Olaparib for PAH: a Multicenter Clinical Trial

Sponsor
Laval University
Study ID
NCT03782818
Phase
PHASE1/PHASE2
Status
Terminated

Conditions

  • Pulmonary Arterial Hypertension

Eligibility Criteria

Sex
ALL
Age
18 Years - 75 Years
Healthy Volunteers
Not accepted

Interventions

  • Olaparib — DRUG
    Olaparib up to 300 mg BID for 24 weeks

Study Details

The main OBJECTIVE of this proposal is to extend our preclinical findings on the role of DNA damage and poly(ADP-ribose) polymerases (PARP) inhibition as a therapy for a devastating disease, pulmonary arterial hypertension (PAH), to early-phase clinical trials. We, and others, have published strong evidence that DNA damage accounts for disease progression in PAH and showed that PARP1 inhibition can reverse PAH in several animal models1. Interestingly, PARP1 inhibition is also cardioprotective. Olaparib, an orally available PARP1 inhibitor, can reverse cancer growth in animals and humans with a good safety profile, and is now approved for the treatment of ovarian cancer in Canada, Europe and the USA. The time is thus right to translate our findings in human PAH. The primary objective of this Phase 1B study is to confirm the safety of using olaparib in PAH patients, and precise the sample size of a future Phase 2 trial. In addition to safety, efficacy signals will thus be assessed.

Key Dates

Start date
Nov 20, 2019
Status verified
Feb 2025
Primary completion
Oct 31, 2024
Completion
Oct 31, 2024

Study Design

Enrollment
17 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Olaparib
    After a 4-week pre-treatment phase to ensure that patients are on stable doses of PAH medication, patients will be given progressive doses of olaparib up to 300 mg BID for 24 weeks.

Primary Outcome Measure

Occurrence of treatment-emergent AEs at week 24 [ Time Frame: Week 24 ]

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