Evaluation of safety and efficacy of inhibitor of phosphodiesterase 5 to patients with dystrophinopathy

Evaluation of safety and efficacy of inhibitor of phosphodiesterase 5 to patients with dystrophinopathy - Safety and efficacy of PDE5 inhibitor to dystrophinopathy

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000032431

Enrollment

Registered

2018-04-30

Start date

2012-08-03

Completion date

Unknown

Last updated

2026-06-29

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

Conditions

patients with dystrophinopathy

Interventions

We use shildenafil as PDE5 inhibitor. Observational period is assigned from the day before shildenafil administration to 28 days after finishing administration. Shildenafil administration is pl

0.25mg/kg (start~day 3),0.5mg/kg (day 4 ~ day 7), and 1.0mg/kg (day 8~ day 14).

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: patients with dystrophinopathy based on gene analysis or muscle biopsy findings

Exclusion criteria

Exclusion criteria: patients with 24-hour mechanical ventilation (not include night NPPV) hypertension(Sys>135mmHg or Diast>90mmHg) heart failure with BNP>100pg/ml or LVEF<50% arrhythmia (Af,AF, VT,PSVT) diabetus mellitus

Design outcomes

Primary

Measure	Time frame
manual muscle test	—

Secondary

Measure	Time frame
6 minutes walk test(ambulant case) VC, MIC, CPF serum CK, CD34, BNP EF (UCG) evaluation time: before administration of PDE5 inhibitor, 14 days after administration, 28 days after finishing administration	—

Countries

Japan

Contacts

Public ContactToshio Saito

National Hospital Organization Toneyama National Hospital Division of clinical research

saitot@toneyama.go.jp+81-6-6853-2001

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026