Study of NNZ-2566 in Patients With Traumatic Brain Injury Under EFIC

NCT01366820 | Completed Phase 2 DMC

• 1 other identifier: Neu-2566-TBI-002
• Study Type: interventional
• Target: 261
• Locations: 1 country
• Sites: 6

Brief Summary

The purpose of this study is to determine whether NNZ-2566 is safe and effective in the treatment of Traumatic Brain Injury (TBI).

Conditions

Brain Injuries

Outcome Measures

Primary Outcomes (1)

• Reduced incidence, compared to placebo, of adverse events (AEs) and serious adverse events (SAEs)

  AEs to discharged or Day 30 post randomization, whichever occurs first, and SAEs through to 3 months (defined as 12-14 weeks), post randomization.

Secondary Outcomes (4)

• Evidence of efficacy in modifying global outcomes by evaluating Glasgow Outcome Scale - Extended (GOS-E) and activities of daily living (Mayo-Portland Adaptability Inventory - 4th Edition (MPAI-4))

  1 month (defined as 4-6 weeks) and 3 months (defined as 12-14 weeks), post randomization.

• Improvement in cognitive and neuropsychological functioning.

  1 month (defined as 4-6 weeks) and at 3 months (defined as 12-14 weeks), post randomization.

• Modification of the acute physiological processes in TBI by evaluating electroencephalographic (EEG) determinants in patients with moderate to severe TBI (defined as GCS 4-12), and biomarker levels.

  Baseline through to 72 hours post-start of infusion.

• Blood pharmacokinetics (PK) of an intravenous (i.v) dose of NNZ-2566 when administered as a 10-minute infusion immediately followed by a 72-hour infusion.

  Start of infusion through to 12 hours post infusion.

Study Arms (2)

NNZ-2566

EXPERIMENTAL

20 mg/kg intravenous bolus infusion of NNZ-2566 over 10 minutes followed by a continuous intravenous infusion of 6 mg/kg/h (n=133) intravenous infusion of NNZ-2566 for a total of 72 consecutive hours.

Drug: NNZ-2566

Sodium Chloride (0.9%) for Injection

PLACEBO COMPARATOR

Intravenous bolus infusion of Sodium Chloride (0.9%) for Injection over 10 minutes followed by a continuous intravenous infusion of Sodium Chloride (0.9%) for Injection for a total of 72 consecutive hours.

Drug: Placebo

Interventions

NNZ-2566 DRUG

Solution for intravenous infusion. Intravenous bolus infusion over 10 minutes followed by a continuous intravenous maintenance infusion for a total of 72 consecutive hours.

Also known as: Glycyl-L-2-Methylprolyl-L-Glutamic Acid

NNZ-2566

Placebo DRUG

Sodium Chloride 0.9% Injection

Also known as: Sodium Chloride 0.9% Injection

Sodium Chloride (0.9%) for Injection

Eligibility Criteria

• Age: 16 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Non-penetrating TBI.
• Age 16-75 years.
• Admission to hospital.
• Post resuscitation GCS 4-12.
• Have at least one reactive pupil.
• Able to receive investigational product within 8 hours of injury.
• Hemodynamically stable after resuscitation (systolic blood pressure (SBP) \>100 mm Hg).
• Able to read and write English and have sufficient motor dexterity prior to injury to undertake the neuropsychological and activities of daily living (ADL) testing, in the opinion of the investigator, at 1 month (defined as 4-6 weeks) and 3 months (defined as 12-14 weeks) post injury.

You may not qualify if:

• Penetrating brain injury.
• Spinal cord injury.
• Presence or known history of prior cerebral injury requiring hospitalization that would, in the opinion of the Investigator, interfere with or bias the assessment of efficacy.
• Non-traumatic brain injury.
• Known history of any medical or psychiatric disorder, or any severe concomitant disease that would, in the opinion of the Investigator, interfere with or bias the assessment of efficacy.
• Significant non-central nervous system (CNS) injuries sustained at the time of the TBI would, in the opinion of the Investigator, interfere with or bias the assessment of efficacy.
• Weight \>150 kg.
• Participation in another clinical trial within the previous 4 weeks.
• Clinical state requiring greater than 6 L blood, colloid or crystalloid fluid resuscitation prior to randomization.
• Pregnant or nursing mothers. Women of child-bearing potential must have a negative urine or blood test prior to randomization.
• Prior enrollment in this study.
• A marked baseline prolongation of corrected QT/QTc interval \>450 ms.
• History of risk factors for torsade de pointes (e.g. heart failure, hypokalemia (serum potassium at screening \<3.0 mmol/L)or family history of long QT syndrome).

Sponsors & Collaborators

• Neuren Pharmaceuticals Limited: lead

Study Sites (6)

Arrowhead Regional Medical Center

Colton, California, 92324, United States

Location

University of California, Davis Medical Center

Sacramento, California, 95817, United States

Location

The Queen's Medical Center

Honolulu, Hawaii, 96813, United States

Location

Detroit Receiving Hospital and University Health Center

Detroit, Michigan, 48201, United States

Location

Sinai Grace Hospital

Detroit, Michigan, 48235, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

MeSH Terms

Conditions

Brain Injuries

Interventions

trofinetide; Sodium Chloride

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Craniocerebral Trauma; Trauma, Nervous System; Wounds and Injuries

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• Ross R Bullock, M.D., PhD

  University of Miami, Lois Pope Life Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 29, 2011
• First Posted: June 6, 2011
• Study Start: February 1, 2013
• Primary Completion: January 1, 2016
• Study Completion: January 1, 2016
• Last Updated: February 5, 2018

Record last verified: 2018-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)