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Trial record 11 of 413 for:    sodium phosphate

Open-label, Long-term, Extension Treatment Using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients With Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study (OLE-IEDAT)

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ClinicalTrials.gov Identifier: NCT03563053
Recruitment Status : Recruiting
First Posted : June 20, 2018
Last Update Posted : May 14, 2019
Sponsor:
Information provided by (Responsible Party):
Erydel

Brief Summary:
This is an international (North America, Europe, Africa, Asia and Australia), multi-center, prospective, open-label treatment study, designed to continue to provide the study medication to all patients who completed 12 months of treatment (including those treated with placebo) in the IEDAT-02-2015 trial, completed the study assessments, do not present safety contraindication to continuation of treatment, and provided informed consent. The study aims to collect information on the long-term safety and efficacy of the trial treatment.

Condition or disease Intervention/treatment Phase
Ataxia Telangiectasia Combination Product: EryDex System Phase 3

Detailed Description:

This is an international (North America, Europe, Africa, Asia and Australia), multi-center, prospective, open-label treatment study, designed to continue to provide the study medication to all patients who completed 12 months of treatment (including those treated with placebo) in the IEDAT-02-2015 trial, completed the study assessments, do not present safety contraindication to continuation of treatment, and provided informed consent. The study aims to collect information on the long-term safety and efficacy of the trial treatment. Patients meeting all selection criteria will receive monthly infusions of EDS-EP (dose range of ~14-22 mg DSP/infusion). If this dose of EDS-EP is not tolerated, the patient should be discontinued from the study. During the study, long-term efficacy assessments will be performed every 6 months, while safety parameters will be assessed at each monthly visit.

The ICARS, EQ-5D-5L and the CGI-C/S will be administered by a site rater. All patients enrolled in this study will have participated in Study IEDAT-02-2015, and there will be no de novo enrollment of new patients.

The Principal Investigator will ask all patients who meet the above requirements, and determine their interest in continuing to receive treatment with the study medication in a new protocol. The Principal Investigator will then determine the eligibility of the patients on the basis of his/her clinical judgement of patients' status and their safety.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Open-label, Long-term, Extension Treatment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label, Long-term, Extension Treatment Using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients With Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study
Actual Study Start Date : June 12, 2018
Estimated Primary Completion Date : August 2019
Estimated Study Completion Date : August 2019


Arm Intervention/treatment
Experimental: active drug
~14-22 mg dexamethasone sodium phosphate (DSP)
Combination Product: EryDex System
EryDex System is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EDS) creating the EDS-end product which is infused into the patients




Primary Outcome Measures :
  1. Long term safety/tolerability of EDS-EP: # of pts with treatment related Adverse Events (CTCAE4.0) [ Time Frame: 1 year ]
    AEs will be listed and summarized by body system and preferred term. Incidence of AEs(%),intensity and relatedness to study drug will be reported. SAEs and events newly occurring or worsening after administration of study medication will be summarized. AEs that result in death/discontinuation of the study medication will be listed separately

  2. Long term safety/tolerability of EDS-EP: lab parameters according to clinical practice such as hematology, biochemistry, urinalysis, HbA1c (haemoglobin A1c or glycated haemoglobin test), CD4+ lymphocytes count, CRP (C reactive protein) [ Time Frame: 1 year ]
    Values will be listed and summarized as normal or abnormal, according to local normal ranges, depending on the lab assessment (not possible to provide unique range and unit of measure due to changes from lab to lab). Abnormal and clinically notable values will be identified and listed for each parameter

  3. Long term safety/tolerability of EDS-EP: 12-lead ECG (Electrocardiography) [ Time Frame: 1 year ]
    Results will be listed and summarized as normal or abnormal, according guidelines (PR interval: < 100 msec or > 210 msec; QRS interval: < 50 msec or > 120 msec; QTc interval: > 450 msec; Heart rate: < 50 bpm (sinus bradycardia) or > 120 bpm (sinus tachycardia); Morphology: presence of T-wave inversion, abnormal R-waves, pathological Q-waves, significant ST elevation or depression). Abnormal and clinically notable values will be identified and listed

  4. Long term safety/tolerability of EDS-EP: physical and neurological exam [ Time Frame: 1 year ]
    Results will be listed and summarized as normal or abnormal, according to local assessment. Examination includes general appearance,skin,neck(including thyroid),eyes/ears,nose,mouth,throat,lungs,heart, abdomen,back,lymphnodes,extremities,nervous system. Abnormal and clinically notable values will be identified and listed for each parameter

  5. Long term safety/tolerability of EDS-EP: Columbia Suicide Severity Rating Scale (CSSRS) [ Time Frame: 1 year ]
    Outcome will be listed and summarized on the basis of the answers: answer of "yes" to any of the 6 questions may indicate a need for referral to a trained mental health professional and an answer of "yes" to questions 4, 5 or 6 indicate high-risk

  6. Long term safety/tolerability of EDS-EP: Bone Mineral Density [ Time Frame: 1 year ]
    Z-scores will be listed and summarized as normal or abnormal, following guidelines provided in the 2013 International Society for Clinical Densitometry Official Pediatric Position. Abnormal and clinically notable values will be identified and listed for each parameter

  7. Long term safety/tolerability of EDS-EP: Tanner staging [ Time Frame: 1 year ]
    The results (stages from I to V, according to the age, from 0 to 18) will be listed and summarized. Abnormal and clinically notable values will be identified and listed for each parameter

  8. Long term safety/tolerability of EDS-EP: sterility test [ Time Frame: 1 year ]
    Outcome can be Positive or negative. Results will be listed and summarized


Secondary Outcome Measures :
  1. To evaluate the long-term effect of EDS-EP on health related Quality of Life (EQ-5D-5L scale: EuroQol 5 dimension 5 level scale) [ Time Frame: 1 year ]
    Descriptive analyses will be performed to evaluate the long-term effects of EDS-EP on health-related Quality of Life (QoL), assessed by EQ-5D-5L scale. EQ-5D-5L descriptive system scoring: from 5, min/worst, to 25, best/max); EQ-VAS (EQ Visual Analogue scale) scoring: from 0, min/worst, to 100, best/max



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient completed the double-blind period in the IEDAT-02-2015 trial and must have completed the final (Visit 15/Month 12) efficacy assessments of IEDAT-02-2015.
  • Patient tolerated the study medication, without any evidence of steroid adverse events, or treatment-related severe/ serious adverse events
  • Body weight > 15 kg.
  • The patient and his/her parent/caregiver (if below the age of consent), or a legal representative, has provided written informed consent to participate. If consent is provided solely by the caregiver in accordance with local regulations, the patient must provide assent to participate in the study
  • Patient does not present safety contraindication for continuation of treatment, as determined by the Principal Investigator (PI) according to the procedures described below.

Procedure for selecting patients for further treatment in IEDAT-03-2018:

The Principal Investigator will ask all patients who meet the above requirements, and determine their interest in continuing to receive treatment with the study medication in a new protocol. The Principal Investigator will then determine the eligibility of the patients on the basis of his/her clinical judgement of patients' status and their safety.

Exclusion Criteria:

General

  1. Females that are

    1. pregnant, or are breast-feeding (for EU countries only);
    2. of childbearing potential, pregnant, or are breast-feeding (for US and Rest of World countries).

    Females of childbearing potential using adequate birth control, as determined by their Health Care Provider, will be eligible.

  2. A disability that may prevent the patient from completing all study requirements.
  3. Current participation in a clinical study with another investigational drug.

    Medical History and Current Status

  4. CD4+ lymphocytes count <400/mm3 (for patients 6 years of age) or <200/mm3 (for patients >6 years).
  5. Current neoplastic disease.
  6. Severe impairment of the immunological system.
  7. Severe or unstable pulmonary disease.
  8. Uncontrolled diabetes. Patients with diabetes that has been stabilized (i.e. no hypoglycemic or hyperglycemic episodes in the past 3 months) will be eligible.
  9. Any other severe, unstable, or serious disease or condition that in the Investigator's opinion would put the patient at risk for imminent lifethreatening morbidity, need for hospitalization, or mortality.
  10. Eligibility of patients with abnormal laboratory test values will be determined by the Investigator.
  11. Confirmed hemoglobinopathies, e.g. hemoglobin C disease, sickle cell anemia, or thalassemia.
  12. Moderate or severe renal and/or hepatic impairment.
  13. Patients who experienced moderate/ severe steroid side effects, or moderate/ severe adverse events associated with the study medication administered in the IEDAT-02 study.

    Prior/Concomitant Medication

  14. Requires treatment with an oral or parenteral steroid. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids will be permitted.
  15. Requires any other concomitant medication prohibited by the protocol.
  16. Use of any drug that is a strong inducer/inhibitor of CYP3A4.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03563053


Contacts
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Contact: Irene Maccabruni, BS +393458415028 irene.maccabruni@erydel.com
Contact: Jarno Vannucchi, BS +393440680094 jarno.vannucchi@erydel.com

Locations
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United States, California
Ataxia Center and HD Center of Excellence - UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Susan Perlman, MD         
United States, Maryland
Division of Pediatric Allergy and Immunology - Johns Hopkins Hospital Recruiting
Baltimore, Maryland, United States, 21287-3923
Contact: Howard M. Lederman, M.D., Ph.D.         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Steve Wu, MD         
United States, Texas
Department of Pediatrics Division of Child and Adolescent Neurology Mitochondrial Clinic - University of Texas Medical School Recruiting
Houston, Texas, United States, 77030
Contact: Mary Kay Koenig, MD         
Australia, Victoria
Department of Neurology Royal Children's Hospital Recruiting
Parkville, Victoria, Australia, 3052
Contact: Monique M Ryan, MD         
Belgium
Laboratory of Pediatric Immunology UZ Leuven Recruiting
Leuven, Belgium, 3000
Contact: Isabelle Meyts, MD         
Germany
Klinik für Kinder- und Jugendmedizin, Allergologie, pneumologie und Mukoviszidose, Universitätsklinikum Frankfurt Recruiting
Frankfurt, Germany, 60590
Contact: Stefan Zielen, MD         
India
National Institute of Mental Health and Neurosciences (NIMHANS) Department of Neurology Not yet recruiting
Bangalore, India, 560 029
Contact: Ravi Yadav, MD         
Vijaya Health Centre Department of Neurology Not yet recruiting
Chennai, India, 600 026
Contact: Suresh Kumar, MD         
Nizam's Institute of Medical Sciences Department of Neurology Not yet recruiting
Hyderabad, India, 500 082
Contact: Rupam Borgohain, MD         
Jaslok Hospital and Research Center Department of Pediatric Neurology Recruiting
Mumbai, India, 400 026
Contact: Anaita Udwadia-Hegde, MD         
P.D. Hinduja National Hospital and Medical Research Centre Department of Pediatric Neurology Not yet recruiting
Mumbai, India, 400016
Contact: Vrajesh Udani, MD         
Poland
Department of Clinical Immunology - The Children's Memorial Health Institute Recruiting
Warsaw, Poland, 04-730
Contact: Barbara Pietrucha, MD         
Spain
Servicio de Neurolgia Pediatrica, Hospital Materno-Infantil La Paz Recruiting
Madrid, Spain, 28046
Contact: Samuel Ignacio Pascual, MD         
Sponsors and Collaborators
Erydel

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Responsible Party: Erydel
ClinicalTrials.gov Identifier: NCT03563053     History of Changes
Other Study ID Numbers: IEDAT-03-2018
First Posted: June 20, 2018    Key Record Dates
Last Update Posted: May 14, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Dexamethasone 21-phosphate
Ataxia
Cerebellar Ataxia
Ataxia Telangiectasia
Telangiectasis
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Vascular Diseases
Cardiovascular Diseases
Spinocerebellar Ataxias
Neurocutaneous Syndromes
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Dexamethasone
Dexamethasone acetate
BB 1101
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents