Not Yet RecruitingPhase 4

A Study of Romiplostim for the Treatment of Refractory Transfusion-dependent NSAA

China40 participantsStarted 2024-08-02

Plain-language summary

Efficacy and safety of Romiplostim in the treatment of refractory transfusion-dependent NSAA.

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Age ≥18 years, male or female.
✓. Diagnosis consistent with refractory transfusion dependence NSAA defines refractory as patients who have failed to respond to at least 6 months of prior first-line treatment with adequate doses of cyclosporine (3-5 mg/kg) and who have been treated with an adequate dose of at least one povidone for 3 months. Definition of transfusion dependence: at least 1 component transfusion on average every 8 weeks and duration of transfusion dependence ≥ 4 months.
✓. Satisfy at least one of the following conditions at the time of enrolment: haemoglobin \<90 g/L, platelets \<30×10\^9/L, neutrophils \<1.0×10\^9/L.
✓. Agree to sign the consent form.
✓. An Eastern Cooperative Oncology Group (ECOG) score of 0-2.

✕. Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS).
✕. Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML).
✕. PNH clones ≥50%.
✕. Haematopoietic stem cell transplantation (HSCT) prior to enrolment.
✕. Prior treatment with ATG.
✕. Infection or haemorrhage uncontrolled by standard therapy.
✕. Allergy to roprostin.
✕. Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.

Overall response rate (ORR)

Timeframe: 3 months

Overall response rate (ORR)

Timeframe: 6 months

CRR

Timeframe: 3 months

CRR

Timeframe: 6 months

NCT IDNCT06535685

SponsorPeking Union Medical College Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-03-30

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Age ≥18 years, male or female.
✓. Diagnosis consistent with refractory transfusion dependence NSAA defines refractory as patients who have failed to respond to at least 6 months of prior first-line treatment with adequate doses of cyclosporine (3-5 mg/kg) and who have been treated with an adequate dose of at least one povidone for 3 months. Definition of transfusion dependence: at least 1 component transfusion on average every 8 weeks and duration of transfusion dependence ≥ 4 months.
✓. Satisfy at least one of the following conditions at the time of enrolment: haemoglobin \<90 g/L, platelets \<30×10\^9/L, neutrophils \<1.0×10\^9/L.
✓. Agree to sign the consent form.
✓. An Eastern Cooperative Oncology Group (ECOG) score of 0-2.

✕. Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS).
✕. Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML).
✕. PNH clones ≥50%.
✕. Haematopoietic stem cell transplantation (HSCT) prior to enrolment.
✕. Prior treatment with ATG.
✕. Infection or haemorrhage uncontrolled by standard therapy.
✕. Allergy to roprostin.
✕. Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.