NeuroNEXT: Network for Excellence in Neuroscience Clinical Trials

NeuroNEXT will provide a robust, standardized, and accessible infrastructure to facilitate rapid development and implementation of protocols in neurological disorders affecting adult and/or pediatric populations. The network includes multiple Clinical Sites, one Clinical Coordinating Center (CCC) and one Data Coordinating Center (DCC).

You DO NOT need to be part of the NeuroNEXT infrastructure to apply to conduct a study within the network. Applications from academic investigators, advocacy groups/foundations, small businesses and the pharmaceutical industry are welcomed.

 

NeuroNEXT Executive Committee (NEC)

Permanent members

• Christopher S. Coffey
• Merit Cudkowicz

Rotating members

• Anthony Amato
• Brenda Banwell
• Stacey Clardy
• Angie Molloy
• Aleks Videnovic
• Dan Woo
• Claudia Chiriboga

Data Coordinating Center: University of Iowa

Clinical Coordinating Center: Massachusetts General Hospital

NeuroNEXT Clinical Sites

     Current

 

How to Apply to NeuroNEXT

NeuroNEXT is intended to facilitate the testing of novel therapeutics (drugs, biologics, devices) for neurological disorders. These novel therapeutics may come from academic investigators or directly from the biotechnology or pharmaceutical sector. NeuroNEXT aims to support scientifically sound phase 2 clinical trials that provide data for clear go/no-go decisions. Biomarker validation studies are also considered either independently or as part of a therapeutic trial.

Working with NeuroNEXT is a cooperative venture between NINDS, the NeuroNEXT network and the applicant. NINDS will provide guidance to potential applicants with input from the Division of Clinical Research (DCR), the applicable NINDS Extramural office/cluster and the NeuroNEXT Executive Committee (NEC). Potential applicants should contact NINDS DCR in order to discuss the feasibility of conducting the proposed trial through the NeuroNEXT infrastructure.

There are two mechanisms available for potential applicants:

1. Academic and industry investigators may apply for NeuroNEXT Clinical Trials (OTA-24-013(pdf, 311 KB)).
2. Small businesses may apply for NeuroNEXT Clinical Trials (OTA-24-013(pdf, 311 KB)); OR through our Small Business program: NeuroNEXT Small Business Innovation in Clinical Trials (U44) (PAR-21-224).

To learn more about the NeuroNEXT 2-Stage Application and Review process, view our informational webinar!

Potential applicants whose clinical research cannot be supported through NeuroNEXT are encouraged to consider an alternate funding mechanism:

• NINDS Exploratory Clinical Trials (PAR-22-142)
• NINDS Efficacy Clinical Trials (PAR-21-237)

 

New Research Opportunity Announcements for Ultra-rare Gene-based Therapy (URGenT) Clinical Trials conducted within NeuroNEXT

NeuroNEXT is pleased to announce two Research Opportunity Announcements (ROAs) to support clinical trials of gene-based and gene-targeted therapies for ultra-rare neurological diseases.  The URGenT Clinical Trials program will utilize a two-stage application and review process for therapeutics that have an active Investigational New Drug (IND) designation.  Stage 1 applications (OTA-24-011(pdf, 283 KB)) will include scientific background information supporting the gene-therapy product/therapeutic and proposed clinical trial.  Stage 2 Protocol Applications (OTA-24-012(pdf, 381 KB)) will be accepted by invitation only and will include the submission of more detailed information on the proposed clinical trial and related activities. Potential applicants are STRONGLY encouraged to contact NeuroNEXT/URGenT Program Staff to discuss their application and the application process prior to submission.

Referral/Review Process for NeuroNEXT Applications

• Prospective applicant contacts NINDS/DCR to discuss the proposed trial:

Sophie Cho, M.D.
Program Director, Division of Clinical Research
hyunjoo.cho@nih.gov

• NINDS program staff will discuss the proposed trial.
• Trials that have good NINDS program support are referred to the NEC to assess network suitability.
• NeuroNEXT Clinical Trials (OTA) - Applications are accepted on a rolling basis. 
  • Projects that have NINDS program support and are suitable for network performance are asked to submit a formal Stage 1 Preliminary Application for independent/objective review. 
  • After successful review of the Stage 1 Preliminary Application and upon receiving an invitation to proceed to Stage 2, the asset holder will work with the NeuroNEXT to further develop the clinical trial protocol.
  • At Stage 2 the NeuroNEXT CCC becomes the applicant of record for the Stage 2 Protocol Application (OTA-24-014(pdf, 836 KB)).
  • NIH funds to conduct the study are awarded to the NeuroNEXT CCC only after successful completion of both stages of application and review, and approval by the NINDS Director following National Advisory Neurological Disorders and Stroke Council review.
• NeuroNEXT Small Business Innovation in Clinical Trials (U44) - Standard dates apply for scientific merit review of applications coming under the U44 grant mechanism: for detailed information, please refer to Section V. Application Review Information in PAR-21-224.

Current NeuroNEXT-supported Studies

• NN 109 MAGINE (A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy of ManNAc in Subjects with GNE Myopathy
• NN 111 The ExTINGUISH Trial of Inebilizumab in NMDAR Encephalitis

Completed

• NN 101 SMA biomarkers study
• NN 102 SPRINT-MS
• NN 103 Beat MG
• NN 104 RHAPSODY
• NN 105 STAIR (SRX246: Safety, Tolerability, and Activity in IRritable subjects with HD)
• NN 106 CYTO-C (Cytochrome C Oxidase: Biomarker in Newly Diagnosed Glioblastoma Multiforme)
• NN 107 Effects of AFQ056 on Language Learning in Young Children with Fragile X Syndrome
• NN 108 TopCSPN (Topiramate as a Disease Altering Therapy for CSPN)
• NN 110 A Dose Selection Trial of Light Therapy for Impaired Sleep in Parkinson’s Disease

Resources and Tools

Frequently Asked Questions

NeuroNEXT: Network for Excellence in Neuroscience Clinical Trials, Frequently Asked Questions (FAQs)

1. Do I need to fill out a concept synopsis form before applying to NeuroNEXT?

Yes.  Potential applicants should send a completed concept synopsis form to Program Staff prior to submitting a NeuroNEXT application. The concept synopsis is used to assess mission relevance and fit to the program as well as network suitability of the proposed trial. The template for this can be found on our website. 

2. Am I required to have an IND or IDE before applying to NeuroNEXT?

If the proposed intervention is a drug, biologic, or device, an open IND (Investigational New Drug)/IDE (Investigational Device Exemption) will be a funding milestone. The applicants are required to provide documentations from FDA to indicate that the asset is IND/IDE ready.

3. Can small businesses apply to the NeuroNEXT?

Yes.  Small businesses are encouraged to apply to NeuroNEXT.  Small businesses may apply through either NeuroNEXT’s U44 SBIR (Small Business Innovation Research) grant mechanism, or its 2-Stage Other Transaction (OT) process, as applicable. Please reach out to NINDS Program Staff to discuss options.

4. Is there a budget limit on NeuroNEXT applications?

No.  There is no budget limit on NeuroNEXT applications.  The budget proposed will be reviewed to ensure that proposed costs are allowable, reasonable, justified, and fair. In general, NINDS expects the total cost for the proposed project (direct cost, plus F&A) to not exceed $25,000 per randomized subject. Trial budget must be approved by NINDS before Stage 2 application submission.

5. What is an Other Transaction (OT)?

An Other Transaction (OT) is a unique type of legal instrument other than a contract, grant, or cooperative agreement. Generally, this awarding instrument is not subject to the FAR (Federal Acquisition Regulation), or grant regulations unless otherwise noted for certain provisions in the terms and conditions of award. It is, however, subject to the OT Authority that governs the initiative, as well as applicable legislative mandates.

6. Will applications submitted under OT be reviewed within a study section?

No.  Applications submitted for funding under OT are not reviewed within a traditional NIH study section.  NeuroNext OT applications undergo independent/objective review by individuals with appropriate scientific/technical knowledge and expertise. Reviewers’ individual opinions are obtained; consensus is not the goal. Applications are not ranked. Although traditional summary statements are not provided to applicants, feedback from the review will be given in written communication. Conflicts-of-interest of review panel members are appropriately managed during the review process in accordance with standard NIH policies.

7. How will the applicant be informed of the review outcome?

NIH Program Officials will provide applicants with feedback on the independent/objective review and a memo notifying them of the NINDS funding decision.

8. Since Stage 1 and Stage 2 applications are accepted on a rolling basis, when and how often do applications get reviewed?

Review sessions will occur approximately every 6 -8 weeks depending on application volume.

9. Will the same external reviewers conduct both the Stage 1 and Stage 2 reviews?

Reviewers will be selected based on the expertise needed for each stage.  Some reviewers will be invited to participate in both stages of review, so there will be some overlap across the two stages.

10. Is there a pathway for resubmission under the OT funding mechanism? 

The OT mechanism allows us to use an iterative review process such that, depending on the review outcome, applicants may be able to submit their responses or resubmit their proposals to the next review round, as long as they can sufficiently address reviewer concerns. 

11. What is the total timeline from project proposal to funding and how does this differ from the grant application process?

Total time from proposal to funding is variable due to many factors including protocol development and OT agreement negotiations but may be as short as 6-9 months (i.e., shorter than for a grant mechanism).

12. How are Clinical Sites selected for a Clinical Trial?

Clinical Sites are selected based on their available expertise, resources, and access to the population under study, as well as additional factors.  Sites currently funded as part of the NeuroNEXT infrastructure are encouraged to participate, as applicable.  Additional out of network sites may be included ad hoc based on disease population, investigator expertise, and/or other needs.

13. Can non-network Sites submit a proposal?

Yes.  Investigators do not need to be part of existing infrastructure to submit a proposal.  Applications may be submitted by investigators from academia, foundations, or industry.

14. In previous NOFOs, there was a requirement for using a minimum of four NeuroNEXT Sites in each proposal. Is this also required under the OT mechanism?

In general, yes.  However, this requirement may be waived due to the nature of the pathology under investigation, especially for proposals submitted under our new URGenT Clinical Trial Program.

15. Can a foreign (non-U.S.) entity apply to NeuroNEXT?

Yes.  Non-U.S. individuals, organizations and companies can apply to NeuroNEXT. Please note that the asset holder/Stage 1 applicant does not receive funding for a NeuroNEXT Clinical Trial. Other Transactions (OT) funds are provided to the NeuroNEXT Clinical Coordinating Center to administer for conduct of the clinical trial for accepted assets.

   16.  For the Stage 1 Preliminary Application, does the 12 page limit include references?

No.  References are not counted as part of the 12 page limit.

 

 

Contacts