Clinical study of recombinant human thrombopoietin (rhTPO) in platelet engraftment after umbilical cord blood transplantation in lysosomal storage diseases

Status

Recruiting

Phases

Phase 4

Study type

Interventional

Source

ChiCTR

Registry ID

ChiCTR2100047583

Enrollment

Unknown

Registered

2021-06-20

Start date

2021-06-21

Completion date

Unknown

Last updated

2022-02-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

lysosomal storage diseases

Interventions

Experimental group:Subcutaneous injection of recombinant human thrombopoietin (300U/kg)

Eligibility

Sex/Gender

All

Age

No minimum to 18 Years

Inclusion criteria

Inclusion criteria: 1. Aged 0-18 years; 2. Any lysosomal storage disease identified by clinical enzyme assay, DNA analysis, etc.; 3. Patients who are about to receive hematopoietic stem cell cord blood transplantation; 4. Kidney: Serum creatinine = 60ml/min, without dialysis; 5. Heart: Left ventricular ejection fraction must be >= 40% at rest, or fractional shortening >= 26%; 6. Liver: Bilirubin <= 2 times the upper limit of normal (ULN), ALT and AST <= 2 times the upper limit.

Exclusion criteria

Exclusion criteria: 1. Inability to tolerate transplantation procedures, including conditioning regimens; 2. Congestive heart failure caused by congenital heart disease; 3. Uncontrolled bacterial, viral or fungal infection; 4. Evidence of human immunodeficiency virus (HIV) infection or known HIV-positive serology.

Design outcomes

Primary

Measure	Time frame
Platelet quantity;	—

Countries

China

Contacts

Public ContactLiu Rong

liurong201305@sina.com+86 13601123876

Outcome results

None listed

Source: ChiCTR (via WHO ICTRP) · Data processed: Feb 4, 2026