugs within 10 days prior to day 7.
- Obese paediatric patients with body mass index: weight (kg)/[height (m)]² > 30 kg/m².
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: Treograft®, Ovastat®Treosulfan dose per day is to be calculated by using BSA: One dose of Treosulfan per day on three consecutive days (day -6, day -5 and day -4) as intravenous (i.v.) infusion, given over 2 hours. Two background conditioning regimens with Treosulfan are allowed: One regimen consists of a standardised Fludarabine-containing regimen (regimen A) and the other consists of an intensified regimen with Fludarabine and ThioTEPA (regimen B). The investigator decides for each individual patient whether to treat the patient with regimen A or with regimen B. Treosulfan: i.v., BSA adapted: 10, 12 or 14 g/m²/day within 120 min to be administered prior to Fludarabine; Fludarabine: i.v., 30 mg/m2/day on days from -7 to -3 prior to HSCT; ThioTEPA (Regimen B): i.v., 2 x 5mg/kg/day on day -2. | Drug | Treosulfan |
Primary outcome
Freedom from transplant (treatment)-related mortality (TRM)
Time frame: from the day of first administration of study medication until day +100 after HSCT
TRM is defined as death from any transplant-related cause
8 secondary outcomes
Engraftment after HSCT
Time frame: until engraftment
Safety including early toxicity until day +100 after HSCT, serious adverse reactions (SARs) until the end of the longer-term follow-up phase
Time frame: until 12 months after HSCT
Hepatic sinusoidal obstruction syndrome (HSOS), lung toxicity (CTCAE term pulmonary fibrosis), hepatic toxicity and infections of any CTCAE grade (non-serious and serious)
Time frame: until day +100 after HSCT
Donor-type chimerism
Time frame: on day +28, day +100 and 12 months after HSCT
Non relapse mortality (NRM), transplant related mortality (TRM), graft failure rate, incidence of relapse/progression, relapse-free/progression-free survival (RFS/PFS) and overall survival (OS)
Time frame: after 12 months after HSCT and until the end of the longer-term follow-up phase
Incidence and severity of acute (until day +100) and chronic (until 12 months after HSCT) graft versus host disease (aGvHD/cGvHD)
Time frame: until 12 months after HSCT
Use of rescue therapies including donor-lymphocyte infusions (DLIs) and further conditioning regimens
Time frame: until 12 months after HSCT
PK parameters of Treosulfan and its epoxides
Time frame: day -6 prior to HSCT
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 4 May 2020.
- Derived from this study: PubMed 32203268Kalwak K, Mielcarek M, Patrick K, Styczynski J, Bader P, Corbacioglu S, Burkhardt B, Sykora KW, Drabko K, Gozdzik J, Fagioli F, Greil J, Gruhn B, Beier R, Locatelli F, Muller I, Schlegel PG, Sedlacek P, Stachel KD, Hemmelmann C, Moller AK, Baumgart J, Vora A. Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies. Bone Marrow Transplant. 2020 Oct;55(10):1996-2007. doi: 10.1038/s41409-020-0869-6. Epub 2020 Mar 20.
- sponsor's homepageLink provided by the sponsor.
Related trials for Acute Lymphoblastic Leukaemias (ALL)
RecruitingObservational study
A Multi-site Study to Evaluate the Persistence of Protective Immunity to Routine Childhood Vaccinations in Participants With B-ALL/Ly Who Have Received Blinatumomab
B-Cell ALL, B-Cell Acute Lymphoblastic Leukaemia
Not yet recruitingPhase 1
Phase I Clinical Trial of ThINKK Adoptive Immunotherapy After Allogeneic Hematopoietic Transplantation in Children With Leukemia or Neuroblastoma
Leukaemia (Acute Lymphoblastic), Leukaemia (Acute Myeloid)
Active, not recruitingPhase 1/2
A Study of BN104 in the Treatment of Acute Leukemia
ALL, Adult, AML, Adult
Recruiting
Evaluation of Acute and Chronic Nephrotoxicity in Acute Lymphatic Leukemia Patients Using Ultrasound Localization Microscopy
Acute Lymphoblastic Leukaemias (ALL), Long Term Follow-Up
Active, not recruitingObservational study
A Multi-center Retrospective Study of INO Treating B-ALL
Acute Lymphoblastic Leukaemia, Acute Lymphoblastic Leukaemias (ALL)
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.
Ajay Vora, MD, Prof. principal investigator
Great Ormond Street Hospital NHS Trust
24 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Treosulfan in people with Acute Lymphoblastic Leukaemias (ALL) and Acute Myeloid Leukaemias (AML), run by medac GmbH at 24 sites in 6 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 9 inclusion and 15 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 4 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in Germany, Poland, United Kingdom, Italy, Austria and 1 more country. 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 phase mean?
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?
Freedom from transplant (treatment)-related mortality (TRM) (time frame: from the day of first administration of study medication until day +100 after HSCT). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02333058, which also lists 1 linked publication.
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 4 May 2020.