Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease

Part of paid clinical trials in Akron, Ohio.

Sponsor
Pharming Technologies B.V.
Study ID
NCT07514338
Phase
PHASE2
Status
Recruiting
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Conditions

  • Mitochondrial Diseases

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Napazimone — DRUG
    Product: KL1333 (international nonproprietary name: napazimone) Dose: Each subject will be up-titrated to his/her maximum well tolerated dose. The starting dose will be 25 mg KL1333 twice daily (BID; total daily dose of 50 mg). If KL1333 is considered to be well tolerated after 4 weeks of treatment, the dose will be increased to 50 mg KL1333 BID (total daily dose of 100 mg). The dose may be lowered from 50 mg BID to 25 mg BID at the investigator's discretion throughout the study in case of tolerability issues. Frequency: Twice daily Route: Oral

Study Details

The purpose of this study is to investigate if the study medicine, KL1333, is safe, well-tolerated and effective long-term in improving the symptoms of fatigue and impacts on daily living and functional capacity (physical abilities) in people with PMD.

Key Dates

First listed
Apr 7, 2026
Start date
Jun 12, 2026
Status verified
Jul 2026
Primary completion
Mar 31, 2029
Completion
Mar 31, 2029

Study Design

Enrollment
140 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Open label extension
    Subjects will receive medication twice a day for 48 weeks minimum

Primary Outcome Measure

Adverse events [ Time Frame: Through study at least for 48 weeks ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Akron Children's HospitalAkronOhio44308
Bruce Cohen (PRINCIPAL_INVESTIGATOR)

Find similar trials in Akron, OH

By research site
Akron Children's Hospital· Akron, OH

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