Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9
30. juni 2026 oppdatert av: ML Bio Solutions, Inc.
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of BBP-418 (Ribitol) in Participants With Limb-Girdle Muscular Dystrophy 2I/R9 (LGMD2I/R9)
This is an open-label extension (rollover) study designed to evaluate the long-term safety and efficacy of BBP-418 (ribitol) in participants with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) who have previously participated in Study MLB-01-005 (Fortify).
Participants will receive BBP-418 administered orally at protocol-defined doses and schedules. The study will assess long-term safety through monitoring of adverse events, clinical laboratory evaluations, and other safety assessments. Efficacy will be evaluated using functional measures and other clinical endpoints relevant to LGMD2I/R9.
Participants will be followed for up to 36 months, with a final safety follow-up assessment conducted approximately 30 days after the last dose of study drug.
Studieoversikt
Status
Aktiv, ikke rekrutterende
Intervensjon / Behandling
Studietype
Intervensjonell
Registrering (Antatt)
107
Fase
- Fase 3
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiesteder
-
-
-
Brisbane, Australia
- Royal Brisbane and Women's Hospital
-
-
-
-
-
Copenhagen, Danmark
- Rigshospitalet, Neuromuscular Clinic and Research Unit
-
-
-
-
Arkansas
-
Little Rock, Arkansas, Forente stater, 72202
- Arkansas Children's Hospital
-
-
California
-
Orange, California, Forente stater, 92868
- University of California Irvine Medical Center
-
-
Colorado
-
Aurora, Colorado, Forente stater, 80045
- University of Colorado Hospital (UCH) - Anschutz Medical Campus
-
-
Florida
-
Gainesville, Florida, Forente stater, 32608
- University of Florida Health Center for Pediatric Neuromuscular and Rare Diseases
-
-
Iowa
-
Iowa City, Iowa, Forente stater, 52242
- University of Iowa Stead Family Children's Hospital
-
-
Kansas
-
Kansas City, Kansas, Forente stater, 66160
- University of Kansas Medical Center (KUMC)
-
-
Maryland
-
Baltimore, Maryland, Forente stater, 21205
- Kennedy Krieger Institute
-
-
Minnesota
-
Minneapolis, Minnesota, Forente stater, 55455
- University of Minnesota Medical School
-
-
Missouri
-
St Louis, Missouri, Forente stater, 63110
- Washington University School of Medicine
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, Forente stater, 19104
- University of Pennsylvania
-
Philadelphia, Pennsylvania, Forente stater, 19104
- The Children's Hospital of Philadelphia
-
-
Virginia
-
Norfolk, Virginia, Forente stater, 23510
- Children's Hospital of the Kings Daughters
-
-
-
-
-
Milan, Italia
- Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
-
-
-
-
-
Leiden, Nederland
- Leids Universitair Medisch Centrum
-
-
-
-
-
Tromsø, Norge
- Universitetssykehuset Nord-Norge, Department of Neurology
-
-
-
-
-
London, Storbritannia
- UCL Great Ormond Street Hospital
-
Newcastle upon Tyne, Storbritannia
- Newcastle University - John Walton Muscular Dystrophy Research Centre
-
-
-
-
-
Berlin, Tyskland
- Charité Universitätsmedizin Berlin and Max Delbrück Center
-
-
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
- Barn
- Voksen
- Eldre voksen
Tar imot friske frivillige
Nei
Beskrivelse
Inclusion Criteria:
- Completed Study MLB-01-005 on study drug through the final clinic visit (Month 36 or another qualifying end-of-study visit as determined by the Sponsor).
- The participant (or parent/guardian) who signs the ICF understands the study procedures and agrees to participate in the study by giving informed consent (or assent, if <18 years of age).
- Is willing and able to complete all study procedures according to the Schedule of Assessments.
- A WOCBP or a nonsterile male participant must be willing to use an acceptable method of contraception from the time of consent through 30 days after the last dose of study drug in this study.
Exclusion Criteria:
Has developed clinically significant concomitant disease that would, in the Investigator's opinion, be likely to unfavorably impact study participation, including:
- Any significant concomitant medical condition, including psychiatric, cardiac, renal, pulmonary, hepatic, or endocrine disease other than that associated with LGMD2I/R9
- Any other significant laboratory, vital sign, ECG abnormality, clinical history, or finding
- Is pregnant (based on the Baseline / Day 1 pregnancy test result) and/or breastfeeding or planning to conceive children within the projected duration of the study through 30 days after the last dose of study drug in this study.
- Has active suicidal ideation, defined as having a suicide ideation score of 4 (Active Suicidal Ideation with Some Intent to Act, without Specific Plan) or 5 (Active Suicidal Ideation with Specific Plan and Intent) on the C-SSRS at Baseline / Day 1.
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: BBP-418
Drug: BBP-418. Single arm.
|
Drug: BBP-418.
Single arm.
Participants who completed Study MLB-01-005 and meet eligibility criteria will receive BBP-418 (ribitol) taken orally twice daily at 9 or 12g (based on body weight measured) for up to 36 months.
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
Frequency and severity of treatment-emergent adverse events to assess long-term safety of BBP-418.
Tidsramme: 36 months
|
36 months
|
|
Change from baseline in North Star Assessment for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Tidsramme: 36 months
|
36 months
|
Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
Change from baseline in 10MWT (10-meter walk test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Tidsramme: 36 months
|
36 months
|
|
Change from baseline FVC (forced vital capacity) (percent predicted, performed in a sitting position) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Tidsramme: 36 months
|
36 months
|
|
Change from baseline in PUL 2.0 (performance of the upper limb) total score for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Tidsramme: 36 months
|
36 months
|
|
Change from baseline in 100MTT (100-meter timed test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Tidsramme: 36 months
|
36 months
|
|
Change from baseline in Serum CK (creatine kinase) for LGMD2I/R9 to assess long-term biomarker changes in participants.
Tidsramme: 36 months
|
36 months
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
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 (Faktiske)
5. juni 2026
Primær fullføring (Antatt)
1. oktober 2030
Studiet fullført (Antatt)
1. oktober 2030
Datoer for studieregistrering
Først innsendt
25. juni 2026
Først innsendt som oppfylte QC-kriteriene
25. juni 2026
Først lagt ut (Faktiske)
1. juli 2026
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
2. juli 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
30. juni 2026
Sist bekreftet
1. juni 2026
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- MLB-01-007
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
NEI
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Ja
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
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. .