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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 the Research Department at (702) 862-1136, (702) 732-0975 or research@cure4thekids.org.

 

20101221 - Immune Thrombocytopenia (ITP)

Study Title: A Single Arm, Open-label, Long-term Efficacy and Safety Study of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)

Purpose: This is a phase 3b single arm, open label, multicenter study describing the percentage of time pediatric subjects with ITP have a platelet response while receiving romiplostim, defined as a platelet count ≥ 50 x 10^9/L and in the absence of ITP rescue medications in the past 4 weeks.

Closed for enrollment

Investigator: Alan Ikeda, MD
Additional information:http://clinicaltrials.gov/show/NCT02279173

 

 

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).

Open for enrollment

Investigator: Alan Ikeda, MD
Additional information:  http://clinicaltrials.gov/ct2/show/NCT00358943
Secondary Link: https://www.gauchercare.com/healthcare/registry.aspx



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®

Open for enrollment

Investigator: Alan Ikeda, MD
Additional information:http://clinicaltrials.gov/ct2/show/NCT00196742
Secondary Link: https://www.fabrycommunity.com/en/Healthcare/Registry.aspx

 


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

Open for enrollment

Investigator: Alan Ikeda, MD
Additional information: http://clinicaltrials.gov/ct2/show/NCT00144794
Secondary link: https://www.mps1disease.com/en/patients/mpsi-registry.aspx



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®.

Open for enrollment

Investigator: Alan Ikeda, MD
Additional information: http://clinicaltrials.gov/ct2/show/NCT00231400
Secondary Link: https://www.pompe.com/en/healthcare-professionals/pompe-registry.aspx

 


14374 – Venous Thromboembolism

Study Title: 30-day, Open-label, Active-controlled, Randomized Study of the Safety, Efficacy and the Pharmacokinetic and Pharmacodynamic Properties of Oral Rivaroxaban in Children With Various Manifestations of Venous Thrombosis

Purpose: The purpose of this study is to find out whether rivaroxaban is safe to use in children and how long it stays in the body. Safety will be assessed by looking at the incidence and types of bleeding events. There will also be a check for worsening of blood clots.

Open for enrollment

Investigator: Alan Ikeda, MD
Additional information: http://clinicaltrials.gov/show/NCT02309411



GENA-99 – Hemophilia A patients taking Nuwiq

Study Title: Prospective, multinational, non-interventional post-authorisation study to document the long-term immunogenicity, safety, and efficacy of Human-cl rhFVIII (simoctocog alfa) in patients with haemophilia A treated in routine clinical practice

Purpose:  To collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously treated paediatric and adult patients) and routine clinical practice.
Besides aspects such as general product safety and efficacy, there will be a focus on immunogenicity, particularly on inhibitor development. The diagnosis of FVIII inhibitor will be based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.

Open for enrollment

Investigator: Dr. Joseph Lasky
Additional Information: https://clinicaltrials.gov/ct2/show/NCT02962765?term=GENA-99&rank=1 

 

 

BI3023_4003 – Factor 1 Deficiency patients

Study Title: A Multicenter Study on the Retrospective Safety and Efficacy of Fibrinogen Concentrate (Human) (FCH) for Routine Prophylaxis, Treatment of Bleeding or Surgery in Subjects with Congenital Fibrinogen Deficiency with a Prospective Followup Component

Purpose: This is a multicenter, non-interventional, retrospective cohort study with a prospective, observational follow-up component to investigate the safety and efficacy of FCH use in subjects with congenital fibrinogen deficiency. Data will be collected on the safety and efficacy of FCH as used for the treatment of acute bleeding episodes, routine prophylaxis and perioperative bleeding in these subjects.

Open for enrollment

Investigator: Dr. Joseph Lasky
Additional Information: https://clinicaltrials.gov/ct2/show/NCT02427217

 


Boehringer Ingelheim 1160.106  – Venous Thromboembolism

Study Title: Open Label Study Comparing Efficacy and Safety of Dabigatran Etexilate to Standard of Care in Paediatric Patients With Venous Thromboembolism (VTE)

Purpose: This open-label, randomized, parallel-group, active-controlled, multi-centre non-inferiority study of dabigatran etexilate versus standard of care in children from birth to less than 18 years of age will assess the efficacy and safety of dabigatran relative to low molecular weight heparins or vitamin K antagonists for treatment of VTE. This study will also assess the appropriateness of the proposed dabigatran doses for use in paediatric patients using three different formulations of dabigatran (capsules, pellets and oral liquid formulation)

Open for enrollment

Investigator: Alan Ikeda, MD
Additional Information: https://clinicaltrials.gov/ct2/show/NCT01895777

 

 

Boehringer Ingelheim 1160.108 - Venous Thromboembolism, Secondary Prevention

Study Title: This open-label, single arm prospective cohort study will assess the safety of dabigatran etexilate in secondary prevention of venous thromboembolism in paediatric patients. Children from 0 to less than 18 years of age will be eligible to participate.

Open for enrollment

Investigator: Alan Ikeda, MD 
Additional Information: https://clinicaltrials.gov/ct2/show/NCT02197416

 

 

ALN-AT3SC-004, ALN-AT3SC-005, and ALN-AT3SC-009 – Patients with Hemophila A or B

Opening in Mid 2017

Investigator: Dr. Joseph Lasky

 

 

SA-307JG - NEUROMYELITIS OPTICA (NMO) AND NMO SPECTRUM DISORDER (NMOSD)

Study Title: A MULTICENTER, RANDOMIZED, ADDITION TO BASELINE TREATMENT, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF SA237 IN PATIENTS WITH NEUROMYELITIS OPTICA (NMO) AND NMO SPECTRUM DISORDER (NMOSD)

Purpose: This is a multicenter, randomized, addition-to-baseline treatment, double-blind, placebo-controlled, parallel assignment study followed by an open-label extension period. Patients will be randomized to either SA237 120 mg (Group A) or placebo (Group B). Blinded study drug will be administered subcutaneously (SC) at Weeks 0, 2 and 4, and thereafter every 4 weeks (Q4W) in combination with one of the following baseline treatments: azathioprine, mycophenolate mofetil or oral corticosteroids (treatment with both oral corticosteroids and either azathioprine or mycophenolate mofetil will be accepted in patients aged 12 to 17 years at the time of informed consent).

Opening in Early 2017

Investigator: Kanyalakshmi Ayyanar, MD
Additional Information: https://clinicaltrials.gov/ct2/show/NCT02028884?term=SA-307JG&rank=1



Boehringer Ingelheim 1321.7 - 

Study Title: Single Dose, Open Label, Uncontrolled, Safety Trial of Intravenous Administration of Idarucizumab to Paediatric Patients Enrolled From Ongoing Phase IIb/III Clinical Trials With Dabigatran Etexilate for the Treatment and Secondary Prevention of Venous Thromboembolism.

Purpose: The trial objective is to demonstrate the safety of idarucizumab, as assessed by the occurence of patients with drug related adverse events (including immune reactions) and all-cause mortality in paediatric venous thromboembolism patients treated with dabigatran in ongoing clinical trials who require emergency surgery/urgent procedures or patients who have life-threatening or uncontrolled bleeding which requires urgent intervention, when rapid reversal of the anticoalugant effect of dabigatran is needed.

Opening in Early 2017

Investigator: Alan Ikeda, MD
Additional Information: https://clinicaltrials.gov/ct2/show/NCT02197416

 

 

Sickle Cell Omega-3 Treatment Trial (SCOT Trial) - Sickle Cell Disease

Study Title: A Phase 3, Prospective, Randomized, Double-Blind, Placebo Controlled, Parallel-Group, Multi-center Study of SC411 for Sickle Cell Disease

Purpose: The purpose of this study is to determine whether treatment of sickle cell patients with docosahexaenoic omega-3 acid (DHA) is effective in prevention of acute sickle cell crisis.

Opening in Mid 2017

Investigator: Alan Ikeda, MD
Additional Information: https://clinicaltrials.gov/ct2/show/NCT02604368

 

 

GBT440-031 0- Sickle Cell Disease

Study Title: A Phase I Randomised, Placebo-controlled, Double-blind, Single and Multiple Ascending Dose Study of the Tolerability and Pharmacokinetics of GBT440 in Healthy Subjects and Patients With Sickle Cell Disease

Purpose: The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of GBT440 compared with placebo in healthy subjects and subjects with sickle cell disease (SCD).

Opening in Mid 2017

Investigator: Nik Abdul Rashid, MD
Additional Information: https://www.clinicaltrials.gov/ct2/show/NCT02285088



Morquio A Registry Study (110-504)

Indication: Mucopolysaccharidosis Type IVA

Description: The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of MPS IVA; to evaluate the long-term effectiveness and safety of Vimizim®, including, but not limited to, the occurrence of serious hypersensitivity reactions, anaphylaxis, and changes in antibody status; to help the medical community with the development of recommendations for monitoring MPS IVA patients and reports on patient outcomes to optimize patient care; to collect data on other treatment paradigms, and evaluate the prevalences of their use and their effectiveness; to characterize the effects and safety of Vimizim treatment 5 years from enrollment in the Registry for patients under 5 years of age; to monitor pregnancy exposure, including maternal, neonatal, and infant outcomes; and to monitor patients who have completed the MOR-005 and MOR-007 clinical trials. These patients will be encouraged to enroll in the applicable Registry Substudy and will be monitored using the MOR-005 and MOR-007 assessment schedules, respectively.

Principal Investigators: Dr. Angela Berg, Dr. Nicola Longo

Sponsor: BioMarin

Additional Information: https://clinicaltrials.gov/ct2/show/NCT02294877

 


Understanding Hemophilia A and B Drug Dosage Administration Patterns (B1821056)

Indication: Hemophilia A or B
Description: A prospective observational, cross-sectional epidemiological study in U.S. site-based clinical practice settings.
30 sites will enroll approximately 300 patients.  Participating patients - or their caregiver in the case of patients under the age of 18 - will be consented to participate.  Physicians complete a retrospective chart-review on each enrolled patient.  Patients will complete a one-time study questionnaire.

Principal Investigator: Dr. Joseph Lasky

Sponsor: Pfizer

Additional Information: https://clinicaltrials.gov/ct2/show/NCT03248141?term=B1821056&rank=1