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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Johns Hopkins University

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Trial duration

22 Jan 2024 → ongoing

Decision date (initial)

2023-12-11

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

National Institutes of Health, United States Department of Health and Human Services

External identifiers

EU CT number

2022-501438-46-00

ClinicalTrials.gov

NCT05537220

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Safety

The main objective is to evaluate the efficacy of oral NAC 1800 mg twice a day in maintenance of visual function thereby reducing visual disability in patient with retinitis pigmentosa. The trial will also evaluate the long-term safety and tolerability of NAC 1800 mg twice a day for 45 months.

Conditions and MedDRA coding

Retinitis Pigmentosa

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. • Ability and willingness to provide informed consent • Age ≥ 18 and ≤65 years at time of signing Informed Consent Form • Ability and willingness to comply with the study protocol and to participate in all study visits and assessments in the investigator’s judgement • For candidates of childbearing potential: willingness to use a method of contraception • Agreement not to take supplements other than vitamin A

Exclusion criteria 1

1. General Exclusion Criteria • Active cancer within the past 12 months, except for appropriately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, or prostate cancer with Gleason score ≤ 6 and stable prostate specific antigen for > 12 months • Renal failure requiring renal transplant, hemodialysis, peritoneal dialysis, or anticipated to require hemodialysis or peritoneal dialysis during the study • Liver disease, cystic fibrosis, asthma, or chronic obstructive pulmonary disease (COPD), history of thrombocytopenia not due to a reversible cause or other blood dyscrasia • Uncontrolled blood pressure (defined as systolic > 180 and/or diastolic > 100 mmHg while at rest) at screening. If a patient's initial measurement exceeds these values, a second reading may be taken 30 or more minutes later. If the patient's blood pressure must be controlled by antihypertensive medication, the patient may become eligible if medication is taken continuously for at least 30 days. • History of other disease, physical examination finding, or clinical laboratory finding giving reasonable suspicion that oral NAC may be contraindicated or that follow up may be jeopardized • Cerebrovascular accident or myocardial infarction within 6 months of screening • Participation in an investigational study that involves treatment with any drug or device within 6 months of screening • Three relatives already enrolled in study • Pregnant, breast feeding, or intending to become pregnant during the study treatment period. Women of childbearing potential who have not had tubal ligation must have a urine pregnancy test at screening. • Known history of allergy to NAC • Having taken NAC in any form in the past 4 months • Phenylketonuria • Fructose intolerance • Glucose-galactose malabsorption • Sucrase-isomaltase insufficiency • Abnormal laboratory value including the value of alanine aminotransferase (ALT), aspartate aminotransferase (AST), or bilirubin being greater than 1.5 x the upper limit of normal • Any major abnormal findings on blood chemistry, hematology, and renal function lab tests that in the opinion of the Site Investigator and/or the Study Chair makes the candidate not suitable to participate in the trial • HIV or hepatitis B infection. • Severe angina that requires frequent administration of nitrates

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary efficacy endpoint is to determine if the progressive loss in EZ width assessed as the cumulative loss of EZ (calculated as the area under the curve, AUC) between baseline and M45 is significantly smaller in eyes of participants taking NAC 1800 mg bid compared with eyes of participants taking placebo. EZ width will be measured on a spectral domain-optical coherence tomography (SD-OCT) scan through the fovea.

Secondary endpoints 1

1. The secondary efficacy endpoints are to assess the relative efficacy in eyes of participants taking NAC 1800 mg bid compared with eyes of participants taking placebo on the basis of the following endpoints: • Change from baseline mean macular sensitivity measured by microperimetry (MP) at M45. • Change from baseline in BCVA measured by Early Treatment Diabetic Retinopathy Study (ETDRS) protocol at M45.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Johns Hopkins University

Sponsor organisation

Johns Hopkins University

Address

3400 North Charles Street

City

Baltimore

Postcode

21218-2625

Country

United States

Scientific contact point

Organisation

Johns Hopkins University

Contact name

Peter Campochiaro

Public contact point

Organisation

Johns Hopkins University

Contact name

Xiangrong Kong

Locations

3 EU/EEA countries · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Application history

4 submissions — initial application plus substantial / non-substantial modifications