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Open Research Studies

Below is a list and brief description of ongoing CINRG research studies that are enrolling. This list is updated at frequent intervals. Additional information is available at Clinical Trials.gov, by sending an email to info@cinrgresearch.org and requesting information on current CINRG studies.

A Phase II, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD)

Study Details: This study is evaluating the safety and tolerability of a high and low dose exon skipping intravenous intervention in patients with Duchenne muscular dystrophy. The exon skipping intervention, NS-065/NCNP-01, is targeted for the skipping of exon 53.

Inclusion Criteria and Participants: Duchenne muscular dystrophy patients aged 4 to less than 10 years old with a DMD mutation amenable to exon 53 are eligible. Approximately 16 patients will be enrolled in sites in the United States. Participants must be able to complete 3 standard time tests (10 meter walk/run, 4 step climb, and rise from the floor). There are no time limits for these timed test, just the ability to complete them during the screening visit. Participants must be on a stable dose of glucocorticoid (GC) for at least 3 months prior to study entry. Participants are expected to remain on GCs for the duration of the study.

Status: Pre-recruitment

Participants will be seen at a participating CINRG sites. Travel assistance is available to help families travel to the nearest participating site.

Participating CINRG Sites:

  1. Washington University, St. Louis, MO (Principal Investigator: Anne Connolly, MD)
  2. University of California Davis, Sacramento, CA (Principal Investigator: Craig McDonald, MD)
  3. Emory University, Atlanta, GA (Principal Investigator: Han Phan, MD)
  4. Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL (Principal Investigator: Vamshi Rao, MD)
  5. Children’s Hospital of Richmond at VCU, Richmond, VA (Principal Investigator: Amy Harper, MD)
  6. Duke University, Durham, NC (Principal Investigator: Edward Smith, MD)
  7. University of Florida, Gainesville, FL (Principal Investigator: Barry Byrne, MD)
  8. Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA (Principal Investigator: Hoda Abdel-Hamid, MD)

Questions: Please contact Sara Misplay at 412-228-0629 (smisplay@trinds.com)

Sponsor: This study sponsor is NS Pharma

This study is listed at clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT02740972

 

Title: A Phase IIa, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)

Study Details: This study will evaluate the safety and tolerability of a new steroid-like medication called vamorolone in boys with DMD ages = 4 years and < 7 years. Enrolled participants will take the study medication for 14 days followed by a 14 day follow-up period.

The Phase 2a trials are in two parts with different purposes as outlined below:

  • Phase 2a (VBP15-002)
    • Two weeks daily treatment with vamorolone at one of 4 dose levels (0.25, 0.75, 2.0, 6.0 mg/kg/day
    • Pharmacokinetics (drug metabolism), and comparison of DMD children to completed Phase 1 adult studies.
    • Short-term tolerability and safety, and comparison of DMD children to Phase 1 adults

  • Phase 2a extension (VBP15-003)
    • Six months daily treatment with vamorolone at one of 4 dose levels
    • Long-term tolerability and safety
    • Dose-finding
      • Which of the four doses show any safety signals?
      • Tested by pharmacodynamic biomarkers for adrenal suppression, bone turnover, insulin resistance, and immune suppression
      • Tested for changes in body mass index (weight gain)
      • Are the findings in DMD children same or different to Phase 1 adults?
      • Which of the four doses show hints of efficacy (benefit)?
      • Tested by improvements in timed function tests (time to stand from floor, and others)
      • Tested by exploratory pharmacodynamic efficacy biomarkers

With completion of the Phase 2a studies, we hope to have a very good understanding of the degree to which improvements in safety are seen, and a comparison of DMD children and the completed Phase 1 adult studies. We also hope to have hints of doses that show clinical improvement of DMD boys, and these doses will be carried forward into the pivotal Phase 2b studies.

Status: In recruitment.

Participating CINRG Sites:

  1. University of California Davis, Sacramento, CA (Principal Investigator: Craig McDonald, MD)
  2. University of Florida, Gainesville, Florida (Principal Investigator: Barry Byrne, MD)
  3. Nemours Children's Hospital, Orlando FL (Principal Investigator: Richard Finkel, MD)
  4. Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL (Principal Investigator: Nancy Kuntz, MD)
  5. Duke University, Durham, NC (Principal Investigator: Edward Smith, MD)
  6. University of Texas Southwestern Medical Center, Dallas, TX (Principal Investigator: Diana Castro, MD)
  7. Alberta Children's Hospital, Calgary, Canada (Principal Investigator: Jean Mah, MD)
  8. Other CINRG sites may be added to this list

Questions: Please contact Andrea Smith at 412-383-7207 (smithal7@upmc.edu)

Sponsor: This study sponsor is ReveraGen

This study is listed at clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT02760264?term=vbp15-002&rank=1 and https://clinicaltrials.gov/ct2/show/NCT02760277?term=vbp15-003&rank=1

Last Updated: November 17, 2016

 

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