The following neurology clinical trials are available for children, adolescents, and young adults. Learn more about the various studies available below, and reach out to the designated trial contact if you would like more information.

Duchenne Muscular Dystrophy (DMD) Trials

ENDEAVOR

Condition: Ambulatory Duchenne Muscular Dystrophy

Type of Research: Interventional
Principal Investigator: Crystal Proud, MD

Study Specifications: Duchenne muscular dystrophy (DMD) is an X-linked degenerative neuromuscular disease caused by mutations in the dystrophin gene. Treatment options for DMD are limited. SRP-9001 is a gene therapy designed to treat the underlying biological cause of DMD by replacing dysfunctional or missing dystrophin protein with a functional truncated dystrophin, called micro-dystrophin. ENDEAVOR aims to evaluate the safety and expression of systemic gene delivery of SRP-9001.

Point of Contact for the Study: Proud.Research@chkd.org

Limb Girdle Muscular Dystrophy

EMERGENE

Conditions: Limb Girdle Muscular Dystrophy (LGMD) Type 2E (LGMD2E/R4)

Type of Research: Interventional
Principal Investigator: Crystal Proud, MD 

Study Specifications: The limb-girdle muscular dystrophies (LGMDs) are a group of rare, genetically heterogeneous disorders involving progressive weakness and wasting of the shoulder and pelvic girdle musculature. Treatment options for LGMD Type 2E are limited. SRP-9003 is a gene therapy designed to treat the underlying biological cause of LGMD Type 2E. EMERGENE aims to evaluate the safety and expression of the systemic gene delivery of SRP-9003.

Point of Contact for the Study:  Proud.Research@chkd.org

Migraines

Dr. Ryan P. Williams is the Principal Investigator for all five of the migraine research trials being conducted at CHKD! He and his research team are investigating whether or not migraine medications that have been proven helpful for adults, are helpful for children and adolescents. For more information about any of the studies listed below, call or email CHKD’s Clinical Research Unit.

Email:CRU@chkd.org
Phone: (757) 668-5416

AbbVie “Periscope” Study

Conditions: Migraine with or without aura

Type of research: Interventional

Study Specifications: The PERISCOPE Study is evaluating an investigational product for the acute treatment of migraines in children and adolescents between the ages of 6 to 17. The investigational product is currently approved to treat migraines in adults.

You or your child may qualify for the PERISCOPE Study if you or they:

  • Are between the ages of 6-17 years old
  • Have been diagnosed by a doctor with migraine
  • Have been experiencing migraines for at least 6 months
  • Experience between 1 and 14 migraine attacks per month

Trial Website: The PERISCOPE Study | Pediatric Migraine Disease

Pfizer “Fox” Study

Conditions: Migraine with or without aura

Type of research: Interventional

Study Specifications: The Fox study is comparing the efficacy and safety of rimegepant versus placebo in the acute treatment of migraine in children and adolescents between the ages of 6 and 17.

You or your child may qualify for the Fox Study if you or they:

  • Are between the ages of 6-17 years old
  • Have been diagnosed by a doctor with migraine
  • Have been experiencing migraines for at least 6 months
  • Experience between 1 and 8 migraine attacks per month

Trial website: Study Details | Randomized Study in Children and Adolescents With Migraine: Acute Treatment | ClinicalTrials.gov

Amgen “Oasis” Studies

Conditions: Episodic or Chronic Migraine

Type of research: Interventional

Study Specifications: The Oasis studies are being conducted to evaluate the safety and efficacy of erenumab in children and adolescents with episodic OR chronic migraine.

In order to qualify for either of the Oasis studies, your child must:

  • Be between 6-17 years of age
  • Been diagnosed with episodic or chronic migraine
  • Have been experiencing migraines for at least 12 months

Trial websites:

Episodic patients: AMGEN - Efficacy and Safety of Erenumab in Pediatric Participants with Episodic Migraine (amgentrials.com)

Chronic patients: AMGEN - Efficacy and Safety of Erenumab in Pediatric Subjects With Chronic Migraine (amgentrials.com)

Lundbeck “Prospect” Studies

Conditions: Episodic or Chronic Migraine

Type of research: Interventional

Study Specifications: The goal of the Prospect-1 and Prospect-2 studies is to see if the study medicine, eptinezumab, can help reduce the number or headache days per month in children and teens with episodic OR chronic migraine.

To qualify for this study, you or your child must:

  • Be between 6-17 years of age
  • Been diagnosed with episodic or chronic migraine
  • Have been experiencing migraines for at least 6 months

Trial websites:

Episodic patients: A clinical research study for children and teens 6-17 with migraine. (prospect1migrainestudy.com)

Chronic patients: Study Details | A Study With Eptinezumab in Adolescents (12-17 Years) With Chronic Migraine | ClinicalTrials.gov

TEVA “Space” Study

Conditions: Chronic Migraine

Type of research: Interventional

Study Specifications: The TEVA Space study is evaluating the effectiveness of fremanzumab as compared to placebo for the preventative treatment of chronic migraine.

To qualify for this study, you or your child must:

  • Be between 6-17 years of age
  • Been diagnosed with chronic migraine
  • Have been experiencing migraines for at least 6 months

Trial website: NCT04464707 - Home (tevapharm.com)

Spinal Muscular Atrophy Trials

WASHU

Condition: Molecularly confirmed chromosome 5q SMA

Type of Research: Observational
Principal Investigator: Crystal Proud, MD

Study Specifications: Spinal Muscular Atrophy (SMA) is a neuromuscular disease resulting from a progressive deterioration of cells in the spinal cord and brain stem. SPINRAZA (nusinersen) was approved by the US FDA for the treatment of all types of 5q SMA and in patients of all ages. WASHU is a longitudinal, observational study aiming to assess the effectiveness of SPINRAZA on ambulation and upper extremity function.

Email: Proud.Research@CHKD.org