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Myelodysplastic SyndromesPhase 2NCT00098683

Amifostine in Treating Young Patients With Newly Diagnosed De Novo Myelodysplastic Syndromes

A Phase 2 study testing amifostine trihydrate in people with Myelodysplastic Syndromes and Myelodysplastic/Myeloproliferative Neoplasms, run by Children's Oncology Group at 85 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

RATIONALE: Drugs used in chemotherapy, such as amifostine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Read more about this study

Who is it for?
Ages 1 year to 21 years
What is being tested?
amifostine trihydrate
How many people
10 participants
Where
85 sitesin 4 countries
Study dates
Jan 2005to Oct 2009
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 1 year to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

DISEASE CHARACTERISTICS:

  • Histologically confirmed diagnosis of myelodysplastic syndromes (MDS)
  • One of the following subtypes:
  • Refractory anemia (RA)
  • RA with ringed sideroblasts
  • RA with excess blasts
  • Refractory cytopenia with multilineage dysplasia (RCMD)
  • RCMD and ringed sideroblasts
  • MDS, unclassified
  • MDS associated with isolated del 5(q)
  • De novo disease
  • No treatment-induced MDS
  • No juvenile myelomonocytic leukemia
  • No Down syndrome, Fanconi's anemia, or other inherited forms of hypoplastic bone marrow failure

PATIENT CHARACTERISTICS:

Age

  • 1 to 21 at original diagnosis

Performance status

  • Karnofsky 50-100% (patients > 16 years of age)
  • Lansky 50-100% (patients 1 to 16 years of age)

Life expectancy

  • At least 8 weeks

Hematopoietic

  • See Disease Characteristics

Hepatic

  • Bilirubin ≤ 1.5 times upper limit of normal (ULN)
  • ALT < 2.5 times ULN

Renal

  • Radioisotope glomerular filtration rate ≥ 60 mL/min OR
  • Creatinine clearance > 60 mL/min (based on Schwartz formula)
  • Calcium normal

Other

  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Serum electrolytes normal
  • Phosphorus normal
  • Magnesium normal
  • Glucose normal
  • No other concurrent malignancy

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • More than 8 weeks since prior growth factors
  • No concurrent growth factors
  • No concurrent hematopoietic stem cell transplantation
  • No concurrent immunomodulating agents

Chemotherapy

  • No prior amifostine
  • No other concurrent anticancer chemotherapy

Endocrine therapy

  • No concurrent daily steroid therapy

Radiotherapy

  • Not specified

Surgery

  • Not specified

Other

  • No prior therapy for MDS

Trial locations and contacts

85 sites in 4 countries.

85 sites in 4 countries

United States74 sites

  • Children's Hospital Medical Center of Akron

    Akron, Ohio, 44308-1062

  • University of New Mexico Cancer Research and Treatment Center

    Albuquerque, New Mexico, 87131-5636

  • Texas Tech University Health Sciences Center School of Medicine - Amarillo

    Amarillo, Texas, 79106

  • Winship Cancer Institute of Emory University

    Atlanta, Georgia, 30322

  • Children's Hospital of Austin

    Austin, Texas, 78701

  • Alvin and Lois Lapidus Cancer Institute at Sinai Hospital

    Baltimore, Maryland, 21215

  • CancerCare of Maine at Eastern Maine Medial Center

    Bangor, Maine, 04401

  • Maimonides Cancer Center at Maimonides Medical Center

    Brooklyn, New York, 11219

  • Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill

    Chapel Hill, North Carolina, 27599-7295

  • Hollings Cancer Center at Medical University of South Carolina

    Charleston, South Carolina, 29425

  • West Virginia University - Robert C. Byrd Health Sciences Center - Charleston Division

    Charleston, West Virginia, 25302

  • Blumenthal Cancer Center at Carolinas Medical Center

    Charlotte, North Carolina, 28232-2861

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039

  • Rainbow Babies and Children's Hospital

    Cleveland, Ohio, 44106-5000

  • Columbus Children's Hospital

    Columbus, Ohio, 43205-2696

  • Medical City Dallas Hospital

    Dallas, Texas, 75230

  • Children's Medical Center - Dayton

    Dayton, Ohio, 45404-1815

  • Barbara Ann Karmanos Cancer Institute

    Detroit, Michigan, 48201-1379

  • Carole and Ray Neag Comprehensive Cancer Center at the University of Connecticut Health Center

    Farmington, Connecticut, 06360-2875

  • Hurley Medical Center

    Flint, Michigan, 48503

  • Lee Cancer Care of Lee Memorial Health System

    Fort Myers, Florida, 33901

  • Cook Children's Medical Center - Fort Worth

    Fort Worth, Texas, 76104-9958

  • University of Florida Shands Cancer Center

    Gainesville, Florida, 32610-0232

  • Spectrum Health Hospital - Butterworth Campus

    Grand Rapids, Michigan, 49503-2560

  • Greenville Hospital System Cancer Center

    Greenville, South Carolina, 29605

  • Van Elslander Cancer Center at St. John Hospital and Medical Center

    Grosse Pointe Woods, Michigan, 48236

  • Hackensack University Medical Center Cancer Center

    Hackensack, New Jersey, 07601

  • Penn State Cancer Institute at Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033-0850

  • Memorial Cancer Institute at Memorial Regional Hospital

    Hollywood, Florida, 33021

  • Cancer Research Center of Hawaii

    Honolulu, Hawaii, 95813

  • Baylor University Medical Center - Houston

    Houston, Texas, 77030-2399

  • Edwards Comprehensive Cancer Center at Cabell Huntington Hospital

    Huntington, West Virginia, 25701

  • Indiana University Cancer Center

    Indianapolis, Indiana, 46202-5289

  • St. Vincent Indianapolis Hospital

    Indianapolis, Indiana, 46260

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216-4505

  • Kansas Masonic Cancer Research Institute at the University of Kansas Medical Center

    Kansas City, Kansas, 66160-7357

  • Children's Mercy Hospital

    Kansas City, Missouri, 64108

  • East Tennessee Children's Hospital

    Knoxville, Tennessee, 37916

  • Breslin Cancer Center at Ingham Regional Medical Center

    Lansing, Michigan, 48910

  • Sunrise Hospital and Medical Center

    Las Vegas, Nevada, 89109-2306

  • Arkansas Cancer Research Center at University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205

  • Jonathan Jaques Children's Cancer Center at Miller Children's Hospital

    Long Beach, California, 90801

  • Kosair Children's Hospital

    Louisville, Kentucky, 40232

  • Children's Hospital Central California

    Madera, California, 93638-8762

  • Marshfield Clinic - Marshfield Center

    Marshfield, Wisconsin, 54449

  • Miami Children's Hospital

    Miami, Florida, 33155

  • Midwest Children's Cancer Center

    Milwaukee, Wisconsin, 53226

  • Overlook Hospital

    Morristown, New Jersey, 07962

  • Saint Peter's University Hospital

    New Brunswick, New Jersey, 08901

  • Cancer Institute of New Jersey at UMDNJ - Robert Wood Johnson Medical School

    New Brunswick, New Jersey, 08903

  • Ochsner Cancer Institute at Ochsner Clinic Foundation

    New Orleans, Louisiana, 70121

  • Herbert Irving Comprehensive Cancer Center at Columbia University

    New York, New York, 10032

  • Advocate Christ Medical Center

    Oak Lawn, Illinois, 60453

  • Oklahoma University Medical Center

    Oklahoma City, Oklahoma, 73104

  • UNMC Eppley Cancer Center at the University of Nebraska Medical Center

    Omaha, Nebraska, 68198-6805

  • Sacred Heart Cancer Center at Sacred Heart Hospital

    Pensacola, Florida, 32504

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104-9786

  • St. Christopher's Hospital for Children

    Philadelphia, Pennsylvania, 19134-1095

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016-7710

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15213

  • Mayo Clinic Cancer Center

    Rochester, Minnesota, 55905

  • Kaiser Permanente Medical Center - Oakland

    Sacramento, California, 95825

  • Primary Children's Medical Center

    Salt Lake City, Utah, 84113-1100

  • University of Texas Health Science Center at San Antonio

    San Antonio, Texas, 78207

  • Methodist Children's Hospital of South Texas

    San Antonio, Texas, 78229-3993

  • Children's Hospital and Regional Medical Center - Seattle

    Seattle, Washington, 98105

  • Siteman Cancer Center at Barnes-Jewish Hospital

    St Louis, Missouri, 63110

  • All Children's Hospital

    St. Petersburg, Florida, 33701

  • SUNY Upstate Medical University Hospital

    Syracuse, New York, 13210

  • St. Joseph's Cancer Institute at St. Joseph's Hospital

    Tampa, Florida, 33607

  • Albert Einstein Cancer Center at Albert Einstein College of Medicine

    The Bronx, New York, 10461

  • New York Medical College

    Valhalla, New York, 10595

  • Kaplan Cancer Center at St. Mary's Medical Center

    West Palm Beach, Florida, 33407

  • Tod Children's Hospital - Forum Health

    Youngstown, Ohio, 44501

Other trials in United States

Canada9 sites

  • University of Alberta Hospital

    Edmonton, Alberta, T6G 1Z2

  • IWK Health Centre

    Halifax, Nova Scotia, B3K 6R8

  • McMaster Children's Hospital at Hamilton Health Sciences

    Hamilton, Ontario, L8N 3Z5

  • Cancer Centre of Southeastern Ontario at Kingston General Hospital

    Kingston, Ontario, K7L 3N6

  • Montreal Children's Hospital at McGill University Health Center

    Montreal, Quebec, H3H 1P3

  • Hopital Sainte Justine

    Montreal, Quebec, H3T 1C5

  • Centre Hospitalier Universitaire de Quebec

    Québec, G1V 4G2

  • Children's & Women's Hospital of British Columbia

    Vancouver, British Columbia, V6H 3V4

  • CancerCare Manitoba

    Winnipeg, Manitoba, R3E 0V9

Other trials in Canada

Australia1 site

  • Princess Margaret Hospital for Children

    Perth, Western Australia, 6001

Other trials in Australia

Puerto Rico1 site

  • San Jorge Children's Hospital

    Santurce, 00912

Other trials in Puerto Rico

About this study

Official title: A Phase II Study Of Amifostine In Children With Myelodysplastic Syndrome

RATIONALE: Drugs used in chemotherapy, such as amifostine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying how well amifostine works in treating young patients with newly diagnosed de novo myelodysplastic syndromes.

Read the detailed description

OBJECTIVES:

Primary

  • Determine the hematologic effects of amifostine, in terms of, complete and partial response, in pediatric patients with newly diagnosed de novo myelodysplastic syndromes (MDS).
  • Determine the safety and efficacy of this drug in these patients.

Secondary

  • Determine the efficacy of this drug in preventing conversion of MDS to acute myeloid leukemia (AML) in terms of the proportion of patients who remain free of AML at the completion of study treatment.
  • Determine the duration of progression-free remission from MDS conversion to AML in patients treated with this drug.
  • Determine the effect of karyotypic abnormalities on survival and the duration from diagnosis of MDS until conversion to AML in patients treated with this drug.
  • Determine the effect of bone marrow blast count on survival and the duration from diagnosis of MDS until conversion to AML in patients treated with this drug.
  • Determine the effect of the number of cytopenias on survival in patients treated with this drug.
  • Correlate the duration of time from diagnosis of MDS until conversion to AML with survival in patients treated with this drug.

OUTLINE: This is a multicenter study.

Patients receive amifostine IV over 1-3 minutes on days 1, 3, 5, 8, 10, 12, 15, 17, and 19. Treatment repeats every 5 weeks for 2 courses in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease who are planning to undergo matched donor bone marrow or cord blood transplantation continue therapy until transplantation. Patients with stable or responding disease who are not undergoing transplantation may receive up to 4 additional courses of amifostine in the absence of disease progression or unacceptable toxicity.

Following completion of therapy with amifostine, patients are followed monthly for 1 year, every 2 months for 1 year, every 3 months for 1 year, every 6 months for 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 10-20 patients will be accrued for this study within 5-10 months.

Being tested
amifostine trihydrate
Keywords
refractory anemia with excess blasts, refractory anemia, refractory anemia with ringed sideroblasts, de novo myelodysplastic syndromes, myelodysplastic/myeloproliferative neoplasm, unclassifiable, refractory cytopenia with multilineage dysplasia
Medical subject headings
Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases, Anemia, Refractory, with Excess of Blasts, Anemia, Refractory, Myeloproliferative Disorders

What participants receive

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

  • amifostine trihydrateDrug

What is being measured

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

  1. Hematological effects (complete and partial response)

  2. Safety and efficacy

Secondary outcomes (5)

  1. Efficacy

  2. Duration of progression-free remission

  3. Effect of karyotypic abnormalities on survival

  4. Effect of the number of cytopenias on survival

  5. Correlation of the duration of time from diagnosis of myelodysplastic syndromes until conversion to acute myeloid leukemia

Timeline

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

  1. First posted8 Dec 2004 (estimated)
  2. Study startJan 2005
  3. Primary completionOct 2007
  4. Study completionOct 2009

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 2
Primary purpose
Treatment
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00098683
Sponsor's protocol ID
AAML0121
Other IDs
CDR0000398140, COG-AAML0121
Status last verified
Feb 2014
First submitted
7 Dec 2004
Last updated
13 Feb 2014
Results posted
No
Data monitoring committee
Yes

Publications

  • Mathew P, Gerbing R, Alonzo TA, Wallas T, Gong JZ, Jasty R, Jorstad DT, Raimondi SC, Chavez CM, Eisenberg NL, Hirsch B, Gamis A, Smith FO, Arceci RJ. A phase II study of amifostine in children with myelodysplastic syndrome: a report from the Children's Oncology Group study (AAML0121). Pediatr Blood Cancer. 2011 Dec 15;57(7):1230-2. doi: 10.1002/pbc.23164. Epub 2011 Jun 16.

    PubMed 21681928

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing amifostine trihydrate in people with Myelodysplastic Syndromes and Myelodysplastic/Myeloproliferative Neoplasms, run by Children's Oncology Group at 85 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?

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 13 Feb 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 85 sites in United States, Canada, Australia, Puerto Rico. 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?

Hematological effects (complete and partial response). The study lists 2 primary outcomes in all. 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 NCT00098683, 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 13 Feb 2014.

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