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LeukemiaPhase 2NCT01050439

Unrelated Donor Transplant for Malignant and Non-Malignant Disorders

A Phase 2 study testing UDAlloSCT and Therapy in people with Leukemia and Lymphoma, run by Columbia University at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Unrelated matched donor (cord blood, bone marrow or peripheral blood) allogeneic stem cell transplantation (UDAlloSCT) with either myeloablative or reduced intensity conditioning will be well tolerated and result in a high degree of engraftment in patients with selected malignant and non malignant disorders. Read more about this study

Who is it for?
Ages Up to 55 years
Conditions the study is for
Leukemia, Lymphoma, Bone Marrow Failure Syndromes
What is being tested?
UDAlloSCT and Therapy
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
22 participants
Where
1 siteUnited States
Study dates
Nov 2002to Apr 2011
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 Up to 55 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Adequate renal function defined as: serum creatinine 2.0 x normal, or creatinine clearance or radioisotope GFR > 40 ml/min/m2 or > 40 ml/min/1.73 m2 or an equivalent GFR as determined by the institutional normal range.
  • Adequate liver function defined as: total bilirubin < 2.5 x normal; or SGOT (AST) or SGPT (ALT) < 5.0 x normal.
  • Adequate cardiac function defined as: shortening fraction of > 25% by echocardiogram, or ejection fraction of > 40% by radionuclide angiogram or echocardiogram.
  • Adequate pulmonary function defined as: DLCO > 35% by pulmonary function test. For children who are uncooperative, no evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry > 94% in room air.
  • Diseases:
  • CML (CP, AP or BC)
Show all 38Show fewer
  • AML/MDS/JCML
  • ALL
  • Lymphoma (Hodgkin's and non-Hodgkin's)
  • Non-malignant disorders
  • Bone Marrow Failure Syndromes: Patients with the following diagnoses are eligible:
  • Severe Aplastic Anemia:
  • Fanconi Anemia
  • Severe Congenital Neutropenia (Kostmann's Syndrome)
  • Amegakaryocytic Thrombocytopenia
  • Diamond-Blackfan Anemia
  • Infantile Osteopetrosis
  • Schwachman-Diamond Syndrome
  • Dyskeratosis Congenita
  • Other bone marrow failure syndromes at discretion of Principal Investigator
  • Immunodeficiencies:
  • SCIDS, all subtypes
  • Combined Immunodeficiency Syndrome
  • Wiskott-Aldrich syndrome
  • Chronic Granulomatous Disease
  • Chediak-Higashi Syndrome
  • Leukocyte Adhesion Deficiency
  • Other immunodeficiencies at discretion of Principal Investigator
  • Inborn Errors of Metabolism (IEOM):
  • Transplant is recommended for the following disorders: Hurler syndrome (L-iduronidase deficiency, MPS-I), Maroteaux-Lamy syndrome (galactosamine-4-sulfatase deficiency, MP VI), Sly syndrome (glucuronidase deficiency, MPS-VII), Globoid cell Leukodystrophy (galactocerebrosidasedeficiency), Metachromatic leukodystrophy (arylsulfatase A deficiency), Childhood-onset X-linked adrenoleukodystrophy (X-ALD), Fucosidosis (fucosidase deficiency), Mannosidosis, Aspartylglucosaminuria, Niemann-Pick Disease Type B (acid sphingomyelinase deficiency), Gaucher disease (glucocerebrosidase deficiency) Type I (non neuropathic), Other diagnoses may be considered at the discretion of the Principal Investigator
  • For X-ALD patients greater than 5 years of age, IQ > 80 is required. For other patients greater than 5 years of age, IQ > 70 is required.
  • For patients less than 5 years of age, the developmental quotient or clinical neurodevelopmental examination should demonstrate potential for stabilization at a level of functioning where continuous life support (e.g. mechanical ventilation) would not be predicted to be required in the year following transplantation.
  • Histiocytosis:
  • Hemophagocytic Lymphohistiocytosis (HLH)
  • Familial Erythrophagocytic Lymphohistiocytosis
  • Langerhans Cell Histiocytosis
  • Malignant Histiocytosis
  • Other Malignant and non-malignant diseases: Other malignant and non-malignant diseases not listed above may be eligible if deemed appropriate by the Principal Investigator.

Who cannot join

  • Women who are pregnant and/or breast feeding are ineligible

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Columbia University Medical Center

    New York, New York, 10032

Other trials in United States

About this study

Official title: Unrelated Donor Stem Cell Transplant for Patients With Malignant and Non-Malignant Disorders

Unrelated matched donor (cord blood, bone marrow or peripheral blood) allogeneic stem cell transplantation (UDAlloSCT) with either myeloablative or reduced intensity conditioning will be well tolerated and result in a high degree of engraftment in patients with selected malignant and non malignant disorders.

Read the detailed description

This is a non-randomized study to determine the tolerability and degree of engraftment of unrelated matched donor allogeneic stem cell transplantation with either myeloablative or reduced intensity conditioning in patients with selected malignant and non malignant disorders. Patients will receive one of either full intensity or reduced intensity regimen based on the patient's disease status, organ function and performance and determined by the PI.

Being tested
UDAlloSCT, Therapy
Keywords
Allogeneic Stem Cell Transplant, Unrelated donor, Cord blood donor
Medical subject headings
Leukemia, Lymphoma, Bone Marrow Failure Disorders, Immunologic Deficiency Syndromes, Histiocytosis

What participants receive

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

  • UDAlloSCTProcedure or surgery

    unrelated matched donor allogeneic stem cell transplantation (UDAlloSCT)

  • TherapyOther

    Full Intensity Therapy (myeloablative) (TBI + Thiotepa + Cyc) OR Reduced Intensity Therapy (Fludarabine, Busulfan, and Alemutuzumab (FBA))

Study groups in NCT01050439
Study groupRoleWhat they receive
UDAlloSCT + TherapyExperimentalThis is a non-randomized study to test the safety and response of unrelated matched donor allogeneic stem cell transplantation (UDAlloSCT) with either myleoablative (full intensity) or reduced intensity conditioning therapy in patients with selected malignant and non-malignant disorders. UDAlloSCT has been performed in both adults and children as an alternative transplant for patients who lack and HLA-matched family donor in both malignant and non-malignant disease with varying degrees of response.
Receives: Procedure: UDAlloSCT; Other: Therapy

What is being measured

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

  1. Incidence of toxicity related to myeloablative therapy

    To determine the safety and toxicity of myeloablative therapy (TBI + Melphalan) and unrelated donor alloSCT in selected patients with malignant and non-malignant disorders.

    Measured: Up to 10 years from start of study

Secondary outcomes (3)

  1. Incidence of toxicity related to reduced intensity therapy

    To determine the safety and toxicity of reduced intensity therapy (Fludarabine, Busulfan, and Alemtuzumab (FBA) and unrelated donor alloSCT in selected patients with malignant and non malignant disorders

    Measured: Up to 10 years from start of study

  2. Percentage of donor chimerism

    To quantitate the percentage of donor chimerism following both myeloablative and reduced intensity conditioning and unrelated donor alloSCT in selected patients with malignant and non-malignant disorders.

    Measured: Up to 10 years from start of study

  3. Prevalence of progression free survival

    To estimate the progression free survival (PFS), if applicable, event free survival (EFS) and overall survival (OS) following unrelated donor alloSCT in selected patients with malignant and non malignant disorders.

    Measured: Up to 10 years from start of study

Timeline

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

  1. First posted15 Jan 2010 (estimated)
  2. Study startNov 2002
  3. Primary completionApr 2011
  4. Study completionApr 2011

Why it stopped: PI left the institution

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
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
22 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01050439
Sponsor's protocol ID
AAAB3095
Other IDs
CHNY-02-516
Status last verified
Apr 2015
First submitted
3 Jun 2008
Last updated
15 Apr 2015
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing UDAlloSCT and Therapy in people with Leukemia and Lymphoma, run by Columbia University at 1 site in United States.

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 38 inclusion and 1 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: stopped early, as of the sponsor's last update on 15 Apr 2015. 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 United States. 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 was stopped early (reason given: PI left the institution). 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?

Incidence of toxicity related to myeloablative therapy (time frame: Up to 10 years from start of study). 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 NCT01050439.

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 15 Apr 2015.

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