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A study for efficacy of pantethine in the treatment of pantothenate kinase-associated neurodegeneration

A study for efficacy of pantethine in the treatment of pantothenate kinase-associated neurodegeneration

Status
Recruiting
Phases
Unknown
Study type
Observational
Source
ChiCTR
Registry ID
ChiCTR1900021076
Enrollment
Unknown
Registered
2019-01-27
Start date
2018-09-04
Completion date
Unknown
Last updated
2019-01-28

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

Conditions

pantothenate kinase-associated neurodegeneration

Interventions

Experimental group:Standardized basal treatment + pantethine
Control group:Standardized basal treatment

Sponsors

Department of Pediatrics, Peking University First Hospital
Lead Sponsor

Eligibility

Sex/Gender
All
Age
No minimum to 16 Years

Inclusion criteria

Inclusion criteria: Children diagnosed as PKAN with following characteristics: 1. mutations in the pantothenate kinase 2 (PANK2) gene; 2. progressive dystonia; 3. typical MRI patterneye-of-the-tiger; 4. Aged =16 years old.

Exclusion criteria

Exclusion criteria: 1. Patients with severe renal dysfunction (GFR<60ml/min); 2. Patients with severe hepatic dysfunction; 3. Patients with cardiovascular diseases; 4. Patients with hematopoietic system diseases (Hb<60g/L, PLT<50×10^9/L, N<1×10^9/L).

Design outcomes

Primary

MeasureTime frame
UPDRS score;FMscore;—

Countries

China

Contacts

Public ContactYe Wu

Department of Pediatrics, Peking University First Hospital

dryewu@263.net+86 13520756697

Outcome results

None listed

Source: ChiCTR (via WHO ICTRP) · Data processed: Feb 17, 2026