Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

Part of paid clinical trials in Orange, California.

Sponsor
Abliva AB
Study ID
NCT05650229
Phase
PHASE2
Status
Recruiting
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Conditions

  • Primary Mitochondrial Disease

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • KL1333 — DRUG
    Twice daily
  • Placebo — DRUG
    Twice daily

Study Details

The primary objective of the FALCON study is to evaluate the efficacy of KL1333 on selected disease manifestations of primary mitochondrial disease (PMD) following 48 weeks of treatment. This objective involves evaluating the efficacy of KL1333 versus placebo on fatigue symptoms and impacts on daily living as well as on functional lower extremity strength and endurance. Additionally, the study evaluates the safety and tolerability of KL1333.

Key Dates

Start date
Dec 13, 2022
Status verified
Apr 2026
Primary completion
Oct 31, 2027
Completion
Nov 30, 2027

Study Design

Enrollment
180 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: KL1333
    Twice daily
  • Placebo Comparator: Matching Placebo
    Twice daily

Primary Outcome Measure

Change in-patient-reported fatigue symptoms and impacts on daily living measured by Patient-Reported Outcomes Measurement Information System (PROMIS®) Fatigue PMD Short Form [ Time Frame: Baseline and 48 Weeks ]

Central Contacts

Locations (20)

FacilityCityStateZIPSite coordinators
University of California, Irvine - ALS & Neuromuscular CenterOrangeCalifornia92868
Mozaffar Tahseen, MD Professor
[email protected]
714-456-2332
The Regents of the University of California - San DiegoSan DiegoCalifornia292093
Richard Haas, MD
[email protected]
858-246-2288
UCSF Movement Disorders ClinicSan FranciscoCalifornia94158
Swathy Chandrashekhar (PRINCIPAL_INVESTIGATOR)
Children's Hospital Colorado - Center for Cancer and Blood Disorders (CCBD) - Anschutz Medical Campus LocationAuroraColorado80045
Austin Larson, MD
[email protected]
303-724-2338
Yale University School of MedicineNew HavenConnecticut06519
Bhaskar Roy (PRINCIPAL_INVESTIGATOR)
Novel Clinical Research Center, LLCMiamiFlorida33186
Juan Remos (PRINCIPAL_INVESTIGATOR)
Rare Disease Research, LLCAtlantaGeorgia303129
Scott Batchelor, MD
[email protected]
678-883-6897
IU Health University HospitalIndianapolisIndiana46202
Melissa Lah (PRINCIPAL_INVESTIGATOR)
Johns Hopkins University School of MedicineBaltimoreMaryland21205
Hilary Vernon (PRINCIPAL_INVESTIGATOR)
Mayo ClinicRochesterMinnesota55905
Tan Khoon Ghee Queenie, MD
[email protected]
507-284-2120
Washington University School of Medicine - Center for Advanced Medicine (CAM) - Neuroscience CenterSt LouisMissouri63110
Conrad Weihl, MD
[email protected]
314-747-6394
Tekton Marlboro NeurologyMarlboroNew Jersey07746
Amor Mehta (PRINCIPAL_INVESTIGATOR)
Columbia University Irving Medical CenterNew YorkNew York100032
Hirano Michio, MD
[email protected]
212-305-1048
Icahn School of Medicine at Mount SinaiNew YorkNew York10029-6574
Ibrahim Elsharkawi (PRINCIPAL_INVESTIGATOR)
Akron Children's HospitalAkronOhio44307
Josselyn Coppenger
[email protected]
330-543-6164
Bruce Cohen, MD (PRINCIPAL_INVESTIGATOR)
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Zarazuela Zolkipli-Cunningham (PRINCIPAL_INVESTIGATOR)
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15224
Uta Lichter-Konecki, MD
[email protected]
412-692-8865
Jennifer Baker
[email protected]
412-692-6378
Neurology and Neuromuscular Care Center-Neurology Rare Disease CenterFlower MoundTexas75028
Diana Castro (PRINCIPAL_INVESTIGATOR)
Baylor College of Medicine (BCM)HoustonTexas77030
Fernando Scaglia, MD
[email protected]
832-822-4280
The University of Texas Health Science Center at HoustonHoustonTexas77030
William Guerra
[email protected]
Mary Kay Koenig, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Orange, CA

By research site
University of California, Irvine - ALS & Neuromuscular Center· Orange, CAThe Regents of the University of California - San Diego· San Diego, CAUCSF Movement Disorders Clinic· San Francisco, CAChildren's Hospital Colorado - Center for Cancer and Blood Disorders (CCBD) - Anschutz Medical Campus Location· Aurora, COYale University School of Medicine· New Haven, CTNovel Clinical Research Center, LLC· Miami, FL

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