Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva

Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva - Investigator-initiated clinical trial of NPC-12T for Fibrodysplasia Ossificans Progressiva

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000028429

Enrollment

Registered

2017-08-01

Start date

2017-09-07

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

Fibrodysplasia Ossificans Progressiva

Interventions

Initial dose is 1 mg per day for patients with body surface area less than 1.5 m2, or 2 mg per day for patients with that not less than 1.5 m2. Oral intake will be adjusted targeting trough value betw

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients diagnosed as "Definitive" or "Probable" according to the diagnostic criteria proposed by the research group for intractalble diseases organized by MHLW.

Exclusion criteria

Exclusion criteria: 1. Patients with acute or chronic infection 2. Patients with skin sore reaching dermis 3. Patients experiencing flare-up during last 90 days 4. Past usage of mTOR inhibitors or other molecular target drugs relating mTOR pathway 5. Usage of medicine or foods which are prohibited for concurrent use of Sirolimus 6. History of allergy to Sirolimus, Sirolimus derivatives or additive substance 7. Patients with HBV, with the history of HBV or with active type-C hepatitis. 8. Severe abnormality in blood or liver function 9. Uncontrollable abnormal lipid metabolism 10. Renal failure 11. Immunodeficiency including HIV or primary immunodeficiency 12. Patients who are not able to take tablet, or who have gastrointestinal disorders possibly leading to insufficient absorption of Sirolimus 13. Patients who had surgery during 8 weeks before the registry 14. Pregnant, probably pregnant, or breast-feeding women. Patients who do not agree birth control during clinical trial.

Design outcomes

Primary

Measure	Time frame
Objective evaluation of physical function using J-HAQ (or J-CHAQ) at the end of double-blind stage	—

Secondary

Measure	Time frame
Frequency and grade of adverse events and side effects Quantitative assessment of heterotopic bone by whole-body CT Number of newly formed heterotopic bone Number of flare-up episode Assessment by CAJIS	—

Countries

Japan

Contacts

Public ContactJunya Toguchida

Institute for Frontier Life and Medical Sciences, Kyoto University Department of Regeneration Science and Engineering, Laboratory of Tissue Regeneration

togjun@infront.kyoto-u.ac.jp075-751-4134

Outcome results

None listed

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