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Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease (FALCON)

Primary Purpose

Primary Mitochondrial Disease

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
KL1333
Placebo
Sponsored by
Abliva AB
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Mitochondrial Disease focused on measuring Chronic fatigue, Myopathy, Muscle weakness, Exercise intolerance, Diabetes

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Age 18 years or older. A confirmed PMD diagnosis caused by a known pathogenic gene mutation or deletion of the mitochondrial genome (category 6 of the International Classification of Inborn Metabolic Disorders [ICIMD])12 according to American College of Medical Genetics (ACMG)/Association of Molecular Pathology (AMP) criteria1, with multisystemic disease expressions, including: m.3243A>G associated MELAS-MIDD spectrum disorders, single large scale mtDNA deletion associated KSS-CPEO spectrum disorders, other multisystemic mtDNA-related disease (including MERRF). Presence of chronic mitochondrial fatigue: History of mitochondrial fatigue for at least 3 months prior to the Screening Visit AND Presence of at least moderate level of fatigue, assessed by PROMIS® Fatigue PMD Short form raw score ≥ 27 at Screening and Baseline Presence of mitochondrial myopathy defined as: Myopathy (proximal muscle weakness), NMDAS Section III Clinical Assessment, item 5 score ≥ 1, which reads: "mild but clear proximal weakness in hip flexion and shoulder abduction - MRC 4/5". For the inclusion only hip flexion, but not shoulder abduction, should be taken into account. AND / OR Exercise Tolerance: NMDAS Section I, item 9 score ≥ 1, which reads: "unlimited on flat - symptomatic on inclines or stairs". Patients must be able to perform at least 2 repetitions and the maximal capacity must not exceed 17 repetitions in males or 16 repetitions in females in a 30s STS test at screening. Clinically stable, apart from symptoms associated with the diagnosis of mitochondrial disease, at Screening and Baseline, as determined by medical history, physical examination, 12-lead ECG, vital signs measurements, and clinical laboratory evaluations at Screening, as assessed by the investigator. The patient is willing and able to attend study appointments within the specified time windows. Willingness and ability to complete electronic PROs. Willingness to maintain a stable diet during the Screening and study periods. Patients who take any mitochondrial disease-focused vitamins or supplemental therapies, including coenzyme Q10 (CoQ10), has been on a stable dose regimen of these for 3 months prior to randomisation and intends to stay on a stable dose for the duration of the study period. Willingness to suspend treatment with idebenone during the study. Female patient is not pregnant and at least one of the following conditions apply: Not a woman of childbearing potential (WOCBP) WOCBP must agree not to try and become pregnant and use a highly effective method of contraception from the time of informed consent through at least 36 days (~5 half-lives of KL1333 plus 30 days) after the last dose of investigational medicinal product (IMP) administration. Male patients with female partner(s) of childbearing potential must agree to use a male condom in addition to using highly effective contraception throughout the treatment period and for 96 days after the last dose of IMP administration. The requirement to use a male condom also applies to male patients with a pregnant or breastfeeding partner. Female patients must agree not to breastfeed starting at Screening and throughout the study period and for 36 days after the last dose of IMP administration. Female patients must agree to not donate ova throughout the study period and for 36 days after the last dose of IMP administration, and male patients must agree to not donate sperm throughout the study period and for 96 days after the last dose of IMP administration. Exclusion Criteria: Primary mitochondrial disease with predominant neurodegenerative phenotypes, such as, but not limited to, Leigh syndrome, Leber hereditary optic neuropathy (LHON) and Neuropathy ataxia-retinitis pigmentosa syndrome (NARP). Primary mitochondrial disease nuclear DNA mutations or mutations causing mtDNA destabilisation. Genetic mtDNA variants of uncertain significance, likely pathogenic, or pathogenic mutations with degrees of heteroplasmy below what can be considered to definitely cause PMD. General fatigue or muscle weakness due to causes other than mitochondrial disease, in the opinion of the investigator. Significant cardiovascular disease (e.g., sustained or symptomatic arrhythmia; dilated heart chambers or reduced function; Mobitz II atrioventricular block or greater) OR abnormal ECG that is clinically significant, as determined by the investigator. Any QTcF > 450 msec for male patients and > 470 msec for female patients is exclusionary. In the case of an exclusionary QTcF, the ECG can be repeated twice and the average of 3 QTcF intervals should be used to determine the QTcF eligibility. Recent history of unstable disease, inadequately controlled neurological manifestations or not recovered from stroke-like episodes including but not limited to: stroke-like episodes within the last 6 months more than 1 seizure/month within the last 6 months hospitalised for Status Epilepticus within the last 6 months more than 4 days of migraine episodes/month within the last 6 months History of inflammatory bowel disease, gastric erosions, peptic ulcer disease, or gastrointestinal bleeding episodes. Gastroesophageal reflux disease diagnosed by objective endoscopic or radiographic means, and clinically symptomatic at any point over the last 6 months. The patient has one or more clinical laboratory test values outside the reference range, based on the blood and urine samples taken at the Screening Visit, that are of potential risk to the patient's safety, or the patient has, at the Screening Visit: estimated glomerular filtration rate (eGFR) calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation <60 mL/min/1.73 m2 a serum total bilirubin value > 1.5 times the upper limit of the reference range a serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) value > 2 times the upper limit of the reference range The patient has, in the investigator's opinion, severe ataxia, neuropathy, balance problems or other medical condition that would interfere the evaluation of the 30s STS test. Untreated or undertreated sleep apnoea, in the opinion of the investigator. Use of idebenone within 14 days prior to the first dose. Patients have a history of unstable or severe pulmonary, immunological, oncological, hepatic disease, renal disease, or another medically significant illness other than PMD or takes medication that could, in the investigator's opinion, interfere with the assessments of safety, tolerability, or efficacy, or interfere with the conduct or interpretation of the study. The patient is, in the investigator's opinion, unlikely to comply with the protocol e.g. due to cognitive impairment or is unsuitable for any reason. The patient has an immediate family member (defined as family members residing at the same address) who participates in the study. Female patients with a positive pregnancy result at Screening or at Baseline. A patient cannot participate if they received an investigational drug 30 days or 5 half-lives prior to the Screening Visit (whichever is longer), or plans to use an investigational drug (other than the study intervention) during the study Hypersensitivity to the active substance or to any of the excipients or placebo.

Sites / Locations

  • Akron Children's HospitalRecruiting
  • The University of Texas Health Science Center at HoustonRecruiting
  • Hopital Universitaire de Bruxelles (H.U.B)/ Academisch Ziekenhuis BrusselRecruiting
  • Universitair Ziekenhuis GentRecruiting
  • Universitair Ziekenhuis Leuven Gasthuisberg CampusRecruiting
  • Copenhagen Neuromuscular Center, RigshospitaletRecruiting
  • Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier PellegrinRecruiting
  • Hopital Roger Salengro, CHRU de LilleRecruiting
  • Centre Hospitalier Universitaire de Nice, Hopital Pasteur 2Recruiting
  • CHU de NICE - Hôpital Archet 2Recruiting
  • Groupe Hospitalier Pitie-SalpetriereRecruiting
  • CIBERER- IDIBAPS, Faculty of Medicine, University of BarcelonaRecruiting
  • Hospital de la Santa Creu i Sant PauRecruiting
  • Hospital General Universitario de CatalunyaRecruiting
  • Hospital Universitario 12 de OctubreRecruiting
  • Department of Clinical Neurosciences, Addenbrooke's HospitalRecruiting
  • University College London Hospitals Nhs Foundation TrustRecruiting
  • Royal Victoria Infirmary - The Newcastle Upon Tyne Hospitals NHS Foundation TrustRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

KL1333

Matching Placebo

Arm Description

Twice daily

Twice daily

Outcomes

Primary Outcome Measures

Change in-patient-reported fatigue symptoms and impacts on daily living measured by Patient-Reported Outcomes Measurement Information System (PROMIS®) Fatigue PMD Short Form
Change in t-score. Higher scores indicate greater fatigue severity.
Change in 30 Second Site-to-Stand Test.
Change in number of stands

Secondary Outcome Measures

Change in Quality of Life in Neurological Disorders (Neuro-QoL) Lower Extremity Function (Mobility) Short Form
Change in t-score. Higher scores indicate greater functional ability.
Change in Individual Activity Assessments - Interference
Assessing how much Primary Mitochondrial Disease symptoms get in the way of the ability to perform individually selected activities. A 5 point scale ranging from "No interference" to "Completely interferes"
Individual Activity Assessment - Change
Assessing the ability to perform individually selected activities compared to baseline. A 5 point scale ranging from "Much worse" to "Much better"
Change in Patient Global Impression of Severity
Scale ranging from 1 (none) to 4 (severe).
Score on Patient Global Impression of Change
Scale ranging from 2 (much better) to -2 (much worse).
Change in Clinician Global Impression of Severity
Scale ranging from 1 (none) to 4 (severe).
Score on Clinician Global Impression of Change
Scale ranging from 2 (much better) to -2 (much worse).
Change in Newcastle Mitochondrial Disease Adult Scale (NMDAS) - subscales I-III
Measured on a 6-point rating scale from 0 to 5. Total score across all 3 subscales ranges from 0 to 140. Higher scores indicate more extensive and severe system involvements.
Change from baseline in Glycated haemoglobin (HbA1c) for subjects with diabetes
mmol/mol

Full Information

First Posted
December 6, 2022
Last Updated
September 12, 2023
Sponsor
Abliva AB
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1. Study Identification

Unique Protocol Identification Number
NCT05650229
Brief Title
Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease
Acronym
FALCON
Official Title
An Interventional, Randomised, Double-blind, Parallel-group, Placebo-controlled, Flexible-dose, Adaptive Study of the Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
December 13, 2022 (Actual)
Primary Completion Date
October 2025 (Anticipated)
Study Completion Date
December 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Abliva AB

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.
Detailed Description
The FALCON study is investigating whether the study medicine, KL1333, improves fatigue levels and physical abilities of people living with mitochondrial disease. The investigators are also evaluating the tolerability of the study medicine. For this study, the effects of KL1333 are compared with those from a placebo (a pill that looks like the study medicine but contains no active medicine). The study medicine (or placebo) is a tablet that is taken twice daily during the treatment period of 48 weeks. Participation in the FALCON study is divided into 3 parts: Screening and baseline: 8-12 weeks Treatment: 48 weeks Safety follow-up: 5 weeks Total duration: 61 - 65 weeks Patients who complete the screening phase and are enrolled in the study are randomly assigned to receive either the study medicine (KL1333) or placebo (no active medication). Patients are more likely to receive the study medication than placebo (for every five people who take part, three receive KL1333 and two receive placebo). Neither the participants nor the study team know who is receiving the study medicine or placebo and participants are not able to change which treatment they are assigned.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Mitochondrial Disease
Keywords
Chronic fatigue, Myopathy, Muscle weakness, Exercise intolerance, Diabetes

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
180 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
KL1333
Arm Type
Experimental
Arm Description
Twice daily
Arm Title
Matching Placebo
Arm Type
Placebo Comparator
Arm Description
Twice daily
Intervention Type
Drug
Intervention Name(s)
KL1333
Intervention Description
Twice daily
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Twice daily
Primary Outcome Measure Information:
Title
Change in-patient-reported fatigue symptoms and impacts on daily living measured by Patient-Reported Outcomes Measurement Information System (PROMIS®) Fatigue PMD Short Form
Description
Change in t-score. Higher scores indicate greater fatigue severity.
Time Frame
Baseline and 48 Weeks
Title
Change in 30 Second Site-to-Stand Test.
Description
Change in number of stands
Time Frame
Baseline and 48 Weeks
Secondary Outcome Measure Information:
Title
Change in Quality of Life in Neurological Disorders (Neuro-QoL) Lower Extremity Function (Mobility) Short Form
Description
Change in t-score. Higher scores indicate greater functional ability.
Time Frame
Baseline and 48 weeks
Title
Change in Individual Activity Assessments - Interference
Description
Assessing how much Primary Mitochondrial Disease symptoms get in the way of the ability to perform individually selected activities. A 5 point scale ranging from "No interference" to "Completely interferes"
Time Frame
Baseline and 48 Weeks
Title
Individual Activity Assessment - Change
Description
Assessing the ability to perform individually selected activities compared to baseline. A 5 point scale ranging from "Much worse" to "Much better"
Time Frame
48 weeks
Title
Change in Patient Global Impression of Severity
Description
Scale ranging from 1 (none) to 4 (severe).
Time Frame
Baseline and 48 weeks
Title
Score on Patient Global Impression of Change
Description
Scale ranging from 2 (much better) to -2 (much worse).
Time Frame
48 weeks
Title
Change in Clinician Global Impression of Severity
Description
Scale ranging from 1 (none) to 4 (severe).
Time Frame
Baseline and 48 weeks
Title
Score on Clinician Global Impression of Change
Description
Scale ranging from 2 (much better) to -2 (much worse).
Time Frame
48 weeks
Title
Change in Newcastle Mitochondrial Disease Adult Scale (NMDAS) - subscales I-III
Description
Measured on a 6-point rating scale from 0 to 5. Total score across all 3 subscales ranges from 0 to 140. Higher scores indicate more extensive and severe system involvements.
Time Frame
Baseline and 48 weeks
Title
Change from baseline in Glycated haemoglobin (HbA1c) for subjects with diabetes
Description
mmol/mol
Time Frame
Baseline and 48 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age 18 years or older. A confirmed PMD diagnosis caused by a known pathogenic gene mutation or deletion of the mitochondrial genome (category 6 of the International Classification of Inborn Metabolic Disorders [ICIMD])12 according to American College of Medical Genetics (ACMG)/Association of Molecular Pathology (AMP) criteria1, with multisystemic disease expressions, including: m.3243A>G associated MELAS-MIDD spectrum disorders, single large scale mtDNA deletion associated KSS-CPEO spectrum disorders, other multisystemic mtDNA-related disease (including MERRF). Presence of chronic mitochondrial fatigue: History of mitochondrial fatigue for at least 3 months prior to the Screening Visit AND Presence of at least moderate level of fatigue, assessed by PROMIS® Fatigue PMD Short form raw score ≥ 27 at Screening and Baseline Presence of mitochondrial myopathy defined as: Myopathy (proximal muscle weakness), NMDAS Section III Clinical Assessment, item 5 score ≥ 1, which reads: "mild but clear proximal weakness in hip flexion and shoulder abduction - MRC 4/5". For the inclusion only hip flexion, but not shoulder abduction, should be taken into account. AND / OR Exercise Tolerance: NMDAS Section I, item 9 score ≥ 1, which reads: "unlimited on flat - symptomatic on inclines or stairs". Patients must be able to perform at least 2 repetitions and the maximal capacity must not exceed 17 repetitions in males or 16 repetitions in females in a 30s STS test at screening. Clinically stable, apart from symptoms associated with the diagnosis of mitochondrial disease, at Screening and Baseline, as determined by medical history, physical examination, 12-lead ECG, vital signs measurements, and clinical laboratory evaluations at Screening, as assessed by the investigator. The patient is willing and able to attend study appointments within the specified time windows. Willingness and ability to complete electronic PROs. Willingness to maintain a stable diet during the Screening and study periods. Patients who take any mitochondrial disease-focused vitamins or supplemental therapies, including coenzyme Q10 (CoQ10), has been on a stable dose regimen of these for 3 months prior to randomisation and intends to stay on a stable dose for the duration of the study period. Willingness to suspend treatment with idebenone during the study. Female patient is not pregnant and at least one of the following conditions apply: Not a woman of childbearing potential (WOCBP) WOCBP must agree not to try and become pregnant and use a highly effective method of contraception from the time of informed consent through at least 36 days (~5 half-lives of KL1333 plus 30 days) after the last dose of investigational medicinal product (IMP) administration. Male patients with female partner(s) of childbearing potential must agree to use a male condom in addition to using highly effective contraception throughout the treatment period and for 96 days after the last dose of IMP administration. The requirement to use a male condom also applies to male patients with a pregnant or breastfeeding partner. Female patients must agree not to breastfeed starting at Screening and throughout the study period and for 36 days after the last dose of IMP administration. Female patients must agree to not donate ova throughout the study period and for 36 days after the last dose of IMP administration, and male patients must agree to not donate sperm throughout the study period and for 96 days after the last dose of IMP administration. Exclusion Criteria: Primary mitochondrial disease with predominant neurodegenerative phenotypes, such as, but not limited to, Leigh syndrome, Leber hereditary optic neuropathy (LHON) and Neuropathy ataxia-retinitis pigmentosa syndrome (NARP). Primary mitochondrial disease nuclear DNA mutations or mutations causing mtDNA destabilisation. Genetic mtDNA variants of uncertain significance, likely pathogenic, or pathogenic mutations with degrees of heteroplasmy below what can be considered to definitely cause PMD. General fatigue or muscle weakness due to causes other than mitochondrial disease, in the opinion of the investigator. Significant cardiovascular disease (e.g., sustained or symptomatic arrhythmia; dilated heart chambers or reduced function; Mobitz II atrioventricular block or greater) OR abnormal ECG that is clinically significant, as determined by the investigator. Any QTcF > 450 msec for male patients and > 470 msec for female patients is exclusionary. In the case of an exclusionary QTcF, the ECG can be repeated twice and the average of 3 QTcF intervals should be used to determine the QTcF eligibility. Recent history of unstable disease, inadequately controlled neurological manifestations or not recovered from stroke-like episodes including but not limited to: stroke-like episodes within the last 6 months more than 1 seizure/month within the last 6 months hospitalised for Status Epilepticus within the last 6 months more than 4 days of migraine episodes/month within the last 6 months History of inflammatory bowel disease, gastric erosions, peptic ulcer disease, or gastrointestinal bleeding episodes. Gastroesophageal reflux disease diagnosed by objective endoscopic or radiographic means, and clinically symptomatic at any point over the last 6 months. The patient has one or more clinical laboratory test values outside the reference range, based on the blood and urine samples taken at the Screening Visit, that are of potential risk to the patient's safety, or the patient has, at the Screening Visit: estimated glomerular filtration rate (eGFR) calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation <60 mL/min/1.73 m2 a serum total bilirubin value > 1.5 times the upper limit of the reference range a serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) value > 2 times the upper limit of the reference range The patient has, in the investigator's opinion, severe ataxia, neuropathy, balance problems or other medical condition that would interfere the evaluation of the 30s STS test. Untreated or undertreated sleep apnoea, in the opinion of the investigator. Use of idebenone within 14 days prior to the first dose. Patients have a history of unstable or severe pulmonary, immunological, oncological, hepatic disease, renal disease, or another medically significant illness other than PMD or takes medication that could, in the investigator's opinion, interfere with the assessments of safety, tolerability, or efficacy, or interfere with the conduct or interpretation of the study. The patient is, in the investigator's opinion, unlikely to comply with the protocol e.g. due to cognitive impairment or is unsuitable for any reason. The patient has an immediate family member (defined as family members residing at the same address) who participates in the study. Female patients with a positive pregnancy result at Screening or at Baseline. A patient cannot participate if they received an investigational drug 30 days or 5 half-lives prior to the Screening Visit (whichever is longer), or plans to use an investigational drug (other than the study intervention) during the study Hypersensitivity to the active substance or to any of the excipients or placebo.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Communication Manager
Phone
+46462756220
Email
clinicaltrialinfo@abliva.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Amel Karaa, MD
Organizational Affiliation
Massachusetts General Hospital
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Grainne Gorman, MD, PhD
Organizational Affiliation
Wellcome Centre for Mitochondrial Research, Newcastle University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Akron Children's Hospital
City
Akron
State/Province
Ohio
ZIP/Postal Code
44307
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Josselyn Coppenger
Phone
330-543-6164
Email
jcoppenger@akronchildrens.com
First Name & Middle Initial & Last Name & Degree
Bruce Cohen, MD
Facility Name
The University of Texas Health Science Center at Houston
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
William Guerra
Email
William.h.Guerra@uth.tmc.edu
First Name & Middle Initial & Last Name & Degree
Mary Kay Koenig, MD
Facility Name
Hopital Universitaire de Bruxelles (H.U.B)/ Academisch Ziekenhuis Brussel
City
Brussel
Country
Belgium
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Gauthier Remiche, MD
Phone
+3225555676
Email
gauthier.remiche@erasme.ulb.ac.be
First Name & Middle Initial & Last Name & Degree
Christiane Kitoko
Email
Christiane.kitoko@hubruxelles.be
First Name & Middle Initial & Last Name & Degree
Gauthier Remiche, MD
Facility Name
Universitair Ziekenhuis Gent
City
Gent
Country
Belgium
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Fien Demeulemeester
Phone
+32 9 332 57 00
Email
fien.demeulemeester@uzgent.be
First Name & Middle Initial & Last Name & Degree
Dimitri Hemelsoet, MD
Email
dimitri.hemelsoet@uzgent.be
First Name & Middle Initial & Last Name & Degree
Dimitri Hemelsoet, MD
Facility Name
Universitair Ziekenhuis Leuven Gasthuisberg Campus
City
Leuven
Country
Belgium
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kristl Claeys, MD
Email
kristl.claeys@uzleuven.be
First Name & Middle Initial & Last Name & Degree
Kristl Claeys, MD
Facility Name
Copenhagen Neuromuscular Center, Rigshospitalet
City
Copenhagen
Country
Denmark
Individual Site Status
Recruiting
Facility Contact:
Email
nicolai.rasmus.preisler@regionh.dk
First Name & Middle Initial & Last Name & Degree
Nicolai Preisler, MD
Facility Name
Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin
City
Bordeaux
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Aurelien Trimouille, MD
Facility Name
Hopital Roger Salengro, CHRU de Lille
City
Lille
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Celine Tard
Phone
+33 3 20 44 59 62
Email
celine.tard@chu-lille.fr
First Name & Middle Initial & Last Name & Degree
Celine Tard, MD
Facility Name
Centre Hospitalier Universitaire de Nice, Hopital Pasteur 2
City
Nice
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sabrina Sacconi, MD
Phone
+33(0)492037267
Email
sacconi.s@chu-ncie.fr
First Name & Middle Initial & Last Name & Degree
Sabrina Sacconi, MD
Facility Name
CHU de NICE - Hôpital Archet 2
City
Nice
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Véronique Paquis-Flucklinger, MD
Facility Name
Groupe Hospitalier Pitie-Salpetriere
City
Paris
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anthony Behin
Phone
+33 142163713
Email
anthony.behin@aphp.fr
First Name & Middle Initial & Last Name & Degree
Anthony Behin, MD
Facility Name
CIBERER- IDIBAPS, Faculty of Medicine, University of Barcelona
City
Barcelona
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pablo Iglesias García
Phone
+34 932275400
Ext
4840-4622
Email
PIGLESIA@recerca.clinic.cat
First Name & Middle Initial & Last Name & Degree
Gloria Garrabou, MD
Facility Name
Hospital de la Santa Creu i Sant Pau
City
Barcelona
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Núria Vidal Fernandez
Email
mvidalf@santpau.cat
First Name & Middle Initial & Last Name & Degree
Maria Montserrat Olivé Plana, MD
Facility Name
Hospital General Universitario de Catalunya
City
Barcelona
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Susana Andrade Olivié
Phone
+34 931751575
Email
s.andrade@udic.es
First Name & Middle Initial & Last Name & Degree
Josep Gamez Carbonell, MD
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Montserrat Morales, MD
Email
montserrat.morales@salud.madrid.org
First Name & Middle Initial & Last Name & Degree
Montserrat Morales, MD
Facility Name
Department of Clinical Neurosciences, Addenbrooke's Hospital
City
Cambridge
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
Add-tr.mitoteam@nhs.net
First Name & Middle Initial & Last Name & Degree
Rita Horvath, MD
Facility Name
University College London Hospitals Nhs Foundation Trust
City
London
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Louise Germain
Phone
0203 108 6308
Email
l.germain@nhs.net
First Name & Middle Initial & Last Name & Degree
Robert Pitceathly, MD
Facility Name
Royal Victoria Infirmary - The Newcastle Upon Tyne Hospitals NHS Foundation Trust
City
Newcastle
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Grainne Gorman, MD
Phone
+44 (0)191 2820340
Email
grainne.gorman@ncl.ac.uk
First Name & Middle Initial & Last Name & Degree
Grainne Gorman, MD

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

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