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Trial · Interventional

CompletedPhase 2Results postedRegistry data

A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis

Overview

Status
Completed
Phase
Phase 2
Enrollment
43 participants (actual)
Sponsor
Mallinckrodt · Industry
Design
Randomized · Open label (no masking)
Countries
1 country

United States

Start
Jul 2013
Primary completion
Nov 2014
Completion
Dec 2014
First posted
24 Jul 2013 (estimated)
Last update posted
6 Jan 2017 (estimated)
Conditions
Amyotrophic Lateral Sclerosis

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
201320142015
  1. Ph 2

    A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis

    NCT01906658

    Jul 2013 to Dec 2014

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

This 8-week randomized, open-label evaluation will examine the acute safety and tolerability of 4 different dosing regimens of Acthar to inform dose selection for future studies of Acthar in patients with Amyotrophic Lateral Sclerosis (ALS). The study will also investigate the mean rate of change in the ALSFRS-R total score as an exploratory endpoint to help design future studies. This study will enroll up to 40 patients and include an optional 28-week open-label extension period plus a 3-week treatment taper and 1-week follow up period. After completion of Week 8, patients enrolled in a treatment group that is considered safe and tolerable at that time have the option to continue into the open-label extension period. A 3-week treatment taper and a follow-up visit are planned for all patients enrolled in the study, beginning either at Week 8 or at Week 36 if a patient continues into the optional open-label extension period.

Interventions

  • Repository corticotropin injectionDrug

Arms4

  • Experimental

    Acthar 80 U (1.0 mL) SC twice weekly

    Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly

  • Experimental

    Acthar 24 U (0.3 mL) SC daily

    Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily

  • Experimental

    Acthar 56 U (0.7 mL) SC twice weekly

    Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly

  • Experimental

    Acthar 16 U (0.2 mL) SC daily

    Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication

    Time frame · Baseline to Week 8

Secondary outcomes(3)
  1. Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation

    Time frame · Baseline to Week 8

  2. Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication

    Time frame · Baseline to Week 8

  3. Proportion of Subjects With Treatment Emergent Suicidality

    Time frame · Baseline to Week 36

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· Baseline to Week 8

Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication

GroupValue (Participants)
Acthar 80 U (1.0 mL) SC Twice Weekly2
Acthar 24 U (0.3 mL) SC Daily0
Acthar 56 U (0.7 mL) SC Twice Weekly2
Acthar 16 U (0.2 mL) SC Daily0
Secondary and other outcome results(3)
Secondary outcomeCount of participants· Baseline to Week 8

Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation

GroupValue (Participants)
Acthar 80 U (1.0 mL) SC Twice Weekly2
Acthar 24 U (0.3 mL) SC Daily0
Acthar 56 U (0.7 mL) SC Twice Weekly2
Acthar 16 U (0.2 mL) SC Daily0
Secondary outcomeCount of participants· Baseline to Week 8

Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication

GroupValue (Participants)
Acthar 80 U (1.0 mL) SC Twice Weekly0
Acthar 24 U (0.3 mL) SC Daily0
Acthar 56 U (0.7 mL) SC Twice Weekly0
Acthar 16 U (0.2 mL) SC Daily0
Secondary outcomeCount of participants· Baseline to Week 36

Proportion of Subjects With Treatment Emergent Suicidality

GroupValue (Participants)
Acthar 80 U (1.0 mL) SC Twice Weekly1
Acthar 24 U (0.3 mL) SC Daily3
Acthar 56 U (0.7 mL) SC Twice Weekly0
Acthar 16 U (0.2 mL) SC Daily0

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Acthar 80 U (1.0 mL) SC Twice WeeklyActhar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks–2 / 1111 / 11
Acthar 24 U (0.3 mL) SC DailyActhar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks–3 / 1111 / 11
Acthar 56 U (0.7 mL) SC Twice WeeklyActhar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks–1 / 109 / 10
Acthar 16 U (0.2 mL) SC DailyActhar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks–1 / 1111 / 11

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 18 Years up to 80 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * Able to provide informed consent. * Diagnosis of clinically definite ALS, clinically probable-laboratory supported ALS, clinically probable ALS, or clinically possible ALS based on the revised El Escorial criteria. * Patients with ALS ≤ 3 years since symptom onset. Symptom onset is defined as date of first muscle weakness or dysarthria. * Upright slow vital capacity (SVC)≥ 60% of predicted. * If taking riluzole and/or Nuedexta®, stable regimen is required for ≥ 30 days prior to screening. * Medically (either independently or with caregiver assistance) able to comply with study procedures, including subcutaneous (SC) injections of study medication and adherence to concomitant medication restrictions. Exclusion Criteria: * Any medical condition known to have an association with motor neuron dysfunction which might confound or obscure the diagnosis of ALS. * Tracheostomy, diaphragm pacing, or ongoing need for assisted ventilation of any type (e.g., bilevel positive airway pressure) for treatment of ALS-related respiratory dysfunction (vital capacity of \< 60% predicted, nocturnal desaturation, and/or nocturnal hypoventilation). Patients on assisted ventilation for other reasons require approval from the Medical Monitor. (Supplemental oxygen is acceptable). * Recorded diagnosis or evidence of major psychiatric disorder. * Clinically evident cognitive and/or behavioral impairment that in the opinion of the Investigator would impair the ability of the patient to comply with the study procedures. * Therapies and/or Medications: 1. History of prior sensitivity to Acthar or other porcine protein products. 2. Chronic systemic corticosteroid use, defined as \> 20 mg of prednisone or equivalent systemic corticosteroid taken for more than 4 consecutive weeks within 6 months prior to randomization. Topical, inhaled, or intra-articular corticosteroids are allowed. 3. Planned treatment with live or live attenuated vaccines once enrolled in the study. * Participation in another therapeutic (drug or device) investigational study within 30 days prior to screening. * Type 1 or type 2 diabetes mellitus, or patients currently taking hypoglycemic medication. * Contraindication per Acthar Prescribing Information, Appendix D Section 4: scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, or adrenal cortical hyperfunction. 1. For the purposes of this study, osteoporosis is defined as a history of a lumbar spine and/or femoral neck T-score ≤ -2.5 on bone densitometry (DXA), OR osteoporosis requiring pharmacologic therapy, OR a history of non-traumatic low impact hip or vertebral fracture, OR patient reported history of osteoporosis. 2. For the purposes of this study, history of peptic ulcer is defined as ≤ 6 months prior to screening. 3. For the purposes of this study, uncontrolled hypertension is defined as mean systolic blood pressure ≥ 140 mmHg and diastolic blood pressure ≥ 90 mmHg on ≥ 3 seated readings taken at least 5 minutes apart during the screening period. 4. For the purposes of this study, congestive heart failure is defined as New York Heart Association Functional Class III-IV.

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