Trials Sponsors Conditions Drugs Pricing

Conditions

Myelodysplastic Syndromes

Keywords

AnemiaTransfusionAMLMDSCLLp53erythropoiesis

Outcome Measures

Primary Outcomes (1)

1) Interventional Drug Safety: dose limiting toxicities (DLT) of cenersen for each of three increasing dose levels as stipulated by the protocol in patients with lower risk MDS defined as low or intermediate-1 risk by IPSS.
Assessment of adverse events for each dosing level
6 months w/ 24 month follow-up

Secondary Outcomes (2)

Reduction in RBC transfusions
6 months w/ 24month follow-up
Improvement in Hemoglobin (Hb) levels
6 months w/ 24 month follow-up

Study Arms (3)

Group 1

ACTIVE COMPARATOR

cenersen IV infusion 0.3 mg/kg/day (0.1 mg/kg/h x 3 h x 4 days) for 6 cycles Administration of study drug will be over 4 consecutive days at the outset of each 28 day cycle for a total of 6 cycles for each patient. Dexamethasone 20 mg po weekly can be added after week 16 if stable disease but no Hematologic Improvement (HI) is observed.

Drug: cenersenDrug: Dexamethasone

Group 2

ACTIVE COMPARATOR

cenersen IV infusion 0.6 mg/kg/day (0.2 mg/kg/h x 3 h x 4 days) for 6 cycles Administration of study drug will be over 4 consecutive days at the outset of each 28 day cycle for a total of 6 cycles for each patient. Dexamethasone 20 mg po weekly can be added after week 16 if stable disease but no HI is observed.

Drug: cenersenDrug: Dexamethasone

Interventions

cenersenDrug

cenersen is a phosphorothioate antisense oligonucleotide that down-regulates the production of both wild-type and mutant p53, and has a RNase H-dependent mechanism of action

Also known as: Ol(1)p53, EL625, Aezea

Group 1Group 2Group 3

DexamethasoneDrug

Eligibility Criteria

Age18 Years - 95 Years

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

Must have histologically or cytologically confirmed diagnosis of MDS according to WHO classification that meets IPSS low to intermediate-1 risk criteria.
For patients with del(5q) MDS, documented del(5q) MDS by metaphase cytogenetics or FISH analysis with up to 1 additional cytogenetic abnormality other than 1 involving chromosome 7 or chromosome 17.

Sponsors & Collaborators

Eleos, Inc.
Industrylead
H. Lee Moffitt Cancer Center and Research Institute
Othercollaborator

Study Sites (1)

H. Lee Moffitt Cancer Center & Research Institute

Tampa, Florida, 33612, United States

Related Publications (1)

Caceres G, McGraw K, Yip BH, Pellagatti A, Johnson J, Zhang L, Liu K, Zhang LM, Fulp WJ, Lee JH, Al Ali NH, Basiorka A, Smith LJ, Daugherty FJ, Littleton N, Wells RA, Sokol L, Wei S, Komrokji RS, Boultwood J, List AF. TP53 suppression promotes erythropoiesis in del(5q) MDS, suggesting a targeted therapeutic strategy in lenalidomide-resistant patients. Proc Natl Acad Sci U S A. 2013 Oct 1;110(40):16127-32. doi: 10.1073/pnas.1311055110. Epub 2013 Sep 16.
PMID: 24043769BACKGROUND

MeSH Terms

Conditions

Myelodysplastic SyndromesAnemia

Interventions

cenersenOL(1)p53DexamethasoneCalcium Dobesilate

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

PregnadienetriolsPregnadienesPregnanesSteroids

Study Officials

Rami Komrokji, MD
H. Lee Moffitt Cancer Center and Research Institute
PRINCIPAL INVESTIGATOR

Enrollment

9 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1
Allocation: NON RANDOMIZED
Masking: NONE
Purpose: TREATMENT
Intervention Model: PARALLEL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

September 11, 2014

First Posted

September 17, 2014

Study Start

September 2014

Date type not reported

This date has not been verified as actual or estimated.

You may not qualify if:

Receiving MDS treatment except blood transfusion and/or iron chelation within 4 weeks prior to entering study or no recovery from adverse events due to agents administered more than 4 weeks earlier.
no current or prior use of investigational agents within 4 weeks of study entry.
Known history of malignancy diagnosed within 2 years other than non-melanoma skin cancer.
History of allergic reactions attributed to compounds of similar chemical or biologic composition to cenersen.
exclude use of acetaminophen or acetaminophen-containing medications from 1 day before to 1 day after completion of treatment. The active metabolite of acetaminophen, N-acetyl-p-benzoquinone imine (NAPQI), is known to block effects of cenersen \& use of acetaminophen during treatment with study regimen has been associated with a failure to achieve a response in a past clinical trial of cenersen.
Uncontrolled intercurrent illness that would limit compliance with study requirements.
Pregnant women are excluded from this study. Breastfeeding should be discontinued if the mother is treated with cenersen
HIV-positive patients on combination antiretroviral therapy are ineligible because of unknown potential for interactions with cenersen.
Any condition, including presence of laboratory abnormalities, which places subject at unacceptable risk if s/he were to participate in study or confounds ability to interpret data from study according to investigator assessment.
Therapy related MDS.
Clinically significant anemia according to investigator's assessment due to factors such as iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis or gastrointestinal bleeding.
Received hematopoietic growth factors within specified limits prior to treatment (2 weeks for epoetin alpha (Procrit) \& 4 weeks for darbepoetin alpha (Aranesp)).
Active hepatitis B or C or other active liver disease.
Chronic use (\>2 weeks) of greater than physiologic doses of corticosteroid agent (dose equivalent to ≥10mg of prednisone) within 28 days of 1st day of study drug treatment \& during treatment

Study Stopped

A Study of Aezea® (Cenersen) in Transfusion Dependent Anemia Associated With Myelodysplastic Syndrome (MDS)

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (2)

Study Arms (3)

Group 1

Group 2

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

Group 3

You may not qualify if:

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (2)

Study Arms (3)

Group 1

Group 2

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

Group 3

You may not qualify if:

Source record

Locations