Outcome of neurocognitive function after allogeneic hematopoietic stem cell transplantation in type II mucopolysaccharidosis

Status

Recruiting

Phases

Early Phase 1

Study type

Interventional

Source

ChiCTR

Registry ID

ChiCTR1900026503

Enrollment

Unknown

Registered

2019-10-12

Start date

2020-10-01

Completion date

Unknown

Last updated

2020-09-14

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

Conditions

mucopolysaccharidosis

Interventions

HSCT group:allogeneic hematopoietic stem cell transplantation

Natural history group:Symptom management

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1. Aged 0-8 years old boys, MPS type II is X-linked inherit disease. It is found that a few female carriers also present some clinical symptoms. This study only focus on male patients. 2. Diagnosed as mucopolysaccharidosis type II. MPS Type II diagnostic criteria (1-3 must be included): 1) Urine screening shows that DS (dermatan sulfate) and HS (heparin sulfate) are positive; 2) Serum iduronic acid sulfatase activity is lower than the normal value of 10%; 3) Multiple sulfatase deficiency is excluded; 4) Genetic tests if available. 3. Patients who voluntarily participate and sign the informed consent form.

Exclusion criteria

Exclusion criteria: 1. With a neurological disease not caused by MPS; 2. Unable to complete the intelligence test; 3. The donor is a carrier.

Design outcomes

Primary

Measure	Time frame
Chronological age;	—

Countries

China

Contacts

Public ContactJianmin Wang

Shanghai Children's Medical Center, Shanghai Jiao Tong University school of medicine

wangjianmin@scmc.com.cn+86 021-38626161

Outcome results

None listed

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