(not active) Oral N-acetylcysteine for Retinitis Pigmentosa (NAC Attack)
Research type
Research Study
Full title
NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients with Retinitis Pigmentosa
IRAS ID
1007751
Contact name
Xiangrong Kong
Contact email
Sponsor organisation
Johns Hopkins University School of Medicine
Eudract number
2022-003023-17
Clinicaltrials.gov Identifier
Research summary
This study is being done to determine if compared to placebo, 1800 mg NAC twice a day in patients with RP can reduce loss of cone photoreceptors and delay vision loss over a span of 45 months. It will also determine if there are side effects from long-term use of NAC. This is very important information for all current and future patients with RP. If NAC is found to be safe and effective it will be a life-long treatment. It is critical that patients know the potential benefits and risks of taking NAC long-term before making such a commitment. NAC, either given orally or by injection into the blood, has been approved by the Food and Drug Administration (FDA) to treat paracetamol overdose. It is also approved as a formulation that is inhaled to break down excessive mucous in patients with various lung diseases. NAC is not approved by the MHRA for the use in the treatment of RP which is why its use in this study is considered investigational. The regulatory agencies are allowing us to use NAC in this study for all participating sites. To determine whether NAC can slow vision loss in RP, a randomized, placebo-controlled trial is needed. An eligible patient will be assigned by chance through a process like flipping a coin to be in the investigational drug (NAC) group or the placebo group (no NAC). The chance of being in the NAC group is twice the chance of being in the placebo group.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
23/YH/0122
Date of REC Opinion
17 Oct 2023
REC opinion
Further Information Unfavourable Opinion