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Trial record 3 of 8 for:    orphazyme

Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03836716
Recruitment Status : Enrolling by invitation
First Posted : February 11, 2019
Last Update Posted : September 16, 2020
Sponsor:
Information provided by (Responsible Party):
Orphazyme

Tracking Information
First Submitted Date  ICMJE February 7, 2019
First Posted Date  ICMJE February 11, 2019
Last Update Posted Date September 16, 2020
Actual Study Start Date  ICMJE September 19, 2019
Estimated Primary Completion Date August 1, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 2, 2019)
  • Incidence and severity of Treatment-emergent adverse events (TEAEs) [ Time Frame: Over 76 weeks ]
    Treatment-emergent adverse events (TEAEs) summarized overall by seriousness, severity and relationship to treatment
  • Mean and change from baseline to week 76 (or end of trial ) in Creatinine - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Creatinine, unit : (umol/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Bilirubin - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Bilirubin (direct, indirect, and total), unit : (umol/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Alanine aminotransferase (ALAT) - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Alanine aminotransferase (ALAT), unit: U/L. Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Aspartate aminotransferase (ASAT) - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Aspartate aminotransferase (ASAT), unit: U/L. Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Lactate dehydrogenase (LDH) - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Lactate dehydrogenase (LDH), unit: U/L. Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Gamma-glutamyltransferase (GGT) - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Gamma-glutamyltransferase (GGT), unit: U/L. Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Sodium - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Sodium, unit: mmol/L. Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Potassium - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Potassium, unit: mmol/L. Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Blood urea nitrogen (BUN) - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Blood urea nitrogen (BUN), unit : (mmol/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Calcium - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Calcium (total, albumin corrected calcium, ionised), unit : (mmol/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Albumin -(biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Albumin, unit: (g/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Total protein -(biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Total protein, unit: (g/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Creatine kinase (CK) - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Creatine kinase (CK), unit: (U/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Cystatin C - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Cystatin C, unit: (mg/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Glucose - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Glucose, unit: (mg/dL). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Lipids - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Low Density Lipoprotein (LDL), unit: (mmol/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Lipids - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in High Density Lipoprotein (HDL), unit: (mmol/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Lipids - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Triglycerides, unit: (mmol/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Cholesterol - (biochemistry) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Cholesterol, unit: (mmol/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Haemoglobin - (Haematology) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Haemoglobin, unit: (g/L). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Haematocrit - (Haematology) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Haematocrit, reference range: 0.34 - 0.48. Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Thrombocytes - (Haematology) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Thrombocytes, unit: GI/L. Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Erythrocytes - (Haematology) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Erythrocytes, unit: GI/L. Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Leucocytes - (Haematology) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Leucocytes, unit: GI/L . Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Eosinophils - (Haematology) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Eosinophils, measured by differential count in percentage (%). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Neutrophils - (Haematology) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Neutrophils, measured by differential count in percentage (%). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Basophils - (Haematology) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Basophils, measured by differential count in percentage (%). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Monocytes - (Haematology) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Monocytes, measured by differential count in percentage (%). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in Lymphocytes - (Haematology) [ Time Frame: Over 76 weeks ]
    Mean and change from baseline in Lymphocytes, measured by differential count in percentage (%). Results are based on the last on-treatment value.
  • Mean and change from baseline to week 76 (or end of trial ) in urinanalysis [ Time Frame: Over 76 weeks ]
    Clinical Safety Laboratory Tests includes a urinalysis panel measuring: Presence of blood in urine; results being positive or negative. Results are based on the last on-treatment value
  • Mean and change from baseline to week 76 (or end of trial ) in urinanalysis [ Time Frame: Over 76 weeks ]
    Clinical Safety Laboratory Tests includes a urinalysis panel measuring: Presence of protein in urine; results being positive or negative. Results are based on the last on-treatment value
  • Mean and change from baseline to week 76 (or end of trial ) in urinanalysis [ Time Frame: Over 76 weeks ]
    Clinical Safety Laboratory Tests includes a urinalysis panel measuring: Presence of glucose in urine; results being positive or negative. Results are based on the last on-treatment value
  • Mean and change from baseline to week 76 (or end of trial ) in body weight [ Time Frame: Over 76 weeks ]
    Body weight will be measured in kilogram or pound (kg or lb). Results are based on the last on-treatment value
  • Mean and change from baseline to week 76 (or end of trial ) in blood pressure [ Time Frame: Over 76 weeks ]
    Mean and change from baseline to week 76 (or end of trial ) in blood pressure measured in mmHG Results for each parameter are based on the last on-treatment value
  • Mean and change from baseline to week 76 (or end of trial ) in pulse [ Time Frame: Over 76 weeks ]
    Mean and change from baseline to week 76 (or end of trial ) in pulse measured in beats per minute (BPM). Results are based on the last on-treatment value. Results for each parameter are based on the last on-treatment value
  • Mean and change from baseline to week 76 (or end of trial ) in pulse [ Time Frame: Over 76 weeks ]
    Mean and change from baseline to week 76 (or end of trial ) in pulse measured in beats per minute (BPM) Results are based on the last on-treatment value. Results for each parameter are based on the last on-treatment value
  • Incidence of potentially clinically significant abnormalities in body temperature [ Time Frame: Over 76 weeks ]
    Number of clinically significant abnormalities in in body temperature measured in degrees Celsius or Fahrenheit. Results are based on the last on-treatment value.
  • Columbia-Suicide Severity Rating Scale (C-SSRS) over a treatment period of 76 weeks [ Time Frame: Over 76 weeks ]
    The C-SSRS is a detailed questionnaire assessing both suicidal behavior and suicidal ideation through a series of simple, plain language questions, where the scores range between 1-4 with 1 being lowest and 5 being highest of all assessments
Original Primary Outcome Measures  ICMJE
 (submitted: February 8, 2019)
  • Assess long term safety of Arimoclomol in treatment of ALS [ Time Frame: Over 76 weeks ]
    Incidence and severity of Treatment-emergent adverse events (TEAEs)
  • Assess long term safety of Arimoclomol in treatment of ALS [ Time Frame: Over 76 weeks ]
    Mean and change from Baseline to Week 76 (or end of trial) in clinical safety laboratory tests and vital signs
  • Assess long term safety of Arimoclomol in treatment of ALS [ Time Frame: Over 76 weeks ]
    Incidence of potentially clinically significant abnormalities in clinical trial safety laboratory tests and vital signs over a treatment period of 76 weeks
  • Assess long term safety of Arimoclomol in treatment of ALS [ Time Frame: Over 76 weeks ]
    Columbia-Suicide Severity Rating Scale (C-SSRS) over a treatment period of 76 weeks
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 2, 2019)
  • Time to Permanent Assisted Ventilation (PAV)/tracheostomy/death /for subjects entering this trial having completed 76 weeks of randomized treatment in ORARIALS-01 [ Time Frame: Over 76 weeks ]
    Time from baseline to event where PAV is defined as the first of 7 consecutive days on which PAV was used for >22 hours/day for subjects having completed the 76 week treatment period in ORARIALS-01
  • Change in ALS Functional Rating Scale Revised (ALSFRS-R) from Baseline to the end of the trial [ Time Frame: Over 76 weeks ]
    ALSFRS-R is an ordinal rating scale (0 through 4) used to determine the ALS patient's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score (best of 48) from 4 sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function).
  • Change in Slow Vital Capacity (SVC) from Baseline to the end of the trial (for subjects who did not meet the survival endpoint in the ORARIALS-01 trial [ Time Frame: Over 76 weeks ]
    Change in Slow Vital Capacity (a measure of breathing function) over time
Original Secondary Outcome Measures  ICMJE
 (submitted: February 8, 2019)
  • Evaluate long term efficacy of Arimoclomol treatment of ALS [ Time Frame: Over 76 weeks ]
    Time to Permanent Assisted Ventilation (PAV)/tracheostomy/death /for subjects entering this trial having completed 76 weeks of randomized treatment in ORARIALS-01
  • Evaluate long term efficacy of Arimoclomol treatment of ALS [ Time Frame: Over 76 weeks ]
    Time to death (for subjects entering this trial having met the survival endpoint of PAV or tracheostomy in ORARIALS-01
  • Evaluate long term efficacy of Arimoclomol treatment of ALS [ Time Frame: Over 76 weeks ]
    Change in ALS Functional Rating Scale Revised (ALSFRS-R) from Baseline to the end of the trial
  • Evaluate long term efficacy of Arimoclomol treatment of ALS [ Time Frame: Over 76 weeks ]
    Change in Slow Vital Capacity (SVC) from Baseline to the end of the trial (for subjects who did not meet the survival endpoint in the ORARIALS-01 trial
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial
Official Title  ICMJE Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial
Brief Summary A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS)who have completed the ORARIALS-01 trial.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
Non-randomized open label
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE Drug: Arimoclomol
Active treatment
Other Name: Arimoclomol citrate
Study Arms  ICMJE Experimental: Arimoclomol
Arimoclomol, capsule
Intervention: Drug: Arimoclomol
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: August 2, 2019)
231
Original Estimated Enrollment  ICMJE
 (submitted: February 8, 2019)
121
Estimated Study Completion Date  ICMJE August 1, 2022
Estimated Primary Completion Date August 1, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Subject is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures, or in the circumstance that the subject is incompetent, informed consent/assent is provided in accordance with local regulation and/or procedures
  • Subject has completed the ORARIALS-01 trial (i.e., met one of the surrogate survival endpoints of tracheostomy or PAV or has completed the 76 weeks randomized treatment period)
  • Subject completed ORARIALS-01 while on treatment, where on treatment is defined as having taken the last dose of IMP within 2 weeks of the End of Trial visit (whether at week 76 or prior)

Exclusion Criteria:

  • Known or suspected allergy or intolerance to the IMP (Arimoclomol or constituents)
  • Exposure to any other investigational treatment, advanced therapy medicinal product or use of any other prohibited concomitant medications
  • Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants until 3 months after last dose. Pre-menopausal women must have a negative pregnancy test prior to dosing with trial medication.
  • Any of the following medically significant conditions:

    1. Clinically significant renal or hepatic disease OR clinical laboratory assessment (results ≥ 3 times the upper limit of normal [ULN] for aspartate aminotransferase and/or alanine aminotransferase, bilirubin ≥ 2 times the ULN, or creatinine ≥ 1.5 times the ULN).
    2. Any new condition or worsening of existing condition which, in the opinion of the investigator, would put the subject at undue risk.
  • Any serious adverse event or moderate/severe adverse event from the ORARIALS-01 trial which is ongoing at the time of transitioning to ORARIALS-02 and assessed as probably related to IMP
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Canada,   France,   Germany,   Italy,   Netherlands,   Poland,   Spain,   Sweden,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03836716
Other Study ID Numbers  ICMJE ORARIALS-02
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Orphazyme
Study Sponsor  ICMJE Orphazyme
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Michael Benatar, MD PhD University of Miami
PRS Account Orphazyme
Verification Date September 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP