- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT01900093
Evaluation of the Response to Acthar Gel Therapy in Patients Who Failed Intravenous Methylprednisolone (IVMP) for MS Relapses
1. september 2017 oppdatert av: Aaron Miller
An Open-Label Prospective Proof-Of-Concept Trial to Evaluate the Response to Acthar Gel Therapy in Patients With Multiple Sclerosis (MS) Relapses Who Have Failed To Make an Adequate Recovery After Treatment With High-Dose Intravenous Methylprednisolone
Although ACTHAR gel is approved for the treatment of acute relapses, it is currently not widely used.
Thus, we propose to conduct a small open-label proof-of-concept trial to evaluate the response to ACTHAR gel therapy in patients who have failed to make a satisfactory recovery after treatment with high dose IVMP.
Documentation of the clinical course as well as any adverse events related to IVMP use will be made.
The investigators propose to study the potential benefit of a 14-day course (the dose historically used since the landmark clinical trial of Rose et al1) of subcutaneous ACTHAR gel in 10 patients who have demonstrated inadequate improvement after a course of IVMP, 1000 mg daily, for 5 treatments (over a maximum of 8 days).
These would be patients for whom PLEX would be considered as a treatment possibility.
The primary outcome measure will be improvement in the targeted neurological deficit, as measured on the appropriate functional system score (FSS) of the EDSS.
Studieoversikt
Detaljert beskrivelse
This is an open-label, small, proof-of-concept study examining the safety, tolerability, and extent of recovery of a two-week course of subcutaneous Acthar Gel therapy in patients with MS relapse who have failed to make a satisfactory recovery after treatment with high dose methylprednisolone.
Eligible patients will be given 80 units of Acthar for 14 days.
Patients will be evaluated at baseline, at 1 week of Acthar treatment, at completion of Acthar treatment, and 1 week after completion of treatment.
For those who do not undergo plasmapheresis an additional evaluation will be conducted 2 weeks after completion of treatment.
Monitoring will include blood pressure determination and blood sugar determination.
Studietype
Intervensjonell
Registrering (Forventet)
10
Fase
- Ikke aktuelt
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiesteder
-
-
New York
-
New York, New York, Forente stater, 10029
- Rekruttering
- Icahn School of Medicine at Mount Sinai
-
Hovedetterforsker:
- Aaron Miller, MD
-
-
Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
18 år til 65 år (Voksen, Eldre voksen)
Tar imot friske frivillige
Nei
Kjønn som er kvalifisert for studier
Alle
Beskrivelse
Inclusion Criteria:
- Ages 18-65
- EDSS of 2.0 - 7.5 (inclusive)
- Sustained a significant MS exacerbation affecting vision, brainstem, motor, or cerebellar function AND were initially treated with a 5 day course of IVMP within 10 days of the onset of symptoms.
- Failed to make adequate improvement and must initiate Acthar Gel therapy within two weeks (+/- 72 hours) after the first day of 5 treatments with 1000 mg IVMP, as judged by their treating neurologist.
- Must be able to comply with the requirements of the protocol as determined by the investigator.
- Ability to understand the purpose and risks of the study and provide signed and date informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
Exclusion Criteria:
- Patients whose relapse consists of pure sensory or bowel/bladder symptoms
- History of ACTHAR gel use or other forms of ACTH with failure to improve or with occurrence of significant adverse experiences.
- Diagnosis of scleroderma, systemic fungal infections, ocular herpes simplex, congestive heart failure, and/or uncontrolled hypertension, and/or any clinically relevant medical disease that would put the patient at risk by participating in the study
- Persistent significant or severe infection
- Recent history of drug or alcohol abuse
- Concomitant use or prior use in the preceding 6 months of any investigational drug.
- Pregnant or nursing
- Recent surgery (up to the investigator's discretion what constitutes recent)
- History of, or the presence of, a peptic ulcer
- Known sensitivity to proteins of porcine origin
- Received a live or live attenuated vaccine in the last 30 days before baseline
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
---|---|
Eksperimentell: Acthar Gel
80 units of subcutaneous Acthar Gel therapy daily
|
80 units of subcutaneous Acthar Gel therapy daily
Andre navn:
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
---|---|---|
Improvement in Functional System Score appropriate to the Targeted Neurological Deficit
Tidsramme: at one week
|
Primary outcome measure will be the percentage of patients improving by at least 1 step on the FSS appropriate to their neurological deficit (targeted neurological deficit [TND]} at 1 week following completion of treatment
|
at one week
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
---|---|---|
Improvement in Expanded Disability Status Scale
Tidsramme: baseline and at 1 week
|
Secondary outcome measures will be improvement in TND at completion of ACTHAR Gel therapy, improvement by at least 1 step on EDSS at completion of treatment and at 1 week following completion of treatment.
|
baseline and at 1 week
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Samarbeidspartnere
Etterforskere
- Hovedetterforsker: Aaron Miller, MD, Icahn School of Medicine at Mount Sinai
- Hovedetterforsker: Fred Lublin, MD, Icahn School of Medicine at Mount Sinai
- Hovedetterforsker: Stephen Krieger, MD, Icahn School of Medicine at Mount Sinai
- Hovedetterforsker: Michelle Fabian, MD, Icahn School of Medicine at Mount Sinai
- Hovedetterforsker: Ilana Katz-Sand, MD, Icahn School of Medicine at Mount Sinai
- Hovedetterforsker: Sylvia Klineova, MD, Icahn School of Medicine at Mount Sinai
- Hovedetterforsker: Gretchen Mathewson, NP, Icahn School of Medicine at Mount Sinai
- Hovedetterforsker: Aliza Ben-Zacharia, NP, Icahn School of Medicine at Mount Sinai
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart
1. juli 2013
Primær fullføring (Forventet)
1. mars 2018
Studiet fullført (Forventet)
1. mars 2018
Datoer for studieregistrering
Først innsendt
11. juli 2013
Først innsendt som oppfylte QC-kriteriene
15. juli 2013
Først lagt ut (Anslag)
16. juli 2013
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
6. september 2017
Siste oppdatering sendt inn som oppfylte QC-kriteriene
1. september 2017
Sist bekreftet
1. september 2017
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
- Patologiske prosesser
- Sykdommer i nervesystemet
- Sykdommer i immunsystemet
- Demyeliniserende autoimmune sykdommer, CNS
- Autoimmune sykdommer i nervesystemet
- Demyeliniserende sykdommer
- Autoimmune sykdommer
- Multippel sklerose
- Sklerose
- Fysiologiske effekter av legemidler
- Hormoner
- Hormoner, hormonsubstitutter og hormonantagonister
- Adrenokortikotropisk hormon
Andre studie-ID-numre
- GCO 13-0600
Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .
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