ObesityPhase 1/2NCT02117700
Fatty Liver Disease in Obese Children
A Phase 1/2 study testing N-acetyl cysteine 600 mg once/day and N-acetyl cysteine 600mg twice/day in people with Obesity and Nonalcoholic Fatty Liver Disease, run by Nemours Children's Clinic at 1 site in United States.
Trial summary
Although weight reduction through physical activity-based interventions is the mainstay therapy for nonalcoholic fatty liver disease (NAFLD), its maintenance is difficult and typically unsuccessful. This affirms the extreme need for alternate and/or adjunct therapies. Read more about this study
- Who is it for?
- Ages 7 to 18 years
- Conditions the study is for
- Obesity, Nonalcoholic Fatty Liver Disease, Cardiovascular Disease
- What is being tested?
- N-acetyl cysteine 600 mg once/day and N-acetyl cysteine 600mg twice/day
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2015to Dec 2020
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 7 years and older
- NASH confirmed in a previous biopsy
- HbAIc <6.4%
- ALT > 60 U/L or 1.5 times the upper limit of normal
Who cannot join
- Chronic liver disease including alpha-1-antitrypsin deficiency, Wilson's disease, autoimmune and viral hepatitis
- Medications such as adrenergic β-blockers, steroids and other drugs known to interfere with the measurement of liver enzymes and risk factors for cardiovascular disease
- Heart disease, chronic renal disease, adrenal, hepatic or thyroid dysfunction; active malignancy; and anemia
- History of prior treatment with NAC
- Evidence of hypersensitivity/allergy to NAC
- Alcoholism or drug abuse and smoking
- Inter-current illness over 7 days before the study \& surgery in the past 3 mo.
Trial locations and contacts
1 site in 1 country.
United States1 site
Nemours Children's Clinic/Alfred I duPont Hospital
Jacksonville, Florida, 32207
About this study
Official title: Effect of N-acetyl Cysteine on Non Alcoholic Fatty Liver Disease in Obese Children
Although weight reduction through physical activity-based interventions is the mainstay therapy for nonalcoholic fatty liver disease (NAFLD), its maintenance is difficult and typically unsuccessful. This affirms the extreme need for alternate and/or adjunct therapies. Although convincing data from animal studies and a few adult human studies on the benefits of a natural product, N-acetyl cysteine (NAC), in a variety of liver conditions including NAFLD have emerged, studies in children are scarce. Therefore, the aim of the study is to test the use NAC as an innovative approach to attenuate the progression of NAFD in obese children with biopsy proven NASH. The central hypothesis is that NAC supplementation will reduce liver fat and liver enzymes and ameliorate risk factors of cardiometabolic disease in children with NAFLD.
Read the detailed description
Physical activity (PA)-induced weight reduction, the suggested therapy for noalcoholic liver disease (NAFLD), is difficult and its maintenance is typically unsuccessful in children, affirming the acute need for alternative/adjunct therapies. Although few promising approaches have been reported, the benefits are incongruent and mostly marginal. N-acetyl cysteine (NAC), a derivative of the natural amino acid, cysteine, appears to be promising as an adjunct therapy to PA. Animal and a few adult human studies suggest NAC-induced attenuation of liver abnormalities, oxidative stress, insulin resistance and inflammation. The primary aim of the proposal is to determine in obese children with biopsy proven NASH and elevated liver enzymes the effect of NAC at two different doses on liver fat using magnetic resonance imaging (MRI), liver enzymes and risk factors of cardiometabolic disease. We hypothesize that NAC will produce beneficial effect on these parameters.
- Being tested
- N-acetyl cysteine 600 mg once/day, N-acetyl cysteine 600mg twice/day, Placebo twice/day
- Keywords
- fatty liver, obesity, children, oxidative stress, antioxidants
- Medical subject headings
- Obesity, Non-alcoholic Fatty Liver Disease, Cardiovascular Diseases, Fatty Liver
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-acetyl cysteine 600 mg once/dayDietary supplement
NAC 600 mg once/day + Placebo once/day for 16 weeks
N-acetyl cysteine 600mg twice/dayDietary supplement
N-acetyl cysteine 600 mg twice/day for 16 weeks
Placebo twice/dayOther
Placebo capsules twice/day for 16 weeks
| Study group | Role | What they receive |
|---|---|---|
| N-acetyl cysteine-1 | Experimental | N-acetyl cysteine 600 mg once/day + Placebo once/day for 16 weeks Receives: Dietary Supplement: N-acetyl cysteine 600 mg once/day |
| N-acetyl cysteine-2 | Experimental | N-acetyl cysteine 600 mg twice/day for 16 weeks Receives: Dietary Supplement: N-acetyl cysteine 600mg twice/day |
| Placebo | Placebo comparator | Placebo twice/day for 16 weeks Receives: Other: Placebo twice/day |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in liver fat (MRI) and ALT levels from baseline and at 16 weeks
The primary outcome will be sustained reduction in ALT level, defined as 50% or less of the baseline level or 40 U/L or less and significant changes in liver fat (MRI) at the end of the study. All measurements of biological factors will be performed in the post absorptive (fasted) state.
Measured: Upto 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2014 (estimated)
- Study startApr 2015
- Primary completionApr 2018
- Study completionDec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ObesityStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nemours Children's Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Babu Balagopal, Nemours Children's Clinic
- Investigators
- Babu Balagopal, PhD, Principal investigator, Nemours Children's Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02117700
- Sponsor's protocol ID
- NemoursCC
- Other IDs
- American Diabetes Association
- Status last verified
- Oct 2022
- First submitted
- 16 Apr 2014
- Last updated
- 17 Oct 2022
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Babu Balagopal P, Kohli R, Uppal V, Averill L, Shah C, McGoogan K, Di Guglielmo M, Goran M, Hossain MJ. Effect of N-acetyl cysteine in children with metabolic dysfunction-associated steatotic liver disease-A pilot study. J Pediatr Gastroenterol Nutr. 2024 Sep;79(3):652-660. doi: 10.1002/jpn3.12312. Epub 2024 Jul 8.
PubMed 38973318
Study at a glance
From the registry record, last updated by the sponsor on 17 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of N-acetyl Cysteine on Non Alcoholic Fatty Liver Disease in Obese Children
- ClinicalTrials.gov ID
- NCT02117700
- Acronym / sponsor ID
- NemoursCC
- Keywords
- fatty liver, obesity, children, oxidative stress, antioxidants
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Nemours Children's ClinicUniversity, hospital or other
- Study start
- Apr 2015 (actual)
- Primary completion
- Apr 2018 (actual)
- Study completion
- Dec 2020 (actual)
- First posted
- 21 Apr 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Although weight reduction through physical activity-based interventions is the mainstay therapy for nonalcoholic fatty liver disease (NAFLD), its maintenance is difficult and typically unsuccessful. This affirms the extreme need for alternate and/or adjunct therapies. Although convincing data from animal studies and a few adult human studies on the benefits of a natural product, N-acetyl cysteine (NAC), in a variety of liver conditions including NAFLD have emerged, studies in children are scarce. Therefore, the aim of the study is to test the use NAC as an innovative approach to attenuate the progression of NAFD in obese children with biopsy proven NASH. The central hypothesis is that NAC supplementation will reduce liver fat and liver enzymes and ameliorate risk factors of cardiometabolic disease in children with NAFLD.
Detailed description
Physical activity (PA)-induced weight reduction, the suggested therapy for noalcoholic liver disease (NAFLD), is difficult and its maintenance is typically unsuccessful in children, affirming the acute need for alternative/adjunct therapies. Although few promising approaches have been reported, the benefits are incongruent and mostly marginal. N-acetyl cysteine (NAC), a derivative of the natural amino acid, cysteine, appears to be promising as an adjunct therapy to PA. Animal and a few adult human studies suggest NAC-induced attenuation of liver abnormalities, oxidative stress, insulin resistance and inflammation. The primary aim of the proposal is to determine in obese children with biopsy proven NASH and elevated liver enzymes the effect of NAC at two different doses on liver fat using magnetic resonance imaging (MRI), liver enzymes and risk factors of cardiometabolic disease. We hypothesize that NAC will produce beneficial effect on these parameters.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| N-acetyl cysteine-1 | Experimental | Dietary Supplement: N-acetyl cysteine 600 mg once/dayN-acetyl cysteine 600 mg once/day + Placebo once/day for 16 weeks |
| N-acetyl cysteine-2 | Experimental | Dietary Supplement: N-acetyl cysteine 600mg twice/dayN-acetyl cysteine 600 mg twice/day for 16 weeks |
| Placebo | Placebo comparator | Other: Placebo twice/dayPlacebo twice/day for 16 weeks |
Who may qualify
- Ages
- 7 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Age 7 years and older
- NASH confirmed in a previous biopsy
- HbAIc <6.4%
- ALT > 60 U/L or 1.5 times the upper limit of normal
Key exclusion criteria (7 listed)
- Chronic liver disease including alpha-1-antitrypsin deficiency, Wilson's disease, autoimmune and viral hepatitis
- Medications such as adrenergic β-blockers, steroids and other drugs known to interfere with the measurement of liver enzymes and risk factors for cardiovascular disease
- Heart disease, chronic renal disease, adrenal, hepatic or thyroid dysfunction; active malignancy; and anemia
- History of prior treatment with NAC
- Evidence of hypersensitivity/allergy to NAC
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| N-acetyl cysteine 600 mg once/dayNAC 600 mg once/day + Placebo once/day for 16 weeks | Dietary supplement | N-acetyl cysteine-1 |
| N-acetyl cysteine 600mg twice/dayN-acetyl cysteine 600 mg twice/day for 16 weeks | Dietary supplement | N-acetyl cysteine-2 |
| Placebo twice/dayPlacebo capsules twice/day for 16 weeks | Other | Placebo |
Primary outcome
Change in liver fat (MRI) and ALT levels from baseline and at 16 weeks
Time frame: Upto 16 weeks
The primary outcome will be sustained reduction in ALT level, defined as 50% or less of the baseline level or 40 U/L or less and significant changes in liver fat (MRI) at the end of the study. All measurements of biological factors will be performed in the post absorptive (fasted) state.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Oct 2022.
- Derived from this study: PubMed 38973318Babu Balagopal P, Kohli R, Uppal V, Averill L, Shah C, McGoogan K, Di Guglielmo M, Goran M, Hossain MJ. Effect of N-acetyl cysteine in children with metabolic dysfunction-associated steatotic liver disease-A pilot study. J Pediatr Gastroenterol Nutr. 2024 Sep;79(3):652-660. doi: 10.1002/jpn3.12312. Epub 2024 Jul 8.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Babu Balagopal, PhD principal investigator
Nemours Children's Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing N-acetyl cysteine 600 mg once/day and N-acetyl cysteine 600mg twice/day in people with Obesity and Nonalcoholic Fatty Liver Disease, run by Nemours Children's Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in liver fat (MRI) and ALT levels from baseline and at 16 weeks (time frame: Upto 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02117700, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Oct 2022.