Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients.

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2006-006845-14-FR

Enrollment

Registered

2007-02-15

Start date

2007-03-20

Completion date

Unknown

Last updated

2022-04-04

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

Conditions

Spinal Muscular Atrophy (SMA) MedDRA version: 9.1 Level: LLT Classification code 10041582 Term: Spinal muscular atrophy

Interventions

Product Code: TRO19622 Pharmaceutical Form: Capsule* CAS Number: 22033-87-0 Current Sponsor code: TRO19622 Other descriptive name: 4-cholesten-3-one,oxime Concentration unit: mg milligram(s) Concentra

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients with SMA type Ib, II or III Between 6-25 years old Having a "Motor Function Measure" (MFM) score superior or equal to 12 Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Concomitant treatments with drugs being investigated in a clinical trial or with drugs likely to interfere with TRO19622 absorption (ezetimibe, fibrates) Associated diseases (cardiovascular disorders, hemostasis disorders, renal insufficiency or confirmed hepatic insufficiency)

Design outcomes

Primary

Measure	Time frame
Main Objective: Pharmacokinetic;Secondary Objective: Safety;Primary end point(s): Safety and pharmacokinetic	—

Countries

France

Outcome results

None listed

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