Open Protocols

The Children’s Specialty Center of Nevada, a program of Cure 4 the Kids Foundation is currently involved with the following studies.  If you would like more information on these studies, how to get involved,  or want to find out about other studies for which you might qualify, please contact Jessica Long, Clinical Research Associate at (702) 732-0975 or jlong@cure4thekids.org.

 

Systemic Juvenile Idiopathic Arthritis (sJIA) Study (NP25737)

Study Title: A Phase I Pharmacokinetic and Safety Study of Tocilizumab (TCZ) in Patients Less Than 2 Years Old With Active Systemic Juvenile Idiopathic Arthritis (sJIA)

Purpose: This study will investigate the pharmacokinetics and safety of RoActemra/Actemra (tocilizumab) in patients less than 2 years old with active systemic juvenile idiopathic arthritis. Patients will receive RoActemra/Actemra infusions every 2 weeks. The anticipated time on study treatment is 12 weeks.

Investigator: Jonathan Bernstein, MD

Date Opened for Enrollment: August 9, 2012

ClinicalTrials.Gov: http://clinicaltrials.gov/ct2/show/NCT01455701

 

Pompe Study (AGLU07510)

Study Title: A Phase 4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Purpose: A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 L and 4000 L scales in patients who have been diagnosed with infantile-onset Pompe disease. Patients will be treated with alglucosidase alfa 160 L scale product in the US and 4000 L scale product in the regions outside the US.

Investigator: Jonathan Bernstein, MD

Date Opened for Enrollment: August 9, 2012

ClinicalTrials.Gov: http://clinicaltrials.gov/ct2/show/NCT01597596

 

Sickle Cell Disease Study (GLUSCC09-01)

Study Title: A Phase III, Prospective, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of L-Glutamine Therapy for Sickle Cell Anemia and Sickle βo-thalassemia

Purpose: The purpose of this research is to test the effects of L-glutamine on red blood cells of patients with Sickle Cell Anemia or Sickle ß0-Thalassemia. Glutamine is a part of protein and is a part of a person’s normal diet. It is often used as a nutritional supplement or as medication for various medical problems such as intestinal problems. The benefits of participating in this study may be a decrease in pain and other problems caused by sickle cell disease.

Investigator: Alan Ikeda, MD

Closed for Enrollment

ClinicalTrials.Gov: http://clinicaltrials.gov/ct2/show/NCT01179217

 

Immune Thrombocytopenia (ITP) Study (20080279)

Study Title: A Phase 3 Randomized, Double Blind, Placebo Controlled Study to Determine the Safety and Efficacy of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)

Purpose: The purpose of this study is to evaluate the efficacy of romiplostim in the treatment of thrombocytopenia in pediatric subjects with Immune Thrombocytopenia Purpura (ITP) as measured by durable platelet response.

Investigator: Alan Ikeda, MD

Date Opened for Enrollment: May22, 2012

ClinicalTrials.Gov: http://clinicaltrials.gov/ct2/show/NCT01444417

 

Immune Thrombocytopenia (ITP) Open-Label Study (20090340)

Study Title: An Open Label Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune (Idiopathic) Thrombocytopenia Purpura (ITP)

Purpose: This is an extension study designed to assess the safety and durability of platelet count increases with romiplostim treatment of thrombocytopenic subjects with Immune (Idiopathic) Thrombocytopenia Purpura. This study is available to pediatric subjects who have completed a previous romiplostim ITP study and meet the eligibility criteria of this study.

Investigator: Alan Ikeda, MD

Date Opened for Enrollment: December 6, 2012

ClinicalTrials.Gov: http://clinicaltrials.gov/show/NCT01071954

 

International Collaborative Gaucher Group (ICGG) Gaucher Registry (10-090)

 Study Title: International Collaborative Gaucher Group (ICGG) Gaucher Registry

Purpose: The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient’s treating physician.

The objectives of the Registry are:

  • To enhance the understanding of the variability, progression, and natural history of Gaucher disease with the ultimate goal of better guiding and assessing therapeutic interventions;
  • To provide the Gaucher medical community with recommendations for monitoring patients and to provide reports on patient outcomes to help optimize patient care; and
  • To evaluate the long-term effectiveness of enzyme replacement therapy (ERT).

Investigator: Jonathan Bernstein, MD

Date Opened for Enrollment: June 8, 2012

ClinicalTrials.Gov: http://clinicaltrials.gov/ct2/show/NCT00358943

 

Fabry Disease Registry (10-091)

Study Title: Fabry Disease Registry

Purpose: The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient’s treating physician.

The objectives of the Registry are:

  • To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease;
  • To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
  • To characterize and describe the Fabry population as a whole; and
  • To evaluate the long-term safety and effectiveness of Fabrazyme®

Investigator: Jonathan Bernstein, MD

Date Opened for Enrollment: June 8, 2012

ClinicalTrials.Gov: http://clinicaltrials.gov/ct2/show/NCT00196742

 

Mucopolysaccharidosis I (MPS I) Registry (10-092)

Study Title: Mucopolysaccharidosis I (MPS I) Registry

Purpose: The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities.

The objectives of the Registry are:

  • To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase)
  • To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I
  • To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care

Investigator: Jonathan Bernstein, MD

Date Opened for Enrollment: June 8, 2012

ClinicalTrials.Gov: http://clinicaltrials.gov/ct2/show/NCT00144794

 

Pompe Disease Registry (10-093)

Study Title: Pompe Disease Registry

Purpose: The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient’s treating physician.

The objectives of the Registry are:

  • To enhance the understanding of the variability, progression, and natural history of the key manifestations of Pompe disease;
  • To assist the Pompe medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
  • To characterize and describe the Pompe disease population as a whole; and
  • To evaluate the long-term effectiveness and safety of available treatment options including ERT with Myozyme®.

Investigator: Jonathan Bernstein, MD

Date Opened for Enrollment: June 8, 2012

ClinicalTrials.Gov: http://clinicaltrials.gov/ct2/show/NCT00231400