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Rare Disease Research, LLC | Atlanta, GA

Research site
(Unclaimed)
Location
1730 Northeast Expressway NE, Atlanta, Georgia, United States of America

Site insights

Top conditions

Top treatments

Galcanezumab
Ataluren
GTX-102
EDG-5506
Cannabidiol
NNZ-2591
Ecopipam
BBP-418
Ribitol
Risdiplam

Data sourced from clinicaltrials.gov

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Active trials

21 of 23 total trials

A Phase 2b Study of Zagociguat in Patients With MELAS (PRIZM)

PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral z...

Not yet enrolling
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Drug: zagociguat 30mg
Drug: zagociguat 15mg

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a ce...

Enrolling
Genetic Diseases, X-Linked
Muscular Diseases
Biological: Placebo
Biological: CAP-1002

The main purpose of this study is to evaluate the efficacy and safety of galcanezumab for the preventive treatment of chronic migraine in participant...

Enrolling
Chronic Migraine
Drug: Galcanezumab
Drug: Placebo

The main purpose of this study is to evaulate the efficacy and safety of galcanezumab in participants 6 to 17 years of age for the preventive treatme...

Enrolling
Episodic Migraine
Drug: Placebo
Drug: Galcanezumab

The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; I...

Active, not recruiting
Metachromatic Leukodystrophy (MLD)
Drug: SHP611

The primary objective of the study is to evaluate the safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT)...

Active, not recruiting
Angelman Syndrome
Drug: GTX-102

The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to \[...

Enrolling
Tourette Syndrome
Drug: Ecopipam

This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of Cannabidiol administered as ZYN00...

Enrolling
Fragile X Syndrome
Drug: Placebo
Drug: ZYN002 - transdermal gel

This Phase 3 multicenter study evaluates the maintenance of efficacy, safety and tolerability of ecopipam tablets in children, adolescents and adults...

Enrolling
Tourette Disorder
Drug: Ecopipam Hydrochloride

The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

Begins enrollment in 1 month
Angelman Syndrome
Drug: GTX-102

A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based...

Enrolling
Spinal Muscular Atrophy
Drug: Risdiplam

The purpose of this study is to test the long-term safety of rimegepant in the acute treatment of moderate or severe migraine in children and adolesc...

Enrolling
Acute Treatment of Migraine
Drug: Rimegepant / BHV3000

EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional meas...

Invitation-only
Becker Muscular Dystrophy
Drug: Sevasemten

A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON s...

Enrolling
Becker Muscular Dystrophy
Drug: Placebo
Drug: Sevasemten 10 mg

The purpose of this study is to test the safety and efficacy of BHV-3000 versus placebo in the acute treatment of moderate or severe migraine in chil...

Enrolling
Pediatric Migraine
Drug: Rimegepant/BHV3000
Drug: Matching placebo
Locations recently updated

The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intraven...

Enrolling
Duchenne Muscular Dystrophy (DMD)
Drug: Placebo
Drug: DYNE-251

The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participan...

Enrolling
Myotonic Dystrophy 1
Other: Placebo
Drug: PGN-EDODM1

The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDB...

Invitation-only
Duchenne Muscular Dystrophy
Drug: Ataluren

Migraine is a common neurological disorder typically characterized by attacks of throbbing, moderate to severe headache, often associated with nausea...

Enrolling
Migraine
Drug: Placebo-Matching Ubrogepant
Drug: Ubrogepant

SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous...

Active, not recruiting
Mitochondrial Myopathies
Mitochondrial DNA Mutation
Drug: Placebo
Drug: Elamipretide

Trial sponsors

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