Study in Hematological Malignancies

• Register: RPCEC
• Last refreshed on: 29 April 2024
• Main ID: RPCEC00000190
• Date of registration: 14/11/2014
• Prospective Registration: No
• Primary sponsor: Center for Genetic Engineering and Biotechnology (CIGB).
• Public title: Study in Hematological Malignancies
• Scientific title: Exploratory study of CIGB-300 treatment in refractory or relapsing acute leukemias, elderly acute myeloid leukemia and myelodysplastic syndrome with excess blasts. - EHPMA Study
• Date of first enrolment: 24/12/2012
• Target sample size: 10
• Recruitment status: Complete
• URL: https://rpcec.sld.cu/en/trials/RPCEC00000190-En
• Study type: Interventional
• Study design: Allocation: N/A: single arm study. Masking: Open. Control group: Uncontrolled. Assignment: Single group. Purpose: Treatment
• Phase: 1-2

Countries of recruitment

Cuba

Contacts

• Name: Yanelda Garcia Vega
• Affiliation: Center for Genetic Engineering and Biotechnology.

• Name: Yanelda Garcia Vega
• Affiliation: Center for Genetic Engineering and Biotechnology.

Key inclusion & exclusion criteria

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.

Age minimum: 18 years
Age maximum: None
Gender: Male/Female

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).

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.

Secondary Outcome(s)

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.

Secondary ID(s)

IG/CIGB-300I/LAR/1101

Source(s) of Monetary Support

HeberBiotec S.A. CHEMO Group

Secondary Sponsor(s)

Cuban Ministry of Public Health (MINSAP)