Allogenic Stem Cell Transplantation for Children, Adolescents and Young Adults With Relapsed or Refractory AML

NCT00606723 | Completed Phase 3 DMC

• 2 other identifiers: AML SCT-BFM 20072007-004517-34
• Study Type: interventional
• Target: 154
• Locations: 3 countries
• Sites: 24

Brief Summary

1. 1.To evaluate whether stem cell transplantation from a matched sibling donor is equivalent to a matched unrelated donor in in a second complete remission of acute myeloid leukemia (AML).
2. 2.To evaluate whether stem cell transplantation (SCT) after chemotherapy (FLAMSA-schema) increases survival compared to a threshold derived from historical data
3. 3.To evaluate whether SCT from haploidentical donors for children having no matched donor will result in better survival with acceptable toxicity.

Conditions

Acute Myeloid Leukemia (AML)

Keywords

relapsed or refractory AML; in children, adolescents and young adults

Outcome Measures

Primary Outcomes (3)

• To evaluate whether stem cell transplantation (SCT) from a matched sibling donor (MSD) is equivalent to a matched unrelated donor (MUD) in second complete remission (CR2).

  day 100

• To evaluate whether "FLAMSA" increases leukemia free survival (LFS) and overall survival (OS) as compared to a historic control group

  day 100

• To evaluate whether SCT from haploidentical donors for children having no matched donor will result in an acceptable toxicity profile and a better LFS as compared to historic controls.

  day 100

Secondary Outcomes (5)

• Prospective evaluation of event free survival (EFS), LFS, and OS after SCT from either a MSD or a MUD

  day 100

• To evaluate whether it is feasible to standardize transplantation procedures in children with AML within the AML-Berlin/Frankfurt/Münster (BFM) study network

  day 100

• Decrease of transplantation associated mortality by standardized donor selection criteria

  day 100

• To further evaluate the contribution of immunomediated effects for the treatment of children suffering from very high risk AML

  day 100

• Prospective evaluation of late toxicities

  day 100 and year 5

Study Arms (1)

1

EXPERIMENTAL

Group I: Relapsed AML-patients with blast cell reduction to \<20% before the second course of induction therapy. These patients will receive conventional SCT. Group II: Patients with non response to frontline treatment of AML, patients with blast cells \<20% before the second course of induction therapy who do not achieve a second remission and relapsed AML-patients with blast cells \>=20% before the second course of induction therapy. If these patients have a matched donor (MSD/MD) they will receive SCT with "FLAMSA". Group III: Patients who are eligible for Group II but have no matched donor. These patients will receive SCT from a haploidentical donor.

Biological: Hematopoietic stem cells from bone marrow or peripheral blood

Interventions

Hematopoietic stem cells from bone marrow or peripheral blood BIOLOGICAL

\> = 2 x 10\*8 nucleated cells (WBC)/kg body weight of the recipient or rather \> = 4 x 10\*6 nucleated CD34+ cells / kg body weight are required for engraftment. Suspension of stem cells is administered via intravenous infusion.

1

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Aged between 0-21 years
• Patients suffering from either refractory de novo AML or relapsed AML or patients with very high risk AML in CR1
• In sexually active patients two reliable contraception methods are used. This includes every combination of a hormonal contraceptive (such as injection, transdermal patch, implant, cervical ring) or of an intrauterine device(IUD) with a barrier method (e.g. diaphragm, cervical cap, or condom) or with a spermicide.
• Written informed consent of patient, parents or legal guardians

You may not qualify if:

• Severe renal impairment (GFR \< 30% predicted for age)
• Pregnant or lactating females
• Current participation in another clinical trial
• Patients ≥ 12 years old for Group 1 ("BuCyMel") (patients younger that 12 years continue to be included)

Sponsors & Collaborators

• Hannover Medical School: lead

Study Sites (24)

Graz University Hospital

Graz, 8036, Austria

Location

Innsbruck University Hospital

Innsbruck, 6020, Austria

Location

St. Anna Children Hospital

Vienna, 1090, Austria

Location

Teaching Hospital Motol

Prague, 15006, Czechia

Location

University Hospital Freiburg

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

Location

Heidelberg University Hospital

Heidelberg, Baden-Wurttemberg, 69120, Germany

Location

University Children's Hospital Tübingen

Tübingen, Baden-Wurttemberg, 72076, Germany

Location

University Children's Hospital Ulm

Ulm, Baden-Wurttemberg, 89075, Germany

Location

University Children's Hospital Erlangen-Nürnberg

Erlangen, Bavaria, 91054, Germany

Location

Dr. von Haunersches Kinderspital

München, Bavaria, 80337, Germany

Location

University Hospital Würzburg

Würzburg, Bavaria, 97080, Germany

Location

J.W. Goethe University Hospital and Faculty of Medicine

Frankfurt am Main, Hesse, 60590, Germany

Location

Gießen University Hospital

Giessen, Hesse, 35385, Germany

Location

Hannover Medical School, Department of Paediatrics, Paediatric Hematology and Oncology

Hanover, Lower Saxony, 30625, Germany

Location

University Hospital Greifswald

Greifswald, Mecklenburg-West Pomerania, 17475, Germany

Location

Düsseldorf University Hospital

Düsseldorf, North Rhine-Westphalia, 40225, Germany

Location

University Hospital Essen

Essen, North Rhine-Westphalia, 45122, Germany

Location

Münster University Hospital

Münster, North Rhine-Westphalia, 48149, Germany

Location

Carl Gustav Carus University Children's Hospital Dresden

Dresden, Saxony, 01307, Germany

Location

University Children's Hospital Halle

Halle, Saxony-Anhalt, 06097, Germany

Location

University Hospital Kiel

Kiel, Schleswig-Holstein, 24105, Germany

Location

University Children's Hospital Jena

Jena, Thuringia, 07724, Germany

Location

Charité Campus Rudolf Virchow Hospital

Berlin, 13353, Germany

Location

Hamburg-Eppendorf University Hospital

Hamburg, 20246, Germany

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Recurrence

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Martin Sauer, Prof. Dr.

  Hannover Medical School

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 21, 2008
• First Posted: February 4, 2008
• Study Start: April 1, 2010
• Primary Completion: February 24, 2021
• Study Completion: February 24, 2021
• Last Updated: March 22, 2021

Record last verified: 2021-03

Locations

AU (3); CZ (1); DE (20)