Erika
Augustine
,
MD, MS

Erika Augustine, MD, MS
Associate Chief Science Officer, Director of the Clinical Trials Unit
Kennedy Krieger Institute

707 North Broadway
Baltimore, MD 21205
United States

Centers & Programs

About

Dr. Augustine received her A.B., Biological Anthropology from Harvard College, and her Doctorate of Medicine from the University of Rochester and then trained in Pediatric Neurology at Boston Children’s Hospital, serving as Chief Resident.  She returned to the University of Rochester for parallel fellowships in movement disorders and experimental therapeutics.  She is regarded highly as a clinician, scientist, mentor, and leader.

At Kennedy Krieger, Dr. Augustine will work closely with Dr. Amy Bastian, our chief science officer, to help develop our clinical and translational research strategy and oversee research operations.  Dr. Augustine will be responsible for the development, growth, structure and financial management of our clinical trials functions. She will direct the Institute’s clinical trials unit, be the primary liaison with the Johns Hopkins School of Medicine Institute for Clinical and Translational Research (ICTR), and work closely with the Office of Human Research Administration on human subjects trials oversight and regulatory compliance.

Related Links and Research

PubMed Link

 

U01NS101946 (PI: Augustine)

Meaningful Outcomes and Multi-Site Readiness for Clinical Trials in Juvenile Neuronal Ceroid Lipofuscinosis

This project will validate sensitive measures of how patients feel and function, validate neuroimaging biomarkers of disease progression, and prepare these tools for multi-site use to set the stage for rigorous and efficient clinical trials in CLN3 disease.

Role: Principal Investigator


P50NS108676 (PI: Dorsey)

A Udall Center at the University of Rochester

Goal: To establish a Udall Center at the University of Rochester. The Center will work collaboratively with other Udall Centers as well as independently to define the causes of and discover improved treatments for Parkinson’s disease. The major goals of this project are to develop novel digital biomarkers for the diagnosis and monitoring of progression in individuals with idiopathic Parkinson disease.

Role: Associate Director, Administrative Core


U24 TR002260 (MPI: Zand and Ossip)

CTSA Coordinating Center for Leading Innovation and Collaboration (CLIC)

The University of Rochester’s Coordination Center for Leading Innovation and Collaboration (CLIC) aims to maximize the potential of the CTSA Consortium in advancing translational science by actively catalyzing Consortium activities to promote efficiency and high quality research, training, and network building.

Role: Co-Investigator


U10NS077265 (PI: Holloway)

NINDS NeuroNEXT

University of Rochester Clinical Site within the NEXT Network of Clinical Trial Sites

The goal of this project is to partner with other centers and the NIH, and to work with the Data and Clinical Coordination Centers, to implement and develop early-stage clinical trials, covering a broad range of disease entities, whether from academic, foundation or industry discoveries.

Role: Co-Investigator (URMC), Chair, Recruitment, Retention, and Diversity Committee (network-wide)


5 UL1 TR000042 (PIs: Zand and Bennett)

5 KL2 TR000095 (PIs: Zand and Bennett)

5 TL1 TR000096 (PIs: Zand and Bennett)

NIH/National Center for Research Resources and National Center for Advancing Translational Sciences

The University of Rochester’s Clinical and Translational Science Institute

The major goal of this project is to make the University of Rochester Clinical and Translational Science Institute an academic home for clinical and translational sciences, providing a centralized, integrated infrastructure.

Role: Strategic Leadership Group Member, Approach and Governance Key Function


1K12NS098482-01 (PI: Augustine)

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Child Neurologist Career Development Program

The overarching goal of this national program is to provide support for training talented, young, child neurologists interested in pursuing a clinical research career.

Role: Diversity Officer; Founder- Minority Research Scholars Program


1R01NS094292-01 (PI: Bankiewicz)

The Regents of the University of California / NINDS

A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients (AADC)

The overall objective of this study is to determine the safety and efficacy of AAV2-hAADC delivered to the substantia nigra pars compacta (SNc) and ventral tegmental area (VTA) in children with aromatic L-amino acid decarboxylase (AADC) deficiency.

Role: Medical Monitor


Active Research Support – Industry


Houston Methodist Research Institute (PI: Ashizawa)

NIH U01 Subcontract

Clinical Trial Readiness for SCA1 and SCA3

The goal of this study is to enroll preclinical and early-phase research participants with SCA1 or SCA3 repeat expansions to assist networks in preparing for clinical trials.

Role: Site Investigator


Completed Research Support

Biohaven Pharmaceutical (Schneider

BHV4157-201

A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of BHV-4157 in Adult Subjects with Spinocerebellar Ataxia

The goal of this study is to compare the efficacy of BHV-4157 versus placebo on ataxia symptoms in subjects with spinocerebellar ataxia.

Role: Site Principal Investigator


T32NS007338 (PI: Griggs)

NIH

Experimental Therapeutics of Neurological Disorders

The major goals are to train clinical neuroscientists in: 1) the clinical pathophysiologic and pharmacologic aspects of neurologic diseases, 2) the design, implementation, data management and statistical analysis of controlled clinical trials, and 3) methodologies for quantification of the natural history of nervous system disease.

Role: Fellow (7/1/08-6/30/09)


U01NS43128 (PI: Kieburtz)

NIH/NINDS

Neuroprotection Studies in PD: A Coordinating Center

This project establishes an academically based clinical trials coordination center to handle all aspects of performing trials in experimental treatments in PD.

Role: Medical Monitor (2008-2014)


K12NS066098 (PI: Mink)

NIH/NINDS

Neurological Sciences Academic Development Award

The specific aims of this project are: 1) to give junior faculty the opportunity to transition from mentored trainees to independent investigators in clinical research, 2) to foster thinking skills and a knowledge base that will allow supported candidates to apply basic neurobiological principles to achieve a mechanistic understanding of disease so they can translate them into therapeutic and diagnostic options for disorders of the developing nervous system, 3) to foster the academic careers of junior faculty by providing the guidance and framework necessary to conduct scientifically sound, ethical, and clinically relevant research in neurobiology with the mentoring necessary for career planning as future leaders in academic child neurology. Role: Scholar

 


Batten Disease Support and Research Association (PI: Augustine)

Phase II, Randomized, Placebo-Controlled Trial of Mycophenolate in JNCL

The major goal of this study is to evaluate the safety and tolerability of short-term administration of mycophenolate mofetil in ambulatory children with juvenile neuronal ceroid lipofuscinosis (JNCL).

Role: Principal Investigator. Active study conduct complete, manuscript in development.

Note – trial was supported by grants from the FDA and Batten Disease Support and Research Association


R01FD003908 (PI: Augustine)

Food and Drug Administration

Phase II, Randomized, Placebo-Controlled Trial of Mycophenolate in JNCL

The major goal of this study is to evaluate the safety and tolerability of short-term administration of mycophenolate mofetil in ambulatory children with juvenile neuronal ceroid lipofuscinosis (JNCL).

Role: Principal Investigator. Active study conduct complete, manuscript in development.

Note – trial was supported by grants from the FDA and Batten Disease Support and Research Association


International Essential Tremor Foundation (PI: Augustine)

Clinical Characteristics of Essential Tremor and Enhanced Physiologic Tremor in Childhood

The overarching aim of this study is to evaluate clinical, physiologic, and functional aspects of Essential Tremor and Enhanced Physiologic Tremor in children and adolescents.

Role: Principal Investigator


New York State Department of Health (PI: Holloway)

Empire Clinical Research Investigator Program

Optimizing Patient Journey, Improving Outcomes

The goal of this project is to promote development of clinician researchers, provide seed funding for future center grants and to improve research teams around the Center’s theme: Optimizing Patient Journey, Improving Patient Outcomes. One project of this program will be establishing clinical trial outcomes in Juvenile Batten Disease.

Role: Investigator


Horizon Pharmaceuticals, Inc. (PI: Casaceli)

A Randomized MC, DB, PC Efficacy Study of ACTIIMMUNE in Children and Young Adults with Friedreich’s Ataxia (STEADFAST)

The goal of the study is to evaluate the effect of ACTIMMUNE (interferon [IFN]-γ 1b) versus placebo on the change from Baseline to Week 26 in neurological outcome as measured by Friedreich’s Ataxia Rating Scale.

Role: Medical Monitor


NBI-98854-1403 (PI: Augustine)

Neurocrine Biosciences

A Phase 1b, Open-Label, Multiple-Dose study to Evaluate the Safety, Tolerability, Pharmacokinetics and, Pharmacodynamics of NBI-98854 in Children and Adolescents with Tourette Syndrome

The goal of the study is to evaluate the safety and tolerability of multiple, ascending (increasing) doses of NBI-98854 in pediatric subjects with Tourette Syndrome.

Role: Site Principal Investigator


Meaningful Endpoints for Phase III Clinical Trials in Juvenile Batten Disease (PI: Adams)

Batten Disease Support and Research Association

The major aim of this project is to develop clinically meaningful endpoints for efficacy (Phase III) clinical trials in Juvenile Neuronal Ceroid Lipofuscinosis.

Role: Co-Investigator


NBI-98854-1501 (PI: Augustine)

Neurocrine Biosciences

A Phase 2, randomized, double blind, placebo-controlled study to Evaluate the Safety, Tolerability, and Efficacy of NBI-98854 in Children and Adolescents With Tourette Syndrome The goal of the study is to evaluate the safety and efficacy of continued doses of NBI-98854 in pediatric subjects with Tourette Syndrome.

Role: Site Principal Investigator.



NBI-98854-1601 (PI: Augustine)

Neurocrine Biosciences

An Open-Label extension to Evaluate the Safety, Tolerability, and Efficacy of NBI-98854 in Children and Adolescents With Tourette Syndrome

The goal of the study is to evaluate the safety and efficacy of continued doses of NBI-98854 in pediatric subjects with Tourette Syndrome.

Role: Site Principal Investigator


Empire Clinical Research Investigator Program (ECRIP) (PI: Holloway)

New York State Department of Health

Utilizing New Tools to Improve Population Health

The goal of this project is to foster the development of new clinician researchers, and to unify new and existing research teams around the Center research theme of Improving Population Health through Enabling Technologies and Biomedical Informatics using new tools, in the form of telemedicine and biomedical informatics, to expand access to clinical care and to improve care delivery, across NYS.

Role: Trainee Mentor, Laurie Seltzer, DO


U54TR001456 (PI: Mink)

Dystonia Coalition / NIH

Pilot Study of Dystonia Rating Scales in Children with Primary (Isolated) Dystonia

The major goal of this study is to determine a valid and reliable clinical rating scale for childhood dystonia to improve capture of natural history studies, assess the efficacy of new therapies, and improve individual patient care by facilitating an objective assessment of disease progression and treatment response.

Role: Co-Investigator


U01DD001007 / 17-3236/PO #2000029934 (PI: Adams)

University of South Carolina’s Center for Disease Control & Prevention-funded Disability Research and Dissemination Center

Screening for Tics and Tic Disorders

The major goal of this study is to improve identification of tics, tic disorders, and co-occurring conditions in children, by evaluating new screening and diagnostic measures.

Role: Co-Investigator


17-3236/PO #2000029934 (Adams)

University of South Carolina’s CDC-funded Disability Research and Dissemination Cntr

Screening for Tics and Tic Disorders

The major goal of this study is to improve identification of tics, tic disorders, and co-occurring conditions in children, by evaluating new screening and diagnostic measures.

Role: Investigator


Batten Research Alliance (PI: Mink)

Natural History and Biorepository Database for INCL and LINCL

The major goal of this study is to establish and maintain a linked database containing quantitative natural history data and biorepository links for individuals with Infantile and Late-Infantile Forms of Neuronal Ceroid Lipofuscinosis (Batten Disease).

Role: Co-Investigator