Parkinson DiseasePhase 1NCT04685265
A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of anle138b in Parkinson's Disease
A Phase 1 study testing anle138b in people with Parkinson Disease, run by MODAG GmbH at 1 site in United Kingdom.
Trial summary
The purpose of this study is to determine the safety, tolerability and blood levels of orally administered anle138b as well as the effect of food and early signs of efficacy in patients with mild to moderate Parkinson´s disease. Read more about this study
- Who is it for?
- Ages 40 to 80 years
- Conditions the study is for
- Parkinson Disease
- What is being tested?
- anle138b
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 22 Dec 2020to 22 Dec 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females with a diagnosis of idiopathic PD as defined by the Movement Disorders Society criteria (either fulfilling criteria for "Clinically Established PD" or for "Clinically Probable PD").
- Body mass index (BMI) 18.5 to 35.0 kg/m2 or 18.5 to <40.0 kg/m2 (Cohorts D and E) as measured at screening.
- Hoehn and Yahr stage I-III (able to walk unaided).
- Stable medication for PD for 1 month prior to screening at Quotient and anticipated over the study period.
- No history of dementia.
- Must be willing and able to communicate and participate in the whole study.
- Must provide written informed consent.
- Must agree to adhere to the contraception requirements defined in the study protocol.
Who cannot join
- Subjects who have received any IMP in a clinical research study within the 90 days prior to Day 1.
- Subjects who are, or are immediate family members of, a study site or sponsor employee.
- Evidence of current SARS-CoV-2 infection.
- History of any drug or alcohol abuse in the past 2 years.
- Regular alcohol consumption in males >21 units per week and females >14 units per week (1 unit = ½ pint beer, or a 25 mL shot of 40% spirit, 1.5 to 2 units = 125 mL glass of wine, depending on type).
- A confirmed positive alcohol breath test at screening or admission.
Show all 25Show fewer
- Current smokers and those who have smoked within the last 12 months. A confirmed breath carbon monoxide reading of greater than 10 ppm at screening or admission (Cohorts A to C only).
- Current users of e-cigarettes and nicotine replacement products and those who have used these products within the last 12 months (Cohorts A to C only).
- Females of childbearing potential including those who are pregnant or lactating (all female subjects must have a negative highly sensitive serum or urine pregnancy test). A woman is considered of childbearing potential unless she is permanently sterile (hysterectomy, bilateral salpingectomy, and bilateral oophorectomy) or is postmenopausal (had no menses for 12 months without an alternative medical cause and a serum follicle-stimulating hormone [FSH] concentration ≥40 IU/L).
- Male subjects with pregnant or lactating partners.
- Subjects who do not have suitable veins for multiple venepunctures/cannulation as assessed by the investigator or delegate at screening.
- Clinically significant abnormal coagulation (Cohort E only), clinical chemistry, haematology or urinalysis as judged by the investigator.
- Positive hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCV Ab) or human immunodeficiency virus (HIV) antibody results.
- History of clinically significant cardiovascular, renal, hepatic, dermatological, chronic respiratory or gastrointestinal disease or psychiatric disorder, as judged by the investigator.
- Serious adverse reaction or serious hypersensitivity to any drug or the formulation excipients.
- Presence or history of clinically significant allergy requiring treatment, as judged by the investigator. Hay fever is allowed unless it is active.
- Donation of blood or plasma within the previous 3 months or loss of greater than 400 mL of blood.
- Subjects who are taking, or have taken, any prescribed or over-the-counter drug or herbal remedies other than their regular medication according to the referral letter from NUH, and/or up to 2 g of paracetamol per day and/or HRT, in the 14 days before IMP administration.
- Subjects who are taking, or have taken, in the 14 days before IMP administration the drug warfarin.
- Subjects who are taking, or have taken, in the 14 days before IMP administration any drug that is considered to interfere with the objectives of the study, as determined by the concomitant medication oversight committee.
- Subjects who have participated in Cohorts A to C of this study will not be allowed to participate in Cohorts D or E, and subjects who have participated in Cohort D will not be allowed to participate in Cohort E.
- Failure to satisfy the investigator of fitness to participate for any other reason.
- Subjects who have a new headache with features suggestive of raised intracranial pressure, including papilloedema, vomiting, posture-related headache, or headache on waking from sleep (Cohort E only).
- Subjects who have a new headache with focal neurological symptoms, or non-focal neurological symptoms such as change in personality or memory, or an unexplained headache that becomes progressively severe, or an unexplained headache in anyone previously diagnosed with cancer (Cohort E only).
- Medical history or evidence of mass occupying lesion in brain or spinal cord or history of spinal cord injury, which could preclude the procedure of lumbar puncture and CSF collection (Cohort E only).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Oral Doses of anle138b, and to Characterise the Effect of Food of anle138b in Mild to Moderate Parkinson's Disease
The purpose of this study is to determine the safety, tolerability and blood levels of orally administered anle138b as well as the effect of food and early signs of efficacy in patients with mild to moderate Parkinson´s disease.
Read the detailed description
This is a two centre, double-blind, randomised, placebo-controlled, multiple ascending dose study in patients with mild to moderate Parkinson´s disease. It is planned to enrol up to 5 cohorts. Cohorts A-C consist of up to 8 subjects. In Cohorts A and B, subjects will be randomly assigned to receive multiple ascending oral doses of anle138b or matching placebo (6 active investigational medicinal product [IMP], 2 placebo per cohort in cohorts A-C) for 7 days in a sequential escalating manner. Subjects in cohort A and B will be dosed once dialy and subjects in cohort C will be dosed twice a day. Subjects in Cohorts A and B will also receive an additional single oral dose of active IMP or matching placebo on Day 9 of the study for an assessment of the effect of food on the PK of anle138b in PD patients. Subjects in cohort D will be randomly assigned to receive QD doses of anle138b or matching placebo (placebo vs 150 mg vs 300 mg; 1:1:1) for 7 days in fasted state. Subjects in Cohort E will be randomly assigned to receive QD doses of either 300 mg anle138b or matching placebo (1:1) for 28 days taken in the non-fasted state.
- Conditions
- Parkinson Disease
- Being tested
- anle138b, Placebo
- Keywords
- anle138b, alpha-Synuclein, Oligomer modulator, Multiple System Atrophy, Parkinson Disease, Alzheimer Disease, Neurodegenerative diseases, Tauopathies, Amyloid
- Medical subject headings
- Parkinson Disease, Parkinson Disease 4, Autosomal Dominant Lewy Body, Multiple System Atrophy, Alzheimer Disease, Neurodegenerative Diseases, Tauopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anle138bDrug
capsule containing excipient and anle138b
PlaceboDrug
matching placebo capsule containing excipient
| Study group | Role | What they receive |
|---|---|---|
| anle138b | Active comparator | 150 mg and higher dosage Receives: Drug: anle138b |
| Placebo | Placebo comparator | Matching placebo dosage Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Adverse events
Measured: Day 1 to day 14-16: cohorts A-C; day 1 to week 6 post dosing: cohort D
Incidence of treatment-emergent adverse events in PD patients with multiple ascending doses of anle138b taken in the fed state (Cohorts A and B).
Adverse events
Measured: From fed dosing (day 9) to day 12-14
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Blood pressure
Measured: Day 1 to week 1 post dosing: cohorts A-C; day 1 to week 6 post dosing: cohort D
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Heart rate
Measured: Day 1 to week 1 post dosing: cohorts A-C; day 1 to week 6 post dosing: cohort D
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Oral temperature
Measured: Day 1 to week 1 post dosing: cohorts A-C; day 1 to week 6 post dosing: cohort D
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fed state (Cohorts A and B).
Blood pressure
Measured: From fed dosing to 1 week post dosing
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fed state (Cohorts A and B).
Heart rate
Measured: From fed dosing to 1 week post dosing
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fed state (Cohorts A and B).
Oral temperature
Measured: From fed dosing to 1 week post dosing
Incidence of treatment-emergent ECG changes in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Electrocardiogram (ECG) parameters including QT interval corrected for heart rate using Fridericia's formula (QTcF)
Measured: Day 1 to week 1 post dosing: cohorts A-C; day 1 to week 6 post dosing: cohort D
Incidence of treatment-emergent ECG changes in PD patients with multiple ascending doses of anle138b taken in the fed state (Cohorts A and B).
Electrocardiogram (ECG) parameters including QT interval corrected for heart rate using Fridericia's formula (QTcF)
Measured: From fed dosing to 1 week post dosing
Incidence of treatment-emergent changes in physical examination in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Physical examination findings
Measured: Day 1 to week 1 post dosing: cohorts A-C; day 1 to week 6 post dosing: cohort D
Incidence of treatment-emergent changes in physical examination in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Physical examination findings
Measured: From fed dosing to 1 week post dosing
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Clinical laboratory Tests: Hematology
Measured: Day 1 to day 8
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Clinical chemistry: Renal function tests
Measured: Day 1 to day 8
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Clinical chemistry: Hepatic enzymes
Measured: Day 1 to day 8
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Clinical chemistry: Electrolytes
Measured: Day 1 to day 8
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Clinical chemistry: Creatine kinase
Measured: Day 1 to day 8
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Clinical laboratory Tests: Hematology
Measured: From fed dosing to 24 hours post dosing
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Clinical chemistry: Renal function tests
Measured: From fed dosing to 24 hours post dosing
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Clinical chemistry: Hepatic enzymes
Measured: From fed dosing to 24 hours post dosing
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Clinical chemistry: Electrolytes
Measured: From fed dosing to 24 hours post dosing
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Clinical chemistry: Creatine kinase
Measured: From fed dosing to 24 hours post dosing
Incidence of treatment-emergent adverse events in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Adverse events
Measured: Day 1 to week 6 post dosing
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Blood pressure
Measured: Day 1 to week 6 post dosing
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Heart rate
Measured: Day 1 to week 6 post dosing
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Oral temperature
Measured: Day 1 to week 6 post dosing
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Electrocardiogram (ECG) parameters including QT interval corrected for heart rate using Fridericia's formula (QTcF)
Measured: Day 1 to week 6 post dosing
Incidence of treatment-emergent changes in physical examination in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Physical examination findings
Measured: Day 1 to week 6 post dosing
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Clinical laboratory Tests: Hematology
Measured: Day 1 to 24 hours post dosing
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Clinical chemistry: Renal function tests
Measured: Day 1 to 24 hours post dosing
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Clinical chemistry: Hepatic enzymes
Measured: Day 1 to 24 hours post dosing
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Clinical chemistry: Electrolytes
Measured: Day 1 to 24 hours post dosing
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Clinical chemistry: Creatine kinase
Measured: Day 1 to 24 hours post dosing
Secondary outcomes (24)
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
PK parameter: Tlag for anle138b.
Measured: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
PK parameter: Tmax for anle138b.
Measured: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
PK parameter: Cmax for anle138b.
Measured: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohort B).
PK parameter: C12 for anle138b.
Measured: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
PK parameter: C24 for anle138b.
Measured: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
PK parameter: AUC(0-tau) for anle138b.
Measured: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
PK parameter: Lambda-z for anle138b.
Measured: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
PK parameter: T1/2 for anle138b.
Measured: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
PK parameter: Accumulation ratio for Cmax (Day 7) for anle138b.
Measured: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
PK parameter: Accumulation ratio for AUC (Day 7) for anle138b.
Measured: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fed state (Cohorts A and B).
PK parameter: Cmax for anle138b.
Measured: From fed dosing to 48 hours post dosing.
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fed state (Cohorts A and B).
PK parameter: AUC(0-24) for anle138b.
Measured: From fed dosing to 48 hours post dosing.
Effect of multiple ascending doses of anle138b on the motor status of PD patients
Changes from baseline in the "Movement Disorder Society-sponsored revision of the Unified PD Rating Scale" (MDS-UPDRS) ranging from 0 to 260 points with less points meaning less severity.
Measured: Admission to follow-up visit (days 14-16 for cohorts A and B; days 12-14 for cohort C; week 6 for cohorts D and E)
Effect of multiple doses of anle138b on the CSF levels of anle138b in PD patients
Quantification of anle138b
Measured: single time point 3 hours post dose on dosing day 5 (cohort B)
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
PK parameter: Tlag for anle138b
Measured: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
PK parameter: Tmax for anle138b
Measured: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
PK parameter: Cmax for anle138b
Measured: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
PK parameter: C12 for anle138b
Measured: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
PK parameter: C24 for anle138b
Measured: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
PK parameter: AUC(0-tau) for anle138b
Measured: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
PK parameter: Lambda-z for anle138b
Measured: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
PK parameter: T1/2 for anle138b
Measured: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
PK parameter: Accumulation ratio for Cmax for anle138b
Measured: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
PK parameter: Accumulation ratio for AUC for anle138b
Measured: Day 1 to day 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2020
- Study start22 Dec 2020
- Primary completion7 Nov 2022
- Study completion22 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- MODAG GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Aptuit, Quotient Sciences, Nottingham University Hospitals NHS Trust
- Responsible party
- Sponsor
- Investigators
- Nand Singh, BSc, MD, DPM, MFPM, Principal investigator, Quotient Sciences Mere Way Ruddington Fields Ruddington Nottingham NG11 6JS, UKJonathan Evans, MD, Principal investigator, Nottingham University Hospitals NHS Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04685265
- Sponsor's protocol ID
- anle138b-P1-02
- Other IDs
- 2020-004869-38
- Status last verified
- Mar 2023
- First submitted
- 17 Dec 2020
- Last updated
- 22 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Wagner J, Ryazanov S, Leonov A, Levin J, Shi S, Schmidt F, Prix C, Pan-Montojo F, Bertsch U, Mitteregger-Kretzschmar G, Geissen M, Eiden M, Leidel F, Hirschberger T, Deeg AA, Krauth JJ, Zinth W, Tavan P, Pilger J, Zweckstetter M, Frank T, Bahr M, Weishaupt JH, Uhr M, Urlaub H, Teichmann U, Samwer M, Botzel K, Groschup M, Kretzschmar H, Griesinger C, Giese A. Anle138b: a novel oligomer modulator for disease-modifying therapy of neurodegenerative diseases such as prion and Parkinson's disease. Acta Neuropathol. 2013 Jun;125(6):795-813. doi: 10.1007/s00401-013-1114-9. Epub 2013 Apr 19.
PubMed 23604588Levin J, Schmidt F, Boehm C, Prix C, Botzel K, Ryazanov S, Leonov A, Griesinger C, Giese A. The oligomer modulator anle138b inhibits disease progression in a Parkinson mouse model even with treatment started after disease onset. Acta Neuropathol. 2014 May;127(5):779-80. doi: 10.1007/s00401-014-1265-3. Epub 2014 Mar 11. No abstract available.
PubMed 24615514Heras-Garvin A, Weckbecker D, Ryazanov S, Leonov A, Griesinger C, Giese A, Wenning GK, Stefanova N. Anle138b modulates alpha-synuclein oligomerization and prevents motor decline and neurodegeneration in a mouse model of multiple system atrophy. Mov Disord. 2019 Feb;34(2):255-263. doi: 10.1002/mds.27562. Epub 2018 Nov 19.
PubMed 30452793Wegrzynowicz M, Bar-On D, Calo' L, Anichtchik O, Iovino M, Xia J, Ryazanov S, Leonov A, Giese A, Dalley JW, Griesinger C, Ashery U, Spillantini MG. Depopulation of dense alpha-synuclein aggregates is associated with rescue of dopamine neuron dysfunction and death in a new Parkinson's disease model. Acta Neuropathol. 2019 Oct;138(4):575-595. doi: 10.1007/s00401-019-02023-x. Epub 2019 May 31.
PubMed 31165254Wagner J, Krauss S, Shi S, Ryazanov S, Steffen J, Miklitz C, Leonov A, Kleinknecht A, Goricke B, Weishaupt JH, Weckbecker D, Reiner AM, Zinth W, Levin J, Ehninger D, Remy S, Kretzschmar HA, Griesinger C, Giese A, Fuhrmann M. Reducing tau aggregates with anle138b delays disease progression in a mouse model of tauopathies. Acta Neuropathol. 2015 Nov;130(5):619-31. doi: 10.1007/s00401-015-1483-3. Epub 2015 Oct 6.
PubMed 26439832Martinez Hernandez A, Urbanke H, Gillman AL, Lee J, Ryazanov S, Agbemenyah HY, Benito E, Jain G, Kaurani L, Grigorian G, Leonov A, Rezaei-Ghaleh N, Wilken P, Arce FT, Wagner J, Fuhrmann M, Caruana M, Camilleri A, Vassallo N, Zweckstetter M, Benz R, Giese A, Schneider A, Korte M, Lal R, Griesinger C, Eichele G, Fischer A. The diphenylpyrazole compound anle138b blocks Abeta channels and rescues disease phenotypes in a mouse model for amyloid pathology. EMBO Mol Med. 2018 Jan;10(1):32-47. doi: 10.15252/emmm.201707825.
PubMed 29208638
Study at a glance
From the registry record, last updated by the sponsor on 22 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Oral Doses of anle138b, and to Characterise the Effect of Food of anle138b in Mild to Moderate Parkinson's Disease
- ClinicalTrials.gov ID
- NCT04685265
- Acronym / sponsor ID
- anle138b-P1-02
- Conditions
- Parkinson Disease
- Keywords
- anle138b, alpha-Synuclein, Oligomer modulator, Multiple System Atrophy, Parkinson Disease, Alzheimer Disease, Neurodegenerative diseases, Tauopathies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- MODAG GmbHIndustry (pharma, biotech, devices)
- Collaborators
- Aptuit, Quotient Sciences, Nottingham University Hospitals NHS Trust
- Study start
- 22 Dec 2020 (actual)
- Primary completion
- 7 Nov 2022 (actual)
- Study completion
- 22 Dec 2022 (actual)
- First posted
- 28 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine the safety, tolerability and blood levels of orally administered anle138b as well as the effect of food and early signs of efficacy in patients with mild to moderate Parkinson´s disease.
Detailed description
This is a two centre, double-blind, randomised, placebo-controlled, multiple ascending dose study in patients with mild to moderate Parkinson´s disease. It is planned to enrol up to 5 cohorts. Cohorts A-C consist of up to 8 subjects. In Cohorts A and B, subjects will be randomly assigned to receive multiple ascending oral doses of anle138b or matching placebo (6 active investigational medicinal product [IMP], 2 placebo per cohort in cohorts A-C) for 7 days in a sequential escalating manner. Subjects in cohort A and B will be dosed once dialy and subjects in cohort C will be dosed twice a day. Subjects in Cohorts A and B will also receive an additional single oral dose of active IMP or matching placebo on Day 9 of the study for an assessment of the effect of food on the PK of anle138b in PD patients. Subjects in cohort D will be randomly assigned to receive QD doses of anle138b or matching placebo (placebo vs 150 mg vs 300 mg; 1:1:1) for 7 days in fasted state. Subjects in Cohort E will be randomly assigned to receive QD doses of either 300 mg anle138b or matching placebo (1:1) for 28 days taken in the non-fasted state.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| anle138b | Active comparator | Drug: anle138b150 mg and higher dosage |
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo dosage |
Who may qualify
- Ages
- 40 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Males and females with a diagnosis of idiopathic PD as defined by the Movement Disorders Society criteria (either fulfilling criteria for "Clinically Established PD" or for "Clinically Probable PD").
- Body mass index (BMI) 18.5 to 35.0 kg/m2 or 18.5 to <40.0 kg/m2 (Cohorts D and E) as measured at screening.
- Hoehn and Yahr stage I-III (able to walk unaided).
- Stable medication for PD for 1 month prior to screening at Quotient and anticipated over the study period.
- No history of dementia.
Key exclusion criteria (25 listed)
- Subjects who have received any IMP in a clinical research study within the 90 days prior to Day 1.
- Subjects who are, or are immediate family members of, a study site or sponsor employee.
- Evidence of current SARS-CoV-2 infection.
- History of any drug or alcohol abuse in the past 2 years.
- Regular alcohol consumption in males >21 units per week and females >14 units per week (1 unit = ½ pint beer, or a 25 mL shot of 40% spirit, 1.5 to 2 units = 125 mL glass of wine, depending on type).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| anle138bcapsule containing excipient and anle138b | Drug | anle138b |
| Placebomatching placebo capsule containing excipient | Drug | Placebo |
Primary outcome
Incidence of treatment-emergent adverse events in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 14-16: cohorts A-C; day 1 to week 6 post dosing: cohort D
Adverse events
Incidence of treatment-emergent adverse events in PD patients with multiple ascending doses of anle138b taken in the fed state (Cohorts A and B).
Time frame: From fed dosing (day 9) to day 12-14
Adverse events
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to week 1 post dosing: cohorts A-C; day 1 to week 6 post dosing: cohort D
Blood pressure
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to week 1 post dosing: cohorts A-C; day 1 to week 6 post dosing: cohort D
Heart rate
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to week 1 post dosing: cohorts A-C; day 1 to week 6 post dosing: cohort D
Oral temperature
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fed state (Cohorts A and B).
Time frame: From fed dosing to 1 week post dosing
Blood pressure
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fed state (Cohorts A and B).
Time frame: From fed dosing to 1 week post dosing
Heart rate
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken in the fed state (Cohorts A and B).
Time frame: From fed dosing to 1 week post dosing
Oral temperature
Incidence of treatment-emergent ECG changes in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to week 1 post dosing: cohorts A-C; day 1 to week 6 post dosing: cohort D
Electrocardiogram (ECG) parameters including QT interval corrected for heart rate using Fridericia's formula (QTcF)
Incidence of treatment-emergent ECG changes in PD patients with multiple ascending doses of anle138b taken in the fed state (Cohorts A and B).
Time frame: From fed dosing to 1 week post dosing
Electrocardiogram (ECG) parameters including QT interval corrected for heart rate using Fridericia's formula (QTcF)
Incidence of treatment-emergent changes in physical examination in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to week 1 post dosing: cohorts A-C; day 1 to week 6 post dosing: cohort D
Physical examination findings
Incidence of treatment-emergent changes in physical examination in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Time frame: From fed dosing to 1 week post dosing
Physical examination findings
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 8
Clinical laboratory Tests: Hematology
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 8
Clinical chemistry: Renal function tests
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 8
Clinical chemistry: Hepatic enzymes
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 8
Clinical chemistry: Electrolytes
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 8
Clinical chemistry: Creatine kinase
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Time frame: From fed dosing to 24 hours post dosing
Clinical laboratory Tests: Hematology
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Time frame: From fed dosing to 24 hours post dosing
Clinical chemistry: Renal function tests
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Time frame: From fed dosing to 24 hours post dosing
Clinical chemistry: Hepatic enzymes
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Time frame: From fed dosing to 24 hours post dosing
Clinical chemistry: Electrolytes
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken in fed state (Cohorts A and B).
Time frame: From fed dosing to 24 hours post dosing
Clinical chemistry: Creatine kinase
Incidence of treatment-emergent adverse events in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to week 6 post dosing
Adverse events
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to week 6 post dosing
Blood pressure
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to week 6 post dosing
Heart rate
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to week 6 post dosing
Oral temperature
Incidence of treatment-emergent changes in vital signs in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to week 6 post dosing
Electrocardiogram (ECG) parameters including QT interval corrected for heart rate using Fridericia's formula (QTcF)
Incidence of treatment-emergent changes in physical examination in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to week 6 post dosing
Physical examination findings
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to 24 hours post dosing
Clinical laboratory Tests: Hematology
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to 24 hours post dosing
Clinical chemistry: Renal function tests
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to 24 hours post dosing
Clinical chemistry: Hepatic enzymes
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to 24 hours post dosing
Clinical chemistry: Electrolytes
Incidence of treatment-emergent changes in clinical laboratory parameters in PD patients with multiple ascending doses of anle138b taken 28 days in the non-fasted state (cohort E).
Time frame: Day 1 to 24 hours post dosing
Clinical chemistry: Creatine kinase
24 secondary outcomes
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohort B).
Time frame: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fasted state (cohorts A-C + D).
Time frame: Day 1 to day 9
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fed state (Cohorts A and B).
Time frame: From fed dosing to 48 hours post dosing.
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the fed state (Cohorts A and B).
Time frame: From fed dosing to 48 hours post dosing.
Effect of multiple ascending doses of anle138b on the motor status of PD patients
Time frame: Admission to follow-up visit (days 14-16 for cohorts A and B; days 12-14 for cohort C; week 6 for cohorts D and E)
Effect of multiple doses of anle138b on the CSF levels of anle138b in PD patients
Time frame: single time point 3 hours post dose on dosing day 5 (cohort B)
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
Time frame: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
Time frame: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
Time frame: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
Time frame: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
Time frame: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
Time frame: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
Time frame: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
Time frame: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
Time frame: Day 1 to day 30
Oral pharmacokinetics (PK) of multiple ascending doses of anle138b in PD patients taken in the non-fasted state (cohort E)
Time frame: Day 1 to day 30
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Mar 2023.
- Background: PubMed 23604588Wagner J, Ryazanov S, Leonov A, Levin J, Shi S, Schmidt F, Prix C, Pan-Montojo F, Bertsch U, Mitteregger-Kretzschmar G, Geissen M, Eiden M, Leidel F, Hirschberger T, Deeg AA, Krauth JJ, Zinth W, Tavan P, Pilger J, Zweckstetter M, Frank T, Bahr M, Weishaupt JH, Uhr M, Urlaub H, Teichmann U, Samwer M, Botzel K, Groschup M, Kretzschmar H, Griesinger C, Giese A. Anle138b: a novel oligomer modulator for disease-modifying therapy of neurodegenerative diseases such as prion and Parkinson's disease. Acta Neuropathol. 2013 Jun;125(6):795-813. doi: 10.1007/s00401-013-1114-9. Epub 2013 Apr 19.
- Background: PubMed 24615514Levin J, Schmidt F, Boehm C, Prix C, Botzel K, Ryazanov S, Leonov A, Griesinger C, Giese A. The oligomer modulator anle138b inhibits disease progression in a Parkinson mouse model even with treatment started after disease onset. Acta Neuropathol. 2014 May;127(5):779-80. doi: 10.1007/s00401-014-1265-3. Epub 2014 Mar 11. No abstract available.
- Background: PubMed 30452793Heras-Garvin A, Weckbecker D, Ryazanov S, Leonov A, Griesinger C, Giese A, Wenning GK, Stefanova N. Anle138b modulates alpha-synuclein oligomerization and prevents motor decline and neurodegeneration in a mouse model of multiple system atrophy. Mov Disord. 2019 Feb;34(2):255-263. doi: 10.1002/mds.27562. Epub 2018 Nov 19.
- Background: PubMed 31165254Wegrzynowicz M, Bar-On D, Calo' L, Anichtchik O, Iovino M, Xia J, Ryazanov S, Leonov A, Giese A, Dalley JW, Griesinger C, Ashery U, Spillantini MG. Depopulation of dense alpha-synuclein aggregates is associated with rescue of dopamine neuron dysfunction and death in a new Parkinson's disease model. Acta Neuropathol. 2019 Oct;138(4):575-595. doi: 10.1007/s00401-019-02023-x. Epub 2019 May 31.
- Background: PubMed 26439832Wagner J, Krauss S, Shi S, Ryazanov S, Steffen J, Miklitz C, Leonov A, Kleinknecht A, Goricke B, Weishaupt JH, Weckbecker D, Reiner AM, Zinth W, Levin J, Ehninger D, Remy S, Kretzschmar HA, Griesinger C, Giese A, Fuhrmann M. Reducing tau aggregates with anle138b delays disease progression in a mouse model of tauopathies. Acta Neuropathol. 2015 Nov;130(5):619-31. doi: 10.1007/s00401-015-1483-3. Epub 2015 Oct 6.
- Background: PubMed 29208638Martinez Hernandez A, Urbanke H, Gillman AL, Lee J, Ryazanov S, Agbemenyah HY, Benito E, Jain G, Kaurani L, Grigorian G, Leonov A, Rezaei-Ghaleh N, Wilken P, Arce FT, Wagner J, Fuhrmann M, Caruana M, Camilleri A, Vassallo N, Zweckstetter M, Benz R, Giese A, Schneider A, Korte M, Lal R, Griesinger C, Eichele G, Fischer A. The diphenylpyrazole compound anle138b blocks Abeta channels and rescues disease phenotypes in a mouse model for amyloid pathology. EMBO Mol Med. 2018 Jan;10(1):32-47. doi: 10.15252/emmm.201707825.
- Sponsor HomepageLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nand Singh, BSc, MD, DPM, MFPM principal investigator
Quotient Sciences Mere Way Ruddington Fields Ruddington Nottingham NG11 6JS, UK
Jonathan Evans, MD principal investigator
Nottingham University Hospitals NHS Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing anle138b in people with Parkinson Disease, run by MODAG GmbH at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events in PD patients with multiple ascending doses of anle138b taken in the fasted state (cohorts A-C + D). (time frame: Day 1 to day 14-16: cohorts A-C; day 1 to week 6 post dosing: cohort D). The study lists 33 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04685265, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Mar 2023.