The Clinical Trials Unit at Kennedy Krieger focuses on studies of many disorders in the medical and mental health field.

Currently Recruiting Trials

Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy (Neurocrine)

This study is currently recruiting

Description: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy (Neurocrine).

Eligibility & Criteria

IRB #: IRB00391760

Principal Investigator: Heather Riordan, MD

Eligible Age Range: All

Gender: All

Diagnosis: Children and adults with Dyskinetic Cerebral Palsy

Click here to view study flyer.


The Balance Study – Bipolar Disorder (Allergan)

This study is currently recruiting

Description: Bipolar depression can change the way a child or teen interacts with the world around them. If you or your child have bipolar depression, we are inviting you to take part in the Balance Study for children and teens, aged 10 to 17 years of age, who have been struggling with bipolar depression. The purpose of this study is to evaluate an investigational drug for bipolar depression in children and teens. The Balance Study will enroll about 380 children and teens with bipolar depression at participating study centers throughout North America and Europe.  

Eligibility & Criteria

IRB #: IRB00281389

Principal Investigator: Bradley Grant, DO

Eligible Age Range:  10 - 17 years

Gender: All

Diagnosis: Bipolar Disorder 
Click here to view study flyer.


A Randomized, Double-Blind, Placebo-Controlled Study in Male Adolescents with Fragile X Syndrome (TETRA 204)

This study is currently recruiting

Description: A Randomized, Double-Blind, Placebo-Controlled, Two-Part Study of BPN14770 in Male Adolescents (Aged 9 To <18 Years) with Fragile X Syndrome.

Eligibility & Criteria

IRB #: IRB00312157

Principal Investigator: Dejan B. Budimirovic, MD

Eligible Age Range: 12 to 18 years

Gender: Male only

Diagnosis: Fragile X Syndrome


A Randomized, Double-Blind, Placebo-Controlled Study in Male Adults with Fragile X Syndrome (TETRA 301)

This study is currently recruiting

Description: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study ofBPN14770 in Male Adults (Aged 18 to 45) with Fragile X Syndrome.

Eligibility & Criteria

IRB #: IRB00353081

Principal Investigator: Dejan B. Budimirovic, MD

Eligible Age Range:  18 to 45 years

Gender: Male Only

Diagnosis: Fragile X Syndrome


ZYN002 Administered as a Transdermal Gel to Children, Adolescents, and Young Adults for Treatment of Fragile X Syndrome

This study is currently recruiting

Description:  A Randomized, Double-Blind, Placebo-Controlled, Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children, Adolescents, and Young Adults with Fragile X Syndrome – RECONNECT

Eligibility & Criteria

IRB #: IRB00281916

Principal Investigator: Dejan B. Budimirovic, MD

Eligible Age Range:  Children, Adolescents, and Young Adults

Gender: All

Diagnosis: Fragile X Syndrome

Click here to view study flyer.


Forward-March Fragile X Study

This study is currently recruiting

Description:  FORWARD-MARCH is the next step following a highly successful research study called FORWARD (Fragile X Online Registry With Accessible Research Database) that was started in 2012. The FORWARD study created the largest database of information on fragile X syndrome (FXS) in the United States. Data from the FORWARD study is being used by researchers to learn about the lives of people with FXS, including medication use, behaviors, and development over time, and share findings with other researchers and clinicians.

Eligibility & Criteria

IRB #: 00337864

Principal Investigator: Dejan B. Budimirovic, MD

Eligible Age Range: Individuals born between 2003-2017

Gender: All

Diagnosis: Fragile X Syndrome


Amgen Osteogenesis Imperfecta Study

This study is currently recruiting.

Description:  A Phase 3, Open-label, Multicenter, Randomized Study to Evaluate the Efficacy and Safety of Romosozumab Compared with Bisphosphates in Children and Adolescents with Osteogenesis Imperfecta

Eligibility & Criteria

IRB #:  IRB00397371

Principal Investigator: Malinda Wu, MD

Eligible Age Range: 5 to 18

Gender: All 

Diagnosis: Osteogenesis Imperfecta


Multimodal brain imaging of the neural effects of methylphenidate in patients with ADHD

This study is currently recruiting.

Description: This research study is being conducted to evaluate how the study drug, methylphenidate, affects brain activity and brain chemistry in righthanded children ages 6-17 years with ADHD. The study consists of three onsite visits and involves neuropsychological testing along with functional MRI and magnetic resonance spectroscopy (MRS) scans after a single dose of each treatment (i.e., placebo, low dose of methylphenidate, and higher dose). Eligible participants may earn up to $300 for completing the study.

Eligibility & Criteria

IRB #: IRB00276357

Principal Investigator: Bradley Grant, DO

Eligible Age Range: 6 to 17 years

Gender: All

Diagnosis: ADHD

Click here to view study flyer.


Guanfacine for Hyperactivity in Children with Down Syndrome (HYP01; HYPEbeGONE_DS)

This study is currently recruiting. 

Description: This research study is being conducted to determine the efficacy of guanfacine immediate release (GIR) for the treatment of hyperactivity/impulsivity and inattention in children with Down Syndrome (DS) ages 6-12 years old after 8 weeks of treatment. 

Eligibility & Criteria

IRB #: IRB00435979 

Principal Investigator: George Capone, MD 

Eligible Age Range: 6 to 12 years Gender: All 

Diagnosis: Non-mosaic Down Syndrome, ADHD symptoms

Click here to view study flyer.


Cerebral Adrenoleukodystrophy (CALYX) 

This study is currently recruiting.

Description: A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects with Cerebral Adrenoleukodystrophy 

Eligibility & Criteria IRB #: IRB00382750 

Principal Investigator: Eric Mallack, MD 

Eligible Age Range: 18+ Gender: Male 

Diagnosis: Cerebral Adrenoleukodystrophy 


Active, but not enrolling:

A Phase 1b, Single Ascending Dose, Randomized, Double-Blind Study to Evaluate SAR439459 for the Treatment of Osteogenesis Imperfecta in Adults

This study is not currently recruiting

Eligibility & Criteria

IRB #: IRB00316494

Principal Investigator: Malinda Wu, MD

Eligible Age Range: 18 years and older

Diagnosis: Osteogenesis Imperfecta


Ultragenyx Study to Assess the Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta

This study is not currently recruiting.

Description: An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single-Blind, Dose-Evaluation Phase and Phase 3 Double-Blind, Placebo-Controlled Phase to Assess The Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta (ULTRAGENYX)

Eligibility & Criteria
IRB #: IRB00316439
Principal Investigator: Malinda Wu, MD
Eligible Age Range: Ages 5 to 26
Gender: All
Diagnosis: Osteogenesis Imperfecta

Epidiolex® For the Treatment Of Anxiety Comorbidity In Refractory Pediatric Epilepsy (EAPE)

This study is not currently recruiting

Description: This research study titled, Epidiolex® For the Treatment Of Anxiety Comorbidity In Refractory Pediatric Epilepsy (EAPE), is being conducted by Dr. Jay Salpekar to evaluate if the drug FDA approved drug, Epidiolex®, helps control anxiety and mood symptoms associated with recurrent seizures in addition to seizure control. The length of the study is 4 months and consists of 11 visits; some are remote and some are onsite.

If you or a family member have any questions or are interested in learning more about the study please contact the study staff by replying to this notice or by contacting the staff directly with the information below.

Eligibility & Criteria

IRB #: IRB00282248

Principal Investigator: Dr. Jay Salpekar

Eligible Age Range: 6-17 years

Gender: All

Diagnosis: Anxiety, Anxiety Disorder, Epilepsy, Seizures

Click here to view study flyer.


Perinatal Arterial Stroke: A Multi-site RCT of Intensive Infant Rehabilitation (I-ACQUIRE)

This study is not currently recruiting

Description: I-ACQUIRE, a form of constraint-induced movement therapy (CIMT), is being evaluated - in two dosages – compated to typical treatments for children with hemiparesis, such as one hour weekly each of occupational therapy and physical therapy. Both dosages involve therapy fice days a week for four consecutive weeks, provided by a therapist trained in I-ACQUIRE. You’re infant or toddler may be eligible if he or she had a stroke very early in life and shows motor weakness or poor movement control on one side (hemiparesis.

Eligibility & Criteria

IRB #: IRB00219451

Principal Investigator: Dr. Joan Carney

Eligible Age Range: 8 months-36 months

Gender: All

Diagnosis: Perinatal Arterial Ischemic Stroke (PAIS)

Click here to view study flyer.


Contact Us

1741 Ashland Avenue
Baltimore, MD 21205
Phone: (443) 923-3850
Email: ResearchTrials@KennedyKrieger.org

Participate in Research

To learn more about open research studies at Kennedy Krieger Institute, please visit our Participate in Research page.