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Rare Disease Research

For the NIH clinical research network, see Rare Diseases Clinical Research Network.
Rare Disease Research
AbbreviationRDR
FounderHan C. Phan
Location
  • United States

Rare Disease Research (RDR) is an independent U.S. clinical research site network that specializes in the conduct of Phase I-IV clinical trials for rare disease indications. RDR specializes in pediatric clinical trials away from the hospital environment and has completed more than 50 clinical trials in multiple rare diseases, including mitochondrial myopathy, myasthenia gravis, multiple system atrophy, and Duchenne muscular dystrophy (DMD).[1][2]

History

Established in 2017, RDR is headed by Han C. Phan, a sleep specialist and associate professor of neurology at the University of Alabama at Birmingham.[3] RDR's three clinical trial sites in North Carolina (Raleigh-Durham),[4][5] Georgia (Atlanta),[6] and Florida (Orlando) are led by clinical investigators with medical specialties in Pediatric Neurology Pediatric Emergency Medicine and Pediatric Cardiology.

In addition to its contribution toward the FDA approval of five therapies to date, RDR has more than 60 clinical trials in the active or beginning phases and accepts patients from all over the United States, and, in many but not all clinical trials, also patients from other countries.[7]

Clinical trial principal investigators affiliated with RDR include the pediatricians Edward Smith, Renata Shih,[8] Anthony Giordano, and Scott Batchelor.[9]

Clinical trial indications

RDR conducts clinical trials in therapeutic areas, including:

Clinical trial sites

RDR has three sites in the U.S. Each site contains a physical therapy center accommodating a gait and partially-weighted ambulation lab and testing resources, including electrocardiography, spirometry, and X-ray capabilities. In addition, each site has a research compounding pharmacy with a controlled mixing environment and storage for refrigerated and room-temperature investigational IV or oral products.[34]

Each site is staffed by nurses, researchers, physical therapists, and other personnel. Additional capabilities include IP Pharmacy, onsite labs including for gene therapies, and RDR's research home health service.[34]

RDR has contracting and Central IRB services to facilitate first subject enrollment within 30–60 days from the beginning of the study date. In addition, RDR coordinates with local providers to offer services for study participants that include imaging, ophthalmology, access to psychology and psychiatry services, and local laboratories.[35]

Partnerships

RDR partners with various contract research organizations (CROs). It is a member of PPD Site Select, PPD Pediatric Investigator Network, Parexel Site Alliance, and works closely with Syneos Health, IQVIA, Worldwide Clinical Trials, Medpace, and many other CROs.[35]

RDR also collaborates with various organizations, including Parent Project Muscular Dystrophy, the Muscular Dystrophy Association, Cure SMA, Friedreich's Ataxia Research Alliance, CureDuchenne,[36] the FSHD Society, the Prader-Willi Syndrome Association, Angelman Syndrome Foundation, Foundation for Angelman Syndrome Therapeutics,[37] Cure VCP, the Jett Foundation, and many others.

References

  1. ^ "Rare Diseases Require Unique Approaches In Clinical Trial Design". www.clinicalleader.com.
  2. ^ Rare Disease Research: Parent Project Muscular Dystrophy. April 2020.
  3. ^ "Han Cam Phan | Children's Healthcare of Atlanta".
  4. ^ Dystrophy, Parent Project Muscular (November 7, 2023). "PPMD Provides $250,000 in Funding to Rare Disease Research to Establish New Clinical Trial Site". Parent Project Muscular Dystrophy.
  5. ^ Dystrophy (PPMD), Parent Project Muscular. "Parent Project Muscular Dystrophy Provides $250,000 in Funding to Rare Disease Research, LLC to Establish New Clinical Trial Site". www.prnewswire.com.
  6. ^ "Georgia Becomes 22nd State to Establish a Rare Disease Advisory Council - National Organization for Rare Disorders". rarediseases.org. May 6, 2022.
  7. ^ "Clinical Trials". Rare Disease Research.
  8. ^ "Renata Shih » Powell Center for Rare Disease Research and Therapy » College of Medicine » University of Florida".
  9. ^ "Meet Our Team". Rare Disease Research.
  10. ^ "A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy". clinicaltrials.gov. February 4, 2025.
  11. ^ a b c d e "A Phase 2, Prospective, Interventional, Open-Label, Multi-Site, Extension Study to Assess the Long-Term Safety and Tolerability of Soticlestat (TAK-935) as Adjunctive Therapy in Subjects With Developmental Epileptic Encephalopathies Including Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome (ENDYMION 1)". clinicaltrials.gov. February 20, 2024.
  12. ^ "A Randomized, Double-Blind, Placebo-Controlled Study of TEV-50717 (Deutetrabenazine) for the Treatment of Dyskinesia in Cerebral Palsy in Children and Adolescents". clinicaltrials.gov. August 14, 2023.
  13. ^ "An Open-Label, Long-Term Safety, Tolerability, and Efficacy Study of TEV-50717 (Deutetrabenazine) for the Treatment of Dyskinesia in Cerebral Palsy in Children and Adolescents (Open RECLAIM-DCP)". clinicaltrials.gov. April 10, 2024.
  14. ^ "CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD)". clinicaltrials.gov. August 19, 2024.
  15. ^ "A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1, Followed by an Open-Label Extension". clinicaltrials.gov. February 2, 2024.
  16. ^ "A Multicenter Open-Label Study on the Safety and Efficacy of Deflazacort (Emflaza) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)". clinicaltrials.gov. May 31, 2022.
  17. ^ "A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)". clinicaltrials.gov. September 25, 2024.
  18. ^ "A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy". clinicaltrials.gov. May 7, 2024.
  19. ^ "A Phase 2a, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study With an Open-Label Extension of RVT-1401 in Myasthenia Gravis Patients". clinicaltrials.gov. November 30, 2023.
  20. ^ "A Phase 2, Randomised, Double-blind, Placebo-controlled, 2-way Crossover Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 in Ambulatory Adults With Type 3 Spinal Muscular Atrophy". clinicaltrials.gov. October 22, 2024.
  21. ^ "A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)". clinicaltrials.gov. October 22, 2024.
  22. ^ "A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients with Angelman Syndrome (AS)". clinicaltrials.gov. January 30, 2025.
  23. ^ "An Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of ZYN002 Administered As a Transdermal Gel to Children and Adolescents with Fragile X Syndrome". clinicaltrials.gov. December 17, 2024.
  24. ^ "A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Single-attack Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Oral Ubrogepant in the Acute Treatment of Migraine With or Without Aura in Children and Adolescents (Ages 6-17)". clinicaltrials.gov. February 3, 2025.
  25. ^ "A Phase 3, Randomized, Double-Blind, Placebo-controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients with Prader-Willi Syndrome". clinicaltrials.gov. November 7, 2024.
  26. ^ "Multicenter, Placebo-Controlled, Double-Blind, Randomized, Parallel-Group, Phase 2b Study to Evaluate the Efficacy and Safety of Ecopipam in Children and Adolescents With Tourette's Syndrome". clinicaltrials.gov. September 8, 2023.
  27. ^ "A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy, Safety, and Tolerability of LP352 in the Treatment of Seizures in Children and Adults With Developmental and Epileptic Encephalopathies". clinicaltrials.gov. January 13, 2025.
  28. ^ "Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)". clinicaltrials.gov. December 22, 2023.
  29. ^ "A Phase 1, First-In-Human, Multiple Part, Single Ascending and Multiple Dose Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria". clinicaltrials.gov. July 27, 2024.
  30. ^ "Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): a Double-blind, Randomized, Placebo-controlled Study". clinicaltrials.gov. January 10, 2025.
  31. ^ "A Phase 3 Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension". clinicaltrials.gov. January 16, 2022.
  32. ^ "A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) NuPower". clinicaltrials.gov. December 9, 2023.
  33. ^ "Phase 2b Randomized, Double-blind, Placebo-controlled Crossover Study Evaluating the Efficacy and Safety of Zagociguat in Participants with MELAS (PRIZM)". clinicaltrials.gov. January 23, 2025.
  34. ^ a b "Facilities". Rare Disease Research.
  35. ^ a b "Partners". Rare Disease Research.
  36. ^ "Video Archives".
  37. ^ "Angelman Syndrome". Rare Disease Research.
Quelle: https://en.wikipedia.org/wiki/Rare_Disease_Research