Attention-deficit Hyperactivity Disorder (ADHD)Phase 1NFC1-GREATNCT02286817
Phase I Single Dose, Open-Label Pharmacokinetic Study and Single-Blind, Placebo-Controlled Dose Escalation Study of NFC-1 in Adolescents With Attention-Deficit Hyperactivity Disorder
A Phase 1 study testing NFC-1 in people with Attention-deficit Hyperactivity Disorder (ADHD), run by Aevi Genomic Medicine, LLC, a Cerecor company at 1 site in United States.
Trial summary
This trial is a Phase 1 study in adolescents with ADHD and genetic disruptions impacting genes in the metabotropic glutamate receptor (mGluR) network. The objectives of the study are to evaluate the safety, tolerability, and pharmacokinetics following single-dose, oral administration of NFC-1 and to evaluate safety… Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Attention-deficit Hyperactivity Disorder (ADHD)
- What is being tested?
- NFC-1
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2015to Mar 2016
- Phase
- Phase 1
- 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 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patient's weight is within 5th to 95th percentile for age
- Patient has ADHD as defined by the Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5) and the Vanderbilt ADHD Rating Scale score (Parent or Teacher) > 16 at baseline with or without conventional ADHD therapy
- Patient has been genotyped (CAP/CLIA certified) to determine whether there are disruptive mutations in genes within the mGluR-network
- Patient has been a non-smoker and/or has not used nicotine or nicotine-containing products for at least approximately 6 months
- Patient is judged to be in good health, other than having ADHD, based on medical history, physical examination, vital signs measurements, and laboratory safety tests performed at the screening visit and/or prior to administration of study drug
- Female patients of reproductive potential will have a negative urine β-hCG test at screening and prior to drug administration. If sexually active, female participant agrees to use (and/or have their partner use) two acceptable methods of birth control beginning at least 2 weeks prior to administration of study drug and throughout the study. Acceptable methods of birth control are abstinence, or 2 of the following: intrauterine device (IUD), diaphragm, spermicides, cervical cap, contraceptive sponge, and condoms
- Patient has no clinically significant abnormality on electrocardiogram (ECG) performed at the screening visit and/or prior to administration of study drug
- Parent/legal guardian and patient understand the study procedures and agrees to the patient's participation in the study as indicated by parental/legal guardian signature on the patient consent form and patient signature on assent form
Who cannot join
- Patient or parent/legal guardian is, in the opinion of the investigator, mentally or legally incapacitated, has significant emotional problems at the time of screening visit or during the conduct of the study. Subjects with prior diagnosis of co-morbid major psychiatric disorders (i.e. aside from ADHD), including major depression, bipolar disease, Tourette syndrome, schizophrenia, autism spectrum disorder or pervasive development disorder, severe anxiety disorders
- Patient has a history of any illness that, in the opinion of the study investigator, might confound the results of the study or poses an additional risk to the patient by their participation in the study
- Patient has a history of clinically significant endocrine, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or diseases. Patients with a history of uncomplicated kidney stones may be enrolled in the study at the discretion of the investigator
- Patient has a history of stroke, chronic seizures, or major neurological disorder
- Patient is pregnant or a nursing mother
- Patient has a history of extreme psychological aversion to blood draws that in the opinion of the investigator or parents would result in compromising the study conduct. Patient has a history of extreme physiologic difficulty in venous access that in the opinion of the investigator and parents would result in compromising the study conduct
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- Patient has a history of inability to swallow whole unadulterated pills, which in the opinion of the investigator or parents would result in compromising the study conduct
- Patient has a systolic or diastolic blood pressure ≥ the 95th percentile for his/her age
- Patient consumes any alcoholic beverages
- Patient consumes excessive amounts of caffeine, defined as greater than 4 servings (1 serving is approximately equivalent to 120 mg of caffeine) of coffee, tea, cola, or other caffeinated beverages per day
- Patient has a history of significant multiple and/or severe allergies, or has had an anaphylactic reaction or significant intolerability to prescription of non-prescription drugs or food
- Patient is currently a regular user (including "recreational use") of any illicit drugs (including marijuana) or has a history of drug (including alcohol) abuse within approximately 3 years
- Patient has had surgery, lost more than 5cc/kg of blood, or participated in another investigational drug trial within 4 weeks prior to the screening visit.
- Laboratory abnormalities that indicate clinically significant hematologic, hepatobiliary, or renal disease
- AST/SGOT > 2.0 times the upper limit of normal
- ALT/SGPT > 2.0 times the upper limit of normal
- Total bilirubin > 2.0 times the upper limit of normal
- Hemoglobin < 9 gm/dL
- White blood cell count < 1,000/ mm3
- Platelet count < 100,000/mm3
- Any investigational drug use within 30 days prior to enrollment
Trial locations and contacts
1 site in 1 country.
United States1 site
Thomas Jefferson University, Clinical Research Unit
Philadelphia, Pennsylvania, 19107
About this study
Official title: Phase I Single Dose, Open-label, Pharmacokinetic Study Followed by Single-blind, Placebo-controlled Dose Escalation Study to Evaluate the Safety, Tolerability, Plasma Concentration Profiles, and Targeted Efficacy of NFC-1 in Adolescents (12-17 Years of Age) With Attention-Deficit Hyperactivity Disorder and Genetic Disruption Impacting Metabotropic Glutamate Receptor Genes (NFC1-GREAT)
This trial is a Phase 1 study in adolescents with ADHD and genetic disruptions impacting genes in the metabotropic glutamate receptor (mGluR) network. The objectives of the study are to evaluate the safety, tolerability, and pharmacokinetics following single-dose, oral administration of NFC-1 and to evaluate safety and tolerability and to obtain evidence for the effect of NFC-1 on ADHD severity and global functioning during and following four weeks of continuous treatment. Exploratory analyses will be performed to assess effect size of specific mGluR-network genes on ADHD based on responsiveness of patients to NFC-1. This study will be conducted at a single clinical site, the Jefferson University Hospital PKU (Philadelphia, PA).
- Being tested
- NFC-1
- Medical subject headings
- Attention Deficit Disorder with Hyperactivity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NFC-1Drug
Single-dose, open label administration to assess safety, tolerability, and pharmacokinetics in adolescents with ADHD and continuous daily administration for four weeks with weekly escalation to evaluate safety, tolerability, and impact on ADHD severity.
| Study group | Role | What they receive |
|---|---|---|
| Single arm of 30 subjects | Experimental | Subjects will be administered single dose of NFC-1 to assess safety, tolerability, and pharmacokinetics, then proceed to continuous, daily administration of NFC-1 for 4 weeks to assess safety, tolerability, and impact on ADHD severity. Receives: Drug: NFC-1 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessing the safety and tolerability of NFC-1 at each dose level based on assessment of adverse events and other safety measurements including vital signs, electrocardiogram, laboratory safety tests.
Measured: 24 hours
Profile pharmacokinetics of NFC-1 in adolescents when administered orally as single dose.
Measured: 24 hours
Assessing the safety and tolerability of NFC-1 during 4 weeks of continuous daily administration based on assessment of adverse events and other safety measurements including vital signs, electrocardiogram, laboratory safety tests.
Measured: 1-4 weeks
Secondary outcomes (5)
The change in Vanderbilt scores during and following 4-week treatment with NFC-1
Measured: 1-4 weeks
The change in Actigraphy scores during and following 4-week treatment with NFC-1
Measured: 1-4 weeks
The change in Quotient ADHD test scores during and following 4-week treatment with NFC-1
Measured: 1-4 weeks
The change in PERMP-MATH test scores during and following 4-week treatment with NFC-1
Measured: 1-4 weeks
The change in Clinical Global Impressions Severity/Improvement scores during and following 4-week treatment with NFC-1
Measured: 1-4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2014 (estimated)
- Study startJan 2015
- Primary completionMay 2015
- Study completionMar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Attention-deficit Hyperactivity Disorder (ADHD)Studies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aevi Genomic Medicine, LLC, a Cerecor company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Thomas Jefferson University, Children's Hospital of Philadelphia, Pearson/Clinical Assessment
- Responsible party
- Sponsor
- Investigators
- Walter Kraft, MD, Principal investigator, Thomas Jefferson UniversityJosephine Elia, MD, Principal investigator, Alfred I. duPont Hospital for Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02286817
- Sponsor's protocol ID
- NFC1-2014
- Acronym
- NFC1-GREAT
- Status last verified
- Oct 2025
- First submitted
- 4 Nov 2014
- Last updated
- 21 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Glessner JT, Khan ME, Chang X, Liu Y, Otieno FG, Lemma M, Slaby I, Hain H, Mentch F, Li J, Kao C, Sleiman PMA, March ME, Connolly J, Hakonarson H. Rare recurrent copy number variations in metabotropic glutamate receptor interacting genes in children with neurodevelopmental disorders. J Neurodev Disord. 2023 Apr 29;15(1):14. doi: 10.1186/s11689-023-09483-z.
PubMed 37120522Slaby I, Hain HS, Abrams D, Mentch FD, Glessner JT, Sleiman PMA, Hakonarson H. An electronic health record (EHR) phenotype algorithm to identify patients with attention deficit hyperactivity disorders (ADHD) and psychiatric comorbidities. J Neurodev Disord. 2022 Jun 11;14(1):37. doi: 10.1186/s11689-022-09447-9.
PubMed 35690720Elia J, Ungal G, Kao C, Ambrosini A, De Jesus-Rosario N, Larsen L, Chiavacci R, Wang T, Kurian C, Titchen K, Sykes B, Hwang S, Kumar B, Potts J, Davis J, Malatack J, Slattery E, Moorthy G, Zuppa A, Weller A, Byrne E, Li YR, Kraft WK, Hakonarson H. Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling. Nat Commun. 2018 Jan 16;9(1):4. doi: 10.1038/s41467-017-02244-2.
PubMed 29339723
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Single Dose, Open-label, Pharmacokinetic Study Followed by Single-blind, Placebo-controlled Dose Escalation Study to Evaluate the Safety, Tolerability, Plasma Concentration Profiles, and Targeted Efficacy of NFC-1 in Adolescents (12-17 Years of Age) With Attention-Deficit Hyperactivity Disorder and Genetic Disruption Impacting Metabotropic Glutamate Receptor Genes (NFC1-GREAT)
- ClinicalTrials.gov ID
- NCT02286817
- Acronym / sponsor ID
- NFC1-GREAT / NFC1-2014
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Aevi Genomic Medicine, LLC, a Cerecor companyIndustry (pharma, biotech, devices)
- Collaborators
- Thomas Jefferson University, Children's Hospital of Philadelphia, Pearson/Clinical Assessment
- Study start
- Jan 2015
- Primary completion
- May 2015 (actual)
- Study completion
- Mar 2016 (actual)
- First posted
- 10 Nov 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.
This trial is a Phase 1 study in adolescents with ADHD and genetic disruptions impacting genes in the metabotropic glutamate receptor (mGluR) network. The objectives of the study are to evaluate the safety, tolerability, and pharmacokinetics following single-dose, oral administration of NFC-1 and to evaluate safety and tolerability and to obtain evidence for the effect of NFC-1 on ADHD severity and global functioning during and following four weeks of continuous treatment. Exploratory analyses will be performed to assess effect size of specific mGluR-network genes on ADHD based on responsiveness of patients to NFC-1. This study will be conducted at a single clinical site, the Jefferson University Hospital PKU (Philadelphia, PA).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm of 30 subjects | Experimental | Drug: NFC-1Subjects will be administered single dose of NFC-1 to assess safety, tolerability, and pharmacokinetics, then proceed to continuous, daily administration of NFC-1 for 4 weeks to assess safety, tolerability, and impact on ADHD severity. |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Patient's weight is within 5th to 95th percentile for age
- Patient has ADHD as defined by the Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5) and the Vanderbilt ADHD Rating Scale score (Parent or Teacher) > 16 at baseline with or without conventional ADHD therapy
- Patient has been genotyped (CAP/CLIA certified) to determine whether there are disruptive mutations in genes within the mGluR-network
- Patient has been a non-smoker and/or has not used nicotine or nicotine-containing products for at least approximately 6 months
- Patient is judged to be in good health, other than having ADHD, based on medical history, physical examination, vital signs measurements, and laboratory safety tests performed at the screening visit and/or prior to administration of study drug
Key exclusion criteria (21 listed)
- Patient or parent/legal guardian is, in the opinion of the investigator, mentally or legally incapacitated, has significant emotional problems at the time of screening visit or during the conduct of the study. Subjects with prior diagnosis of co-morbid major psychiatric disorders (i.e. aside from ADHD), including major depression, bipolar disease, Tourette syndrome, schizophrenia, autism spectrum disorder or pervasive development disorder, severe anxiety disorders
- Patient has a history of any illness that, in the opinion of the study investigator, might confound the results of the study or poses an additional risk to the patient by their participation in the study
- Patient has a history of clinically significant endocrine, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or diseases. Patients with a history of uncomplicated kidney stones may be enrolled in the study at the discretion of the investigator
- Patient has a history of stroke, chronic seizures, or major neurological disorder
- Patient is pregnant or a nursing mother
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 |
|---|---|---|
| NFC-1Single-dose, open label administration to assess safety, tolerability, and pharmacokinetics in adolescents with ADHD and continuous daily administration for four weeks with weekly escalation to evaluate safety, tolerability, and impact on ADHD severity. | Drug | Single arm of 30 subjects |
Primary outcome
Assessing the safety and tolerability of NFC-1 at each dose level based on assessment of adverse events and other safety measurements including vital signs, electrocardiogram, laboratory safety tests.
Time frame: 24 hours
Profile pharmacokinetics of NFC-1 in adolescents when administered orally as single dose.
Time frame: 24 hours
Assessing the safety and tolerability of NFC-1 during 4 weeks of continuous daily administration based on assessment of adverse events and other safety measurements including vital signs, electrocardiogram, laboratory safety tests.
Time frame: 1-4 weeks
5 secondary outcomes
The change in Vanderbilt scores during and following 4-week treatment with NFC-1
Time frame: 1-4 weeks
The change in Actigraphy scores during and following 4-week treatment with NFC-1
Time frame: 1-4 weeks
The change in Quotient ADHD test scores during and following 4-week treatment with NFC-1
Time frame: 1-4 weeks
The change in PERMP-MATH test scores during and following 4-week treatment with NFC-1
Time frame: 1-4 weeks
The change in Clinical Global Impressions Severity/Improvement scores during and following 4-week treatment with NFC-1
Time frame: 1-4 weeks
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 21 Oct 2025.
- Derived from this study: PubMed 37120522Glessner JT, Khan ME, Chang X, Liu Y, Otieno FG, Lemma M, Slaby I, Hain H, Mentch F, Li J, Kao C, Sleiman PMA, March ME, Connolly J, Hakonarson H. Rare recurrent copy number variations in metabotropic glutamate receptor interacting genes in children with neurodevelopmental disorders. J Neurodev Disord. 2023 Apr 29;15(1):14. doi: 10.1186/s11689-023-09483-z.
- Derived from this study: PubMed 35690720Slaby I, Hain HS, Abrams D, Mentch FD, Glessner JT, Sleiman PMA, Hakonarson H. An electronic health record (EHR) phenotype algorithm to identify patients with attention deficit hyperactivity disorders (ADHD) and psychiatric comorbidities. J Neurodev Disord. 2022 Jun 11;14(1):37. doi: 10.1186/s11689-022-09447-9.
- Derived from this study: PubMed 29339723Elia J, Ungal G, Kao C, Ambrosini A, De Jesus-Rosario N, Larsen L, Chiavacci R, Wang T, Kurian C, Titchen K, Sykes B, Hwang S, Kumar B, Potts J, Davis J, Malatack J, Slattery E, Moorthy G, Zuppa A, Weller A, Byrne E, Li YR, Kraft WK, Hakonarson H. Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling. Nat Commun. 2018 Jan 16;9(1):4. doi: 10.1038/s41467-017-02244-2.
- 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.
Walter Kraft, MD principal investigator
Thomas Jefferson University
Josephine Elia, MD principal investigator
Alfred I. duPont Hospital for Children
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NFC-1 in people with Attention-deficit Hyperactivity Disorder (ADHD), run by Aevi Genomic Medicine, LLC, a Cerecor company 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 8 inclusion and 21 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 21 Oct 2025. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
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?
Assessing the safety and tolerability of NFC-1 at each dose level based on assessment of adverse events and other safety measurements including vital signs, electrocardiogram, laboratory safety tests. (time frame: 24 hours). The study lists 3 primary outcomes in all. 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 NCT02286817, which also lists 3 linked publications.
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 21 Oct 2025.