AzTrials
AzTrials

LeukemiaPhase 3NCT00047268

Donor Stem Cell Transplant With or Without Chemotherapy in Treating Children With Primary Myelodysplastic Syndrome

A Phase 3 study testing cytarabine, mercaptopurine and laboratory biomarker analysis in people with Leukemia and Myelodysplastic/Myeloproliferative Neoplasms, run by European Working Group of MDS in Childhood at 1 site in Germany.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

RATIONALE: Giving chemotherapy before a donor stem cell transplant helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and… Read more about this study

Who is it for?
Ages Up to 18 years
What is being tested?
cytarabine, mercaptopurine and laboratory biomarker analysis
Type of treatment
Surgery or procedure
How many people
Not given
Where
1 siteGermany
Study dates
Jul 1998to not given
Phase
Phase 3
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 Up to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

DISEASE CHARACTERISTICS:

  • Morphologically confirmed primary myelodysplastic syndromes (MDS)
  • Diagnosed between July 1, 1998 and June 30, 2002
  • No prior aplastic anemia
  • No prior congenital bone marrow failure syndrome, such as:
  • Fanconi's anemia
  • Kostmann syndrome
  • Shwachman syndrome
  • Dyskeratosis congenital
  • Amegakaryocytic thrombocytopenia
  • Diamond-Blackfan anemia
  • No Down syndrome
  • None of the following cytogenetic or molecular abnormalities:
  • t(8;21)(q22;q22)
  • t(15;17)(q22;q12)
  • inv(16)(p13;q22)
  • No typical clinical and cytogenetic features of acute myeloid leukemia FAB M7 (i.e., acute megakaryocytic leukemia) with fewer than 30% blasts in bone marrow or peripheral blood

PATIENT CHARACTERISTICS:

Age

  • Under 19

Performance status

  • Not specified

Life expectancy

  • Not specified

Hematopoietic

  • See Disease Characteristics

Hepatic

  • Not specified

Renal

  • Not specified

Other

  • No other concurrent illness that would preclude study

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • Not specified

Chemotherapy

  • No prior chemotherapy for MDS

Endocrine therapy

  • Not specified

Radiotherapy

  • No prior radiotherapy for MDS

Surgery

  • Not specified

Trial locations and contacts

1 site in 1 country.

Germany1 site

  • Universitaetskinderklinik - Universitaetsklinikum Freiburg

    Freiburg im Breisgau, D-79106

Other trials in Germany

About this study

Official title: Prospective Study of the Diagnosis and Treatment of Myelodysplastic Syndromes (MDS) in Childhood

RATIONALE: Giving chemotherapy before a donor stem cell transplant helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether donor stem cell transplant is more effective with or without chemotherapy in treating primary myelodysplastic syndrome.

PURPOSE: This phase III trial is studying how well donor stem cell transplant given with chemotherapy works and compares it with donor stem cell transplant without chemotherapy in treating children with primary myelodysplastic syndrome.

Read the detailed description

OBJECTIVES:

  • Determine, by a standard approach, the frequency of different FAB subtypes in children with primary myelodysplastic syndromes.
  • Determine the frequency of cytogenetic and molecular abnormalities in these patients.
  • Determine the survival of patients treated with allogeneic stem cell transplantation with or without induction chemotherapy.
  • Determine the rate of complete remission in patients treated with these regimens.
  • Determine the event-free survival of patients treated with these regimens.
  • Determine the relapse rate, morbidity, and mortality of patients treated with these regimens.
  • Determine different subsets of patients who benefit from these regimens.

OUTLINE: This is a multicenter study. Patients are stratified according to FAB subtype (refractory anemia (RA) or RA with ringed sideroblasts (RARS) vs RA with excess blasts (RAEB) vs RAEB in transformation (RAEB-t) vs juvenile myelomonocytic leukemia (JMML)).

Patients undergo complete medical and physical examination. Patients are screened for the following aberrations: -7, +8, +21, t(8;21), t(15;17), and inv(16). Smears of peripheral blood and bone marrow, as well as bone marrow biopsies and all cytogenetic and molecular studies performed on blood or bone marrow, are evaluated by a panel of international experts.

Patients with progressive RA or RARS undergo allogeneic stem cell transplantation (ASCT) according to EWOG-MDS SCT studies. Patients with stable RA or RARS wait for an optimal donor before undergoing ASCT. Patients with RAEB with fewer than 15% bone marrow blasts undergo ASCT. Patients with RAEB with at least 15% bone marrow blasts and patients with RAEB-t with fewer than 30% bone marrow blasts receive standard acute myeloid leukemia (AML) induction therapy and then undergo ASCT. Patients with RAEB-t with at least 30% bone marrow blasts are considered for standard AML induction therapy.

Patients with advanced JMML undergo evaluation for splenectomy and receive chemotherapy with mercaptopurine and cytarabine every 3-4 weeks (for 1-4 doses). Patients then undergo ASCT.

Patients are followed every 6 months.

PROJECTED ACCRUAL: Not specified

Being tested
cytarabine, mercaptopurine, laboratory biomarker analysis, allogeneic bone marrow transplantation, biopsy
Keywords
juvenile myelomonocytic leukemia, childhood myelodysplastic syndromes, atypical chronic myeloid leukemia, BCR-ABL1 negative, myelodysplastic/myeloproliferative neoplasm, unclassifiable, chronic myelomonocytic leukemia
Medical subject headings
Leukemia, Myelodysplastic-Myeloproliferative Diseases, Leukemia, Myelomonocytic, Juvenile, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Myeloproliferative Disorders, Leukemia, Myelomonocytic, Chronic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Patient numbers in the different FAB subtypes

Secondary outcomes (2)

  1. Survival

  2. Event-free survival

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Jan 2003 (estimated)
  2. Study startJul 1998

Resources

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 3
Primary purpose
Diagnosis
Masking
None (open label)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00047268
Sponsor's protocol ID
CDR0000257581
Other IDs
EWOG-MDS-98, EU-20218
Status last verified
Jul 2007
First submitted
3 Oct 2002
Last updated
17 Sep 2013
Results posted
No

Publications

  • Drexler B, Schwarz-Furlan S, Baumann I, Rudelius M, Nollke P, Lebrecht D, Ramamoorthy S, Rotari N, Karow A, Hirabayashi S, Beier F, Behrens YL, Gohring G, Kalb R, Wlodarski MW, Strahm B, Erlacher M, Niemeyer CM, Yoshimi A. Long-term outcomes of patients with refractory cytopenia of childhood under observation only. Blood Adv. 2025 Aug 26;9(16):4279-4285. doi: 10.1182/bloodadvances.2025016136.

    PubMed 40554414

  • Pastor VB, Sahoo SS, Boklan J, Schwabe GC, Saribeyoglu E, Strahm B, Lebrecht D, Voss M, Bryceson YT, Erlacher M, Ehninger G, Niewisch M, Schlegelberger B, Baumann I, Achermann JC, Shimamura A, Hochrein J, Tedgard U, Nilsson L, Hasle H, Boerries M, Busch H, Niemeyer CM, Wlodarski MW. Constitutional SAMD9L mutations cause familial myelodysplastic syndrome and transient monosomy 7. Haematologica. 2018 Mar;103(3):427-437. doi: 10.3324/haematol.2017.180778. Epub 2017 Dec 7.

    PubMed 29217778

  • Gohring G, Michalova K, Beverloo HB, Betts D, Harbott J, Haas OA, Kerndrup G, Sainati L, Bergstraesser E, Hasle H, Stary J, Trebo M, van den Heuvel-Eibrink MM, Zecca M, van Wering ER, Fischer A, Noellke P, Strahm B, Locatelli F, Niemeyer CM, Schlegelberger B. Complex karyotype newly defined: the strongest prognostic factor in advanced childhood myelodysplastic syndrome. Blood. 2010 Nov 11;116(19):3766-9. doi: 10.1182/blood-2010-04-280313. Epub 2010 Aug 27.

    PubMed 20802024

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing cytarabine, mercaptopurine and laboratory biomarker analysis in people with Leukemia and Myelodysplastic/Myeloproliferative Neoplasms, run by European Working Group of MDS in Childhood at 1 site in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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: status not confirmed, as of the sponsor's last update on 17 Sep 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Germany. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

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?

Patient numbers in the different FAB subtypes. 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 NCT00047268, which also lists 3 linked publications.

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 17 Sep 2013.

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