Amyotrophic Lateral SclerosisPhase 2NCT01906658
A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis
A Phase 2 study testing Repository corticotropin injection in people with Amyotrophic Lateral Sclerosis, run by Mallinckrodt at 17 sites in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Repository corticotropin injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 43 participants
- Where
- 17 sitesUnited States
- Study dates
- Jul 2013to Dec 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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:
- History of prior sensitivity to Acthar or other porcine protein products.
Show all 15Show fewer
- 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.
- 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.
- 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.
- For the purposes of this study, history of peptic ulcer is defined as ≤ 6 months prior to screening.
- 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.
- For the purposes of this study, congestive heart failure is defined as New York Heart Association Functional Class III-IV.
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
United States17 sites
Questcor Investigational Site
Atlanta, Georgia, 30322
Questcor Investigational Site
Birmingham, Alabama, 35233
Questcor Investigational Site
Dallas, Texas, 75214
Questcor Investigational Site
Hershey, Pennsylvania, 17033
Questcor Investigational Site
Houston, Texas, 77030
Questcor Investigational Site
Jacksonville, Florida, 32224
Questcor Investigational Site
Kansas City, Kansas, 66160
Questcor Investigational Site
Lincoln, Nebraska, 68506
Questcor Investigational Site
Memphis, Tennessee, 38104
Questcor Investigational Site
Miami, Florida, 33136
Questcor Investigational Site
Phoenix, Arizona, 85018
Questcor Investigational Site
Pittsburgh, Pennsylvania, 15212
Questcor Investigational Site
Rochester, Minnesota, 55905
Questcor Investigational Site
San Antonio, Texas, 78229
Questcor Investigational Site
San Francisco, California, 94115
Questcor Investigational Site
Stanford, California, 94305
Questcor Investigational Site
Tampa, Florida, 33612
About this study
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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Repository corticotropin injection
- Keywords
- H.P. Acthar Gel, Acthar, amyotrophic lateral sclerosis, ALS
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Repository corticotropin injectionDrug
Also called H.P. Acthar Gel, Acthar, ACTH Gel, ACTH
Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
| Study group | Role | What they receive |
|---|---|---|
| Acthar 80 U (1.0 mL) SC twice weekly | Experimental | Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly Receives: Drug: Repository corticotropin injection |
| Acthar 24 U (0.3 mL) SC daily | Experimental | Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily Receives: Drug: Repository corticotropin injection |
| Acthar 56 U (0.7 mL) SC twice weekly | Experimental | Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly Receives: Drug: Repository corticotropin injection |
| Acthar 16 U (0.2 mL) SC daily | Experimental | Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily Receives: Drug: Repository corticotropin injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication
Measured: Baseline to Week 8
Secondary outcomes (3)
Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation
Measured: Baseline to Week 8
Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication
Measured: Baseline to Week 8
Proportion of Subjects With Treatment Emergent Suicidality
Measured: Baseline to Week 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2013 (estimated)
- Study startJul 2013
- Primary completionNov 2014
- Study completionDec 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mallinckrodt
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01906658
- Sponsor's protocol ID
- QSC01-ALS-01
- Status last verified
- Nov 2016
- First submitted
- 15 Jul 2013
- Last updated
- 6 Jan 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT01906658
- Acronym / sponsor ID
- QSC01-ALS-01
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- H.P. Acthar Gel, Acthar, amyotrophic lateral sclerosis, ALS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- MallinckrodtIndustry (pharma, biotech, devices)
- Study start
- Jul 2013
- Primary completion
- Nov 2014 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 24 Jul 2013 (estimated)
- Results
- Posted 6 Jan 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Acthar 80 U (1.0 mL) SC twice weekly | Experimental | Drug: Repository corticotropin injectionActhar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly |
| Acthar 24 U (0.3 mL) SC daily | Experimental | Drug: Repository corticotropin injectionActhar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily |
| Acthar 56 U (0.7 mL) SC twice weekly | Experimental | Drug: Repository corticotropin injectionActhar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly |
| Acthar 16 U (0.2 mL) SC daily | Experimental | Drug: Repository corticotropin injectionActhar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (15 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Repository corticotropin injectionAlso: H.P. Acthar Gel, Acthar, ACTH Gel, ACTHActhar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks | Drug | Acthar 16 U (0.2 mL) SC daily, Acthar 24 U (0.3 mL) SC daily, Acthar 56 U (0.7 mL) SC twice weekly, Acthar 80 U (1.0 mL) SC twice weekly |
Primary outcome
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
3 secondary outcomes
Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation
Time frame: Baseline to Week 8
Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication
Time frame: Baseline to Week 8
Proportion of Subjects With Treatment Emergent Suicidality
Time frame: Baseline to Week 36
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jan 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Repository corticotropin injection in people with Amyotrophic Lateral Sclerosis, run by Mallinckrodt at 17 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Jan 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jan 2017.