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Nonmalignant NeoplasmPhase 2NCT00895739

Alemtuzumab and Low-Dose Cyclosporine in Treating Patients With Severe Aplastic Anemia or Acquired Marrow Failure

A Phase 2 study testing alemtuzumab and cyclosporine in people with Nonmalignant Neoplasm, run by Federico II University at 1 site in Italy.

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 The sponsor has not verified this record for over two years, so it may be out of date.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: Immunosuppressive therapies, such as alemtuzumab and cyclosporine, may improve bone marrow function and increase blood cell counts. Giving alemtuzumab together with cyclosporine may be an effective treatment for severe aplastic anemia or acquired marrow failure. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Nonmalignant Neoplasm
What is being tested?
alemtuzumab and cyclosporine
How many people
50 participants
Where
1 siteItaly
Study dates
Jun 2006to not given
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

DISEASE CHARACTERISTICS:

  • Diagnosis of 1 of the following:
  • Severe or very severe aplastic anemia, as defined by the following criteria:
  • Meets ≥ 2 of the following criteria:
  • Absolute neutrophil count < 0.5 x 10^9/L (severe) or < 0.2 x 10^9/L (very severe)
  • Platelet count < 20 x 10^9/L
  • Reticulocyte count < 20 x 10^9/L
  • Hypocellular bone marrow (< 30% cellularity) without evidence of fibrosis or malignant cells
  • Single lineage acquired marrow failure (e.g., pure red cell aplasia, agranulocytosis, amegakaryocytic thrombocytopenia)
  • Paroxysmal nocturnal hemoglobinuria clone allowed
  • Failed first-line therapy with antithymocyte globulin (ATG) and cyclosporine OR not eligible for ATG-based studies
  • Failure is defined as lack of hematological response, requirement for chronic immunosuppressive treatment to sustain response, or relapse
  • Not eligible for a low-risk stem cell transplantation
  • No evidence of risky myelodysplastic syndromes (i.e., IPSS 3-4), as defined by the presence of marrow blast excess or karyotypic abnormalities, or other primitive marrow disease
  • No history of constitutional aplastic anemia (e.g., Fanconi anemia or dyskeratosis congenita)

PATIENT CHARACTERISTICS:

  • WHO performance status 0-2
  • Not pregnant or nursing
  • No active malignant tumor within the past 5 years
  • Transaminases ≤ 3 times upper limit of normal (ULN)
  • Albumin ≥ 1.5 g/L
  • Creatinine ≤ 3 times ULN
  • No CMV viremia, as defined by positive PCR or pp65 test
  • No cardiac failure (i.e., ejection fraction < 35%)
  • No other concurrent life-threatening disease (including HIV infection)

PRIOR CONCURRENT THERAPY:

  • No prior allogeneic stem cell transplantation
  • At least 2 weeks since prior cyclosporine or filgrastim (G-CSF)

Trial locations and contacts

1 site in 1 country.

Italy1 site, 1 recruiting

  • Federico II University Medical School

    Naples, 80131

    Bruno Rotoli, MD, 39-081-746-2068, rotoli@unina.it

    Recruiting

Other trials in Italy

About this study

Official title: Alemtuzumab and Low-Dose Cyclosporine-A as Alternative Immunosuppressive Treatment for Severe Aplastic Anemia (SAA) and Single-Lineage Aplastic Patients

RATIONALE: Immunosuppressive therapies, such as alemtuzumab and cyclosporine, may improve bone marrow function and increase blood cell counts. Giving alemtuzumab together with cyclosporine may be an effective treatment for severe aplastic anemia or acquired marrow failure.

PURPOSE: This phase II trial is studying the side effects of giving alemtuzumab together with cyclosporine and to see how well it works in treating patients with severe aplastic anemia or acquired marrow failure.

Read the detailed description

OBJECTIVES:

Primary

  • Determine the safety of alemtuzumab and low-dose cyclosporine, as defined by occurrence of adverse effects, in patients with severe aplastic anemia or single lineage acquired marrow failure.
  • Determine the efficacy of this regimen, in terms of overall survival, hematological response (partial and complete response, including time to response) and failure-free survival (failure is defined as no response, chronic treatment-maintained response, or relapse), in these patients.

Secondary

  • Evaluate the incidence of adverse effects after treatment.
  • Evaluate the long-term safety of alemtuzumab treatment.
  • Determine the time to achieve a complete hematological response.
  • Determine the proportion of patients maintaining hematological response free of any treatment.
  • Determine the incidence of relapse in responding patients.
  • Determine the incidence of severe infections.
  • Determine the requirement for IV antibiotics and antifungal therapy.
  • Determine the requirement for red cell and platelet transfusion.
  • Determine the incidence of CMV reactivation.
  • Determine the kinetics of immune reconstitution.
  • Determine the incidence of paroxysmal nocturnal hemoglobinuria clone (lymphoid or myeloid) development.
  • Determine the incidence of clonal evolution (i.e., karyotypic abnormalities or secondary myelodysplasia/leukemia).

OUTLINE: Patients receive alemtuzumab subcutaneously on days 1-5*. Patients also receive oral cyclosporine beginning on day 7 and continuing for ≥ 180 days, followed by a taper according to clinical condition.

NOTE: *Patients with single lineage aquired marrow failure receive alemtuzumab on days 1-4.

After completion of study therapy, patients will be followed up every 3 months for up to 2 years.

Being tested
alemtuzumab, cyclosporine
Keywords
aplastic anemia
Medical subject headings
Neoplasms, Anemia, Aplastic

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. Safety, as defined by occurrence of adverse effects

  2. Overall survival

  3. Hematologic response (partial and complete response, including time to response)

  4. Failure-free survival (failure is defined as no response, chronic treatment-maintained response, or relapse)

Secondary outcomes (12)

  1. Incidence of adverse effects after treatment

  2. Long-term safety of alemtuzumab treatment

  3. Time to achieve a complete hematological response

  4. Proportion of patients maintaining hematological response free of any treatment

  5. Incidence of relapse in responding patients

  6. Incidence of severe infections

  7. Requirement for IV antibiotics and antifungal therapy

  8. Requirement for red cell and platelet transfusion

  9. Incidence of CMV reactivation

  10. Kinetics of immune reconstitution

  11. Incidence of paroxysmal nocturnal hemoglobinuria (PNH) clone (lymphoid or myeloid) development

  12. Incidence of clonal evolution (i.e., karyotypic abnormalities or secondary myelodysplasia/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 May 2009 (estimated)
  2. Study startJun 2006

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
Non-randomised
Masking
None (open label)
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00895739
Sponsor's protocol ID
UNMS-ALESAA
Other IDs
CDR0000639649, EU-20927, EUDRACT-2008-001151-22
Status last verified
May 2009
First submitted
7 May 2009
Last updated
12 Aug 2013
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing alemtuzumab and cyclosporine in people with Nonmalignant Neoplasm, run by Federico II University at 1 site in Italy.

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 12 Aug 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 Italy. 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?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety, as defined by occurrence of adverse effects. The study lists 4 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 NCT00895739.

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 12 Aug 2013.

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