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Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Childhood and Adolescent Essential Thrombocythemia

Study Purpose

Objectives: To compare the efficacy and safety in childhood and adolescent patients (<20 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs.#46; Interferon Alfa. Study Design: A prospective, open-label, nonrandomized, single-center clinical trial

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages N/A - 19 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - <20 years old.
  • - Male or Female.
  • - Diagnosis of essential thrombocythemia according to the 2016 WHO criteria.
  • - Platelet count ≥ 450 × 109 / L for more than 6 months(If the patient has JAK2 V617F, CALR or MPL gene mutation, the history may be less than 6 months) - Platelet count ≥ 1000 × 109 / L or other therapeutic indications at screening.
  • - The guardians has provided written informed consent prior to enrollment.

Exclusion Criteria:

  • - Known to meet the criteria for primary myelofibrosis or polycythemia vera by 2016 WHO criteria.
  • - Presence of any life-threatening co-morbidity.
  • - Secondary thrombocytosis.
  • - Familial thrombocytosis.
  • - Resistance, or intolerance, or any contraindications to interferon.
  • - Interferon is used in the past 1 month before enrollment.
  • - Patients with previous or present thrombosis or active bleeding.
  • - WBC<4× 109 / L.
  • - HGB<110g/L.
  • - Poor control of thyroid dysfunction.
  • - Patients with a prior malignancy within the last 3 years.
  • - Patients with severe cardiac or pulmonary dysfunction.
  • - Severe renal damage (creatinine clearance < 30 ml / min) - Severe liver dysfunction (ALT or AST > 2.5×ULN) - Patients diagnosed as diabetes with poor control.
  • - Patients with hepatitis B virus, hepatitis C virus replication or HIV infection.
  • - Patients with a history of drug / alcohol abuse (within 2 years before the study) - Patients that have participated in other experimental researches within one month before enrollment.
  • - History of psychiatric disorder.
- Any other circumstances that the investigator considers that the patient is not suitable to participate in the trial

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT04226950
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Institute of Hematology & Blood Diseases Hospital, China
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Lei Zhang, MD
Principal Investigator Affiliation Institute of Hematology & Blood Diseases Hospital, China
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other
Overall Status Recruiting
Countries China
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Essential Thrombocytopenia
Additional Details

This is a prospective, open-label, nonrandomized, single-center clinical trial between Interferon Alfa and Pegylated Interferon Alfa-2b in childhood and adolescent essential thrombocythemia (<20 years). Patients will be divided into the following two treatment groups: 1. Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers; 2. Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2). The current drug therapies and possible risks of Pegylated Interferon Alfa-2b and Interferon Alfa in the treatment of childhood and adolescent essential thrombocythemia will be fully introduced to the guardians (childhood patients) or patients (adolescent patients) by the researchers. Then the patients will be divided into one of the two groups according to the guardians' (childhood patients) or patients' (adolescent patients) will. The dosage will be adjusted according to the results of laboratory examinations and patient tolerance. The patient will be transferred to the other group if intolerance or resistance occurs.

Arms & Interventions

Arms

Active Comparator: Recombinant Interferon Alpha

Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers.

Experimental: Pegylated Interferon Alfa-2b

Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2).

Interventions

Drug: - Recombinant Interferon Alpha

Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers;

Drug: - Pegylated interferon alfa-2b

Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2).

Contact a Trial Team

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International Sites

Tianjin, Tianjin, China

Status

Recruiting

Address

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin, 300020

Site Contact

Lei Zhang, MD

[email protected]

+862223909240