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NCT Number: NCT06613880

Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

This prospective, single center, single-arm study aims to evaluate the efficacy and safety of anti-human T lymphocyte porcine immunoglobulin (p-ALG) in combination with cyclosporine and Romiplostim in treating severe aplastic anemia (SAA). It will include 48 patients with newly diagnosed SAA.

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Key information

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Regenerative Medicine Center and Red Blood Cell Disorders Center

Tianjin, Tianjin Municipality, China

Location status: Recruiting

Location contact

Lele Zhang, PhD

CONTACT

[email protected]

15811139278

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥ 12 years
  • Diagnosis of severe or very severe aplastic anemia
  • Patients are unconditionally or unacceptable undergoing allogeneic hematopoietic stem cell transplantation
  • ECOG performance status ≤2
  • Willing and able to comply with the requirements for this study and written informed consent.

Exclusion criteria

  • Received > 4 weeks of TPO-RA drug before treatment
  • Received > 4 weeks of immunosuppressive therapy before treatment
  • History of thromboembolic disease
  • Intolerance to Romiplostim N01 or cyclosporine
  • Allergy to ALG
  • Presence of uncontrolled active infection
  • Presence of uncontrolled hypertension (≥140/90mmHg), diabetes mellitus (fasting blood glucose≥ 7.0 mmol/L or random blood glucose ≥ 11.1 mmol/L)
  • Abnormal liver and kidney function: Aspartate Transaminase (AST) or Alanine Transaminase (ALT) > 3 ULN, creatinine ≥ 2.5 ULN
  • History of chemoradiotherapy for malignant solid tumors
  • History of other systemic serious illnesses
  • Females who are pregnant/lactating or need pregnancy
  • Patients considered to be ineligible for the study by the investigator for reasons other than the above

Treatment and study plan

standard IST combined with Romiplostim N01

Drug

Romiplostim N01 is a class of TPO receptor agonists. Its TPO peptide can bind to TPO receptors on megakaryocyte precursors in the bone marrow and activate them, triggering a series of intracellular signalling pathways, including JAK2/STAT5, PI3K/Akt, MEK/ERK, and p38, which ultimately lead to gene transcription and increased megakaryocyte proliferation and differentiation.

Primary outcomes

  1. Overall response rate

    Time frame: Within 27 weeks post treatment

    Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).

Secondary outcomes

  1. Overall response rate

    Time frame: Within 3 months post treatment

    Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).

  2. Time to hematological response

    Time frame: Within 27 weeks post treatment

    Duration time was calculated from enrollment to achieve hematological response.

  3. Incidence of adverse events

    Time frame: Within 27 weeks post treatment

    Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event

Study contacts

Contact information is provided by the study sponsor or research team.

Lele Zhang, PhD

CONTACT

[email protected]

15811139278

Meili Ge, PhD

CONTACT

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Collaborators

  • Qilu Pharmaceutical Co., Ltd.

Registry information

Official study title

A Prospective, Single Center, Single-arm Study on the Efficacy and Safety of Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Sep 26, 2024
Registry last updated
Dec 4, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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