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OPEN-LABEL EXTENSION STUDY TO ASSESS THE SAFETY AND LONG-TERM EFFECTIVENESS OF ORALLY ADMINISTERED EFAVIRENZ IN PATIENTS DIAGNOSED WITH ADULT OR LATE-ONSET JUVENILE TYPE C NIEMANN-PICK DISEASE WITH COGNITIVE IMPAIRMENT
EU CTIS ID: 2023-505832-36-01
What this study is testing
To evaluate the safety and effectiveness of efavirenz (EFV) at an oral dose of 25 mg/d, in addition to standard treatment, in patients diagnosed with adult or late-onset juvenile type C Niemann-Pick disease (NP-C) with cognitive impairment.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Patients (>18 years of age) included in the previous Niemann-Pick trial (EudraCT: 2019-004498-18)
- Patients who sign the written informed consent
You likely can't join if
- Patients who (at the principal investigator's discretion) would experience an overdemanding effort (financially and in terms of time) to participate in the extension study.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.