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

NCT04226950 | Recruiting Phase 2

• 1 other identifier: IIT2019001
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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. Interferon Alfa. Study Design: A prospective, open-label, nonrandomized, single-center clinical trial

Conditions

Essential Thrombocytopenia

Keywords

Essential Thrombocythemia; Childhood; Interferon Alfa; Pegylated Interferon Alfa-2b; Adolescent

Outcome Measures

Primary Outcomes (1)

• Change in platelet count

  Proportion of subjects with a continuous platelet count ≤600×109/L or decrease ≥50% (\<1000×109/L ) (at least 12 weeks) from baseline during treatment will be evaluated.

  From the start of study treatment (Day 1) up to the end of month 12

Secondary Outcomes (11)

• The complete hematologic response rates

  From the start of study treatment (Day 1) up to the end of month 12

• Time to response in platelet count

  From the start of study treatment (Day 1) up to the end of month 12

• Impact of therapy on key biomarkers

  From the start of study treatment (Day 1) up to the end of month 12

• Incidence of major cardiovascular and thrombotic events

  From the start of study treatment (Day 1) up to the end of month 12

• Incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation.

  From the start of study treatment (Day 1) up to the end of month 12

Study Arms (2)

Recombinant Interferon Alpha

ACTIVE COMPARATOR

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: Recombinant Interferon Alpha

Pegylated Interferon Alfa-2b

EXPERIMENTAL

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).

Drug: Pegylated interferon alfa-2b

Interventions

Recombinant Interferon Alpha DRUG

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;

Recombinant Interferon Alpha

Pegylated interferon alfa-2b DRUG

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).

Pegylated Interferon Alfa-2b

Eligibility Criteria

• Age: Up to 19 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• \<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

You may not qualify if:

• 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

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Thrombocythemia, Essential

Interventions

peginterferon alfa-2b

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombocytosis; Blood Platelet Disorders; Myeloproliferative Disorders; Bone Marrow Diseases; Hemorrhagic Disorders

Study Officials

• Lei Zhang, MD

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

• Rongfeng Fu

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rongfeng Fu, MD

CONTACT

+862223909009; furongfeng@ihcams.ac.cn

Lei Zhang, MD

CONTACT

+862223909240; zhanglei1@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 10, 2019
• First Posted: January 13, 2020
• Study Start: January 10, 2020
• Primary Completion: October 20, 2024
• Study Completion: November 20, 2024
• Last Updated: July 3, 2024

Record last verified: 2024-04

Locations

CN (1)