- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT00683189
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Objectives/Purpose:
To determine the safety and efficacy of a Vitamin K (Vit K) antagonist (warfarin) in treating Metachromatic Leukodystrophy (MLD).
Studieoversigt
Status
Betingelser
Intervention / Behandling
Detaljeret beskrivelse
Hypothesis:
Vit K has an essential role in biosynthesis of sulfatides and other sphingolopids in the brain. Administering warfarin, a Vit K antagonist, may ameliorate the phenotype in MLD by decreasing t he amount of sphingolipid storage in the neuronal cells.
Study Design Prospective: we will enroll eligible consenting subjects into the study. The study will not include a control group and the families and treating physicians are informed administration of the drug.
- Duration of Treatment: 4 weeks
- Pharmacological Intervention: The patients will receive warfarin 1.5 mg at the beginning of the study period. The dosage then will be adjusted to the INR values on weekly basis.
- Clinical evaluation: The patients will undergo clinical assessment prior to starting the treatment and at the end of the treatment period. The clinical assessment will also include administration of Gross Motor Function Measure (GMFM), a clinical toll for evaluation of motor development in children.
- Urine Sulfatide Quantification: Urine samples for quantification of the sulfatide level will be collected at the time of enrollment, after 2 weeks and at the end of treatment period.
- Blood Monitoring: The patients will undergo blood test for PT/INR at baseline and afterwards, at weekly bases for 4 weeks. The INR will be kept in a safe range of 2-2.5. If the INR is greater than 4.0 the dosage of warfarin will be lowered and another blood draw will be performed in 3 days.
Undersøgelsestype
Tilmelding (Forventet)
Fase
- Ikke anvendelig
Kontakter og lokationer
Studiesteder
-
-
New Jersey
-
Camden, New Jersey, Forenede Stater, 08103
- Cooper University Hospital
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria:
- Children with MLD, 1 to 10 years of age who have received and failed bone marrow transplantation or are excluded from the treatment due to delayed diagnosis or any other reasons.
Exclusion Criteria:
- Any Children with MLD who are eligible for and might receive ABMT.
- Any Children with MLD who suffer with a bleeding disorder, moderate to severe anemia or any other hematological disorders.
- Any contraindications systemic for anti-coagulation
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Tidsramme |
---|---|
Quantitative Neurological Assessment
Tidsramme: 4 weeks
|
4 weeks
|
Urine Sulfatides Quantification
Tidsramme: 4 weeks
|
4 weeks
|
Sekundære resultatmål
Resultatmål |
Tidsramme |
---|---|
Brain MRI
Tidsramme: before and after treatment
|
before and after treatment
|
Samarbejdspartnere og efterforskere
Sponsor
Efterforskere
- Studieleder: Paola Leone, Ph.D., UMDNJ/SOM
- Ledende efterforsker: Mitra Assadi, M.D., The Cooper Health System
Datoer for undersøgelser
Studer store datoer
Studiestart
Primær færdiggørelse (Faktiske)
Studieafslutning (Faktiske)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Skøn)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Skøn)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
- Metaboliske sygdomme
- Hjernesygdomme
- Sygdomme i centralnervesystemet
- Sygdomme i nervesystemet
- Demyeliniserende sygdomme
- Genetiske sygdomme, medfødte
- Metabolisme, medfødte fejl
- Lysosomale opbevaringssygdomme
- Lipidmetabolismeforstyrrelser
- Hjernesygdomme, metaboliske
- Hjernesygdomme, metaboliske, medfødte
- Sphingolipidoser
- Lysosomale opbevaringssygdomme, nervesystemet
- Lipidoser
- Lipidmetabolisme, medfødte fejl
- Leukoencefalopati
- Arvelige demyeliniserende sygdomme i centralnervesystemet
- Sulfatidose
- Leukodystrofi, Metakromatisk
- Antikoagulanter
- Warfarin
Andre undersøgelses-id-numre
- RP#07/063
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