Biomodulin T Intramuscular for Thymus Hypoplasia in Children. Phase III

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.

Scientific title:

Evaluation of the efficacy and safety of Biomodulin T in pediatric patients with thymic hypoplasia associated or not with cellular immunodeficiency. Clinical Trial Phase III. Version 0.1.

Secondary indentifying numbers:

GEC2017BMT018

Issuing authority of the secondary identifying numbers:

Center National of Bioproducts (BioCen)

Source(s) of monetary or material support:

Center National of Bioproducts (BioCen)Ministry of Public Health (MINSAP)

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 :

10/05/2018

Reference number:

672/05.012.17.BNV

Principal investigator

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

First name:

Odalis

Midle name:

Maria

Last name:

de la Guardia Peña

Medical Specialty :

Specialist of 2nd. Degree in Immunology

Affiliation:

Institute of Hematology and Immunology (IHI)

Postal address:

19th between 8 and 10, Vedado

City:

Plaza de la Revolución, La Habana

País:

Cuba

Zip Code:

10800 Box 8070

Telephone:

+53-7846115

Email address:

odalism@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:

Havana, "William Soler" Pediatric Hospital, MD. Concepción Insua Arregui, Specialist of 2nd Degree in Imaging

Research ethics committees:

Institute of Hematology and Immunology, January 25, 2019

"William Soler" Pediatric Hospital, January 25, 2019

Recruitment status

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

Recruitment status:

Recruiting

Date of first enrollment:

03/12/2018

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:

Thymus hypoplasia

Health condition(s) code:

Thymus Gland /abnormalities

Health condition keyword:

Thymic Hypoplasia

Intervention(s):

Patients will receive treatment with Biomodulin T via IM. The doses of one, two or three bulb per week will be differentiated according to the degree of Hypoplasia: Severe Thymic Hypoplasia (STH), Moderate (MTH) and Light (HTL): 1. Scheme 1. Subgroup I without cellular immunodeficiency ) Week 1 to 4: 1 bulb (3 mg of timic fraction) IM, biweekly (3 mg) (First cycle) Week 5 to 8: Rest Week 9 to 12: 1 bulb (3 mg) IM, once a week (3 mg) (Second cycle) Week 13 to 16: Rest Week 17 to 24: 1 bulb (3 mg) IM, once a week (3 mg) (Third cycle) Week 25 to 32: Rest 2. Scheme 2. Subgroup I without cell immunodeficiency (HTM): Week 1 to 4: 1 bulb (3 mg) IM, once a week (3 mg) (first cycle) Week 5 to 8: Rest Week 9 to 12: 1 bulb (3 mg) IM, once a week (3 mg) (Second cycle) Week 13 to 16: Rest Week 17 to 24: 1 bulb IM, once a week (3 mg) (Third cycle) Week 25 to 32: Rest 3. Scheme 3. Subgroup II with cell immunodeficiency (HTS): Week 1 to 4: 1 bulb (3 mg) IM, 3 times per week (9 mg) (First cycle) Week 5 to 8: Rest Week 9 to 12: 1 bulb (3 mg) IM, biweekly (6 mg) (Second cycle) Week 13 to 16: Rest Week 17 to 24: 1 bulb (3 mg) IM, biweekly (6 mg) (Third cycle) Week 25 to 32: Rest 4. Scheme 4. Subgroup II with cell immunodeficiency (HTM): Week 1 to 4: 1 bulb (3 mg) IM, biweekly (6 mg) (First cycle) Week 5 to 8: Rest Week 9 to 12: 1 bulb (3 mg) IM, once a week (3 mg) (Second cycle) Week 13 To 16: Rest Week 17 to 24: 1 bulb (3 mg) IM, once a week (3 mg) (Third cycle) Week 25 to 32: Rest 5. Scheme 5. Subgroup II with cellular immunodeficiency (HTL): Week 1 to 4: 1 bulb (3 mg) IM, once a week (3 mg) (First cycle) Week 5 to 8: Rest Week 9 to 12: 1 bulb (3 mg) IM, once a week (3 mg) (Second cycle) Week 13 to 16: Rest Week 17 to 24: 1 bulb (3 mg) IM, once a week (3 mg) (Third cycle) Week 25 to 32: Rest The first and second cycle of treatment will be administered in all cases. The third cycle will be administered if after the ultrasonographic evaluation of the thymus, it is shown that the area of this, still does not reach its normal value.

Intervention code:

Immunologic Factors

Immunomodulation

Injections, Intramuscular

Intervention keyword:

Biomodulin T

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):

1. Thymic Area (Ultrasound measurement of thymus in mm2). Measurement time: At baseline, 16 weeks and 32 weeks if you complete the third cycle. 2. Degree of thymic hypoplasia (Classification of degree of thymic hypoplasia according to scale in light, moderate or severe). Measurement time: At baseline, 16 weeks and 32 weeks if patient receives the third cycle.

Key secondary outcomes:

1. Infections (Number of infections in the period). Measurement time: At baseline, 16 weeks and 32 weeks if you complete the third cycle. 2. Nutritional Status (Body Mass Index-BMI). Measurement time: At baseline, 16 weeks and 32 weeks if you complete the third cycle. 3. Lymphocyte Subpopulations (Percent of Lymphocyte Markers by Flow Cytometry- CD3+/CD4+, CD3+/CD8+, CD19+, CD56+). Measurement time: At baseline, 16 weeks and 32 weeks if you complete the third cycle . 4. Quality of life (PedsQL Version 4.0 in Spanish Quality of Life questionnaire). Measurement time: At baseline, 16 weeks and 32 weeks if you complete the third cycle. 5. Adverse events-AE (Occurrence of some AE (Yes, No), Description of the AE (Name of adverse event),According to available previous information (Unexpected, Expected), According to its localization the expected AE (World Allergy Organization Criterion: local and systemic AE (0= Nonspecific Symptoms, I= Mild systemic reactions, II= Moderate systemic reactions, III= Severe (nonlife-threatening) systemic reactions, IV= Anaphylactic shock)), Duration of the AE (Time between the start and the end of the AE), Graveness of the AE (Serious, No serious), Attitude regarding the treatment in study (Without changes, Dose modification, Temporary interruption, Definitive interruption), Result of the AE (Recovered, Improved, Persists, Sequels), Causality Relationship of the AE (1.Very likely, 2.Likely, 3.Possible, 4.Unlikely, 5.Not related, 6.Not evaluated)). Measurement time: weekly during the first month and, monthly until month 12.

Selection criterias

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

Gender:

Male/Female

Minimum age:

1 year

Maximum age:

5 years

Inclusion criteria:

1. Patients with radiological confirmation by Thymus Ultrasound of Severe, Moderate or Light Thymic Hypoplasia with or without clinical manifestations of immunodeficiencies 2. Both sexes between the ages of one and five years. 3. That they are virgins of immunostimulating or immunosuppressive treatment, at least during the previous 6 months. 4. Voluntariness of the patient's parent or guardian and giving written informed consent.

Exclusion criteria:

1. Patients with a history of severe allergic reactions and / or severe generalized eczema. 2. Patients with any other type of non-cellular immunodeficiency with exception of IgA partial deficiency. 3. Di George Syndrome. 4. Patients who have been treated with steroids in the previous 45 days. 5. Patients with a diagnosis of tumor or autoimmune disease of any kind. 6. Patients with chronic diseases of the type Diabetes Mellitus Type I or any other endocrinopathy. 7. Patients with severe congenital malformations that cause disability or require medical or surgical treatment. 8. Patients with hemoglobin lower than 9 g/L.

Type of population:

Children

Type of participant:

Patients

Study design

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

Type study:

Interventional

Purpose:

Treatment

Allocation:

Non-randomized controlled trial

Masking:

Open

Control group:

Uncontrolled

Study design:

Parallel

Phase:

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:

Odalis

Middle Name:

María

Last Name:

de la Guardia Peña

Specialty:

Specialist of Second Degree in Immunology

Affiliation:

Institute of Hematology and Immunology

Postal Address:

19th between 8 and 10, Vedado

City:

Plaza de la Revolución, Cuba

País:

Cuba

Zip Code:

13050, Box 6048

Telephone:

+53-7846115

Email :

odalism@infomed.sld.cu [1]

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:

Mary

Middle Name:

Carmen

Last Name:

Reyes Zamora

Specialty:

Specialist of First Degree in Immunology, Master in Clinical Trials

Affiliation:

Department of Clinical Trials, National Center of Bioproducts (BioCen)

Postal Address:

Carretera de Beltran Km 1 ½ S/N

City:

Bejucal, Mayabeque

País:

Cuba

Zip Code:

13050, Box 6048

Telephone:

+53047-066-82201 al 07, Ext.: 1145 and 1147

Email :

mcarmen.reyes@biocen.cu [2]

About study completion

Section to complete the data related to the study completion.

Final enrolment number:

30/12/2019

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:

RPCEC00000247

Date of Registration in Primary Registry:

14/08/2017

Record Verification Date:

2020/04/15

Next update date:

2021/04/15

Link to the spanish version:

Click here [3]