Study in Hematological Malignancies

General information

Section to complete general information about the trial: scientific and public title, protocol identifiers, sponsors and Source(s) of Monetary or Material Support.

Acronym of Public Title:

EHPMA Study

Scientific title:

Exploratory study of CIGB-300 treatment in refractory or relapsing acute leukemias, elderly acute myeloid leukemia and myelodysplastic syndrome with excess blasts.

Acronym of Scientific Title:

EHPMA Study

Secondary indentifying numbers:

IG/CIGB-300I/LAR/1101

Issuing authority of the secondary identifying numbers:

Center for Genetic Engineering and Biotechnology (CIGB).

Source(s) of monetary or material support:

HeberBiotec S.A. CHEMO Group

Authorization for beginning

Section to complete information about the regulatory approval of clinical trial: regulatory agency name, approval date and reference number in the agency.

Regulatory instance to authorize the initiation of the study:

Center for State Control of Drugs, Medical Devices and Equipment (CECMED)

Authorization date :

16/11/2012

Reference number:

05.013.12.B

Principal investigator

Section to complete information about Email address, telephone number and postal address of the Principal Investigator.

First name:

Aramis

Last name:

Nuñez Quintana

Medical Specialty :

2nd Degree Haematology Specialist

Affiliation:

“Hermanos Ameijeiras” Hospital

Postal address:

San Lazaro No. 701 esquina a Belascoaín.

City:

La Habana

País:

Cuba

Zip Code:

10300

Telephone:

+53-78761460

Email address:

anunez@infomed.sld.cu [1]

Clinical sites to participate

Section to complete the data related to the clinical sites involved in the trial: site and responsible investigator for every site.

Countries of recruitment:

Cuba

Clinical sites:

“Gustavo Aldereguia Lima” Hospital, Cienfuegos, Julio Fernandez Aguila, MD, 2nd Degree Haematology Specialist.

“Arnaldo Milian Castro” Hospital, Villa Clara, Rosa Oliday Rios Jimenez, MD, 1st Degree Haematology Specialist.

“Carlos J. Finlay” Hospital, La Habana, Viviana Cristo Perez, MD, 1st Degree Haematology Specialist.

Institute of Hematology and Immunology, La Habana, Carlos Hernandez Padron, MD, 2nd Degree Haematology Specialist.

Research ethics committees:

''Hermanos Ameijerias'' Hospital, June 4, 2012.

“Gustavo Aldereguia” Hospital, May 2, 2012.

“Arnaldo Milian” Hospital, May 25, 2012.

“Carlos J. Finlay”, Hospital, June 13, 2012.

Institute of Hematology and Immunology, April 26, 2012.

Recruitment status

Section to complete information about the recruitment status and the date of first enrolment subject

Recruitment status:

Complete

Date of first enrollment:

24/12/2012

Health condition and Intervention

Section to complete information about the primary medical condition(s) or problem(s) studied and, a characteristics of the intervention(s).

Health condition(s) or Problem(s) studied:

Relapsed or refractory acute leukemia Elderly acute myeloid leukemia (patients >60) Myelodysplastic Syndrome (MDS)

Intervention(s):

Study group (CIGB-300): 5 cycles of an intravenous infusion over 15 minutes daily for three consecutive days every one, with four days of rest between cycles. Dose level by cycle: Cycle 1 (1.6 mg/kg), Cycle 2 (3.2 mg/kg), Cycle 3 (5.3 mg/kg), Cycle 4 (8.0 mg/kg) and Cycle 5 (12.8 mg/kg).

Outcomes and Timepoint

Section to complete information about primary and secondary outcomes including. It includes the metric or method of measurement used and, the time point for every outcome.

Primary outcome(s):

Serious Adverse Events (Occurrence of EA (Yes, No). EA Description (name of event). EA intensity (mild, moderate, severe). Measuring time: post administration at 15 minutes, 30 minutes and 60 minutes, and at any other time when an event is present.

Key secondary outcomes:

Clinical-hematologic response (Complete remission, Partial remission, No response). Measuring time: weeks 6 and 24. Count of blasts in peripheral blood (Responders, Partial responders, No responders). Measuring time: weeks 1-6 and 24. Relapse-Free Survival (Defined only for patients achieving Complete remission: Time measured from the date of achievement of a remission until the date of relapse or death from any cause; patients not known to have relapsed or died at last follow-up are censored on the date they were last examined). Measuring time: 6 months. Event-Free Survival (Time measured from the date of treatment began to the date of treatment failure, or relapse or death from any cause; patients not known to have any of these events are censored on the date they were last examined). Measuring time: 6 months. Survival (time measured from the date of treatment began to the date of death from any cause; patients not known to have died at last follow-up are censored on the date they were last known to be alive). Measuring time: 6 months. Transfusion requirements (Number of transfusion units). Measuring time: week 24. Immunophenotyping (percent of CD3, CD13, CD19 CD33, CD41, CD14, CD15, CD34 measured by flow cytometry). Measuring time: weeks 0 and 6. Differentiation of progenitor cells (Percentage of cell lines of lymphoid and myeloid). Measuring time: weeks 0 and 6. Changes in detectable cytogenetic abnormalities (Quantification of the chromosomal abnormality in the medullary metaphase). Measuring time: weeks 0 and 6. Genetic expression about mechanism of action of CIGB-300 and malignancies disease (Yes or no). Measuring time: weeks 0 and 6.

Selection criterias

Section to complete information about the inclusion and exclusion criteria for participant selection, including age and gender.

Gender:

Male/Female

Minimum age:

18 years

Maximum age:

None

Inclusion criteria:

1) Diagnosis of refractory AL after two cycles myeloablative standard chemotherapy, AL in early relapse (within 12 months) or second relapse or post-transplantation, the elderly AML not candidates for standard induction chemotherapy (3 +7), MDS with RAEB-1 and RAEB-2. 2) Patients no eligible for BMT at the time of inclusion. 3) Aged ≥18 years. 4) ECOG ≤ 3. 5) Life expectancy > 5 weeks. 6) More than two weeks since prior therapy, including QT, or biological therapies. 7) Patients of childbearing potential must use an effective contraceptive method. 8) Express written request of the patient.

Exclusion criteria:

1) AML promyelocytic variant (M3). 2) Pregnancy, postpartum or breastfeeding. 3) Any type of active infection requiring specific treatment. 4) ALT or AST > 5 times the normal reference range. 5) Decompensated chronic diseases (hypertension, diabetes mellitus, chronic renal failure, heart failure, ischemic heart disease or other symptomatic cardiovascular disease, epilepsy, severe mental depression). 6) Medical history as severe allergic urticaria, atopic dermatitis, persistent bronchitis or bronchial asthma or any ingredient in the formulations studied. 7) Obvious mental disability or other limitation that prevents the patient sign the consent or hinder study assessments.

Type of population:

Adults

Type of participant:

Patients

Study design

Section to complete information about the characteristics of the study design.

Type study:

Interventional

Purpose:

Treatment

Allocation:

N/A: single arm study

Masking:

Open

Control group:

Uncontrolled

Study design:

Single group

Phase:

1-2

Target sample size:

Contact for public queries

Section to complete information about Email address, telephone number and postal address of the contact who will respond to general queries, including information about current recruitment status

First Name:

Yanelda

Last Name:

Garcia Vega

Specialty:

Bachelor in Biochemistry, Master in Clinical Laboratory Sciences.

Affiliation:

Center for Genetic Engineering and Biotechnology.

Postal Address:

Ave. 31 e/ 158 y 190 Cubanacan, Playa.

City:

Havana

País:

Cuba

Zip Code:

6162

Telephone:

+537-208-7377 (53-7)-2087377

Email :

yanelda.garcia@cigb.edu.cu [2]

Contact for scientific queries

Section to complete information about Email address, telephone number and postal address of the contact who will respond to scientific queries.

First Name:

Yanelda

Last Name:

Garcia Vega

Specialty:

Bachelor in Biochemistry, Master in Clinical Laboratory Sciences.

Affiliation:

Center for Genetic Engineering and Biotechnology.

Postal Address:

Ave. 31 e/ 158 y 190 Cubanacán, Playa.

City:

Havana

País:

Cuba

Zip Code:

6162

Telephone:

+537-208-7377

Email :

yanelda.garcia@cigb.edu.cu [2]

Registration and Update

Section to complete information about the name of Primary Registry, date of registration and the unique ID number assigned by the registry (RPCEC).

Primary registry:

RPCEC

Unique ID number:

RPCEC00000190

Date of Registration in Primary Registry:

14/11/2014

Record Verification Date:

2014/11/14

Next update date:

2015/11/14

Link to the spanish version:

Click here [3]