| CTRI Number |
CTRI/2025/01/079746 [Registered on: 29/01/2025] Trial Registered Prospectively |
| Last Modified On: |
26/01/2025 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Randomized, Parallel Group Trial |
|
Public Title of Study
|
A study to assess the efficacy and safety of
Leriglitazone in cerebral
Adrenoleukodystrophy.
|
|
Scientific Title of Study
|
A clinical study to assess the efficacy and safety of
Leriglitazone in adult male subjects with cerebral
Adrenoleukodystrophy. |
| Trial Acronym |
NIL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| MT-3-01_Protocol version 4.0, dated 08.04.2024 |
Protocol Number |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Tarun Pandotra |
| Designation |
CEO and Founder |
| Affiliation |
GLOBAL REGULATORY & CONSUMER INSIGHTS |
| Address |
GLOBAL REGULATORY & CONSUMER INSIGHTS,
Sector-18B, Dwarka,
New-Delhi-110078, India
East
DELHI
110078
India |
| Phone |
8860009879 |
| Fax |
|
| Email |
tarunpandotra@gr-cis.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Tarun Pandotra |
| Designation |
CEO and Founder |
| Affiliation |
GLOBAL REGULATORY & CONSUMER INSIGHTS |
| Address |
GLOBAL REGULATORY & CONSUMER INSIGHTS,
Sector-18B, Dwarka,
New-Delhi-110078, India
DELHI
110078
India |
| Phone |
8860009879 |
| Fax |
|
| Email |
tarunpandotra@gr-cis.com |
|
Details of Contact Person
Public Query
|
| Name |
Tarun Pandotra |
| Designation |
CEO and Founder |
| Affiliation |
GLOBAL REGULATORY & CONSUMER INSIGHTS |
| Address |
GLOBAL REGULATORY & CONSUMER INSIGHTS,
Sector-18B, Dwarka,
New-Delhi-110078, India
DELHI
110078
India |
| Phone |
8860009879 |
| Fax |
|
| Email |
tarunpandotra@gr-cis.com |
|
|
Source of Monetary or Material Support
|
| GLOBAL REGULATORY & CONSUMER INSIGHTS
H-1/603, DDA, Multi-Storey,
Sector-18B, Dwarka,
New-Delhi-110078, India |
|
|
Primary Sponsor
|
| Name |
Minoryx Therapeutics S.L |
| Address |
Av. Ernest Lluch 32, TCM3,
08302 Mataró (Barcelona), Spain |
| Type of Sponsor |
Pharmaceutical industry-Global |
|
|
Details of Secondary Sponsor
|
| Name |
Address |
| Minoryx Therapeutics SL |
Av. Ernest Lluch 32, TCM3,
08302 Mataró (Barcelona), Spain |
|
|
Countries of Recruitment
|
Argentina
Brazil
Germany
India
Spain
United Kingdom
United States of America |
|
Sites of Study
|
| No of Sites = 1 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr C S Agarwal |
Sir Ganga Ram Hospital Marg |
Room no. 11, Department of neurology, Old
Rajinder Nagar, New Delhi, Delhi
110060.
New Delhi
DELHI |
8860009879
csyvaa@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 1 |
| Name of Committee |
Approval Status |
| Sir Ganga Ram Hospital Ethics Committee |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: G909||Disorder of the autonomic nervoussystem, unspecified, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
leriglitazone |
leriglitazone is used to treat Cerebral Adrenoleukodystrophy
Composition is 15mg/ml
Administered once in a day. |
| Comparator Agent |
Placebo |
Administered once in a day. |
|
|
Inclusion Criteria
|
| Age From |
18.00 Year(s) |
| Age To |
65.00 Year(s) |
| Gender |
Male |
| Details |
1. Subject is able to read and understand the ICF and has provided written informed
consent to participate in the study.
2. Subject is male and aged 18 years.
3. Subject has genetic confirmation of X ALD.
4. Subject has progressive cALD, defined as GdE plus brain lesions.
5. Subjects for whom HSCT is not recommended by the investigator or subject is not
willing to undergo HSCT.
6. Subject has a Loes score 0.5 and 12 at Screening.
7. Subject does not have major functional disability in the Major Functional
Disabilities Neurological Function Score (MFD-NFS), other than wheelchair
bound or total incontinence, which will be allowed as these are considered
expected symptoms of AMN in the time course of the disease.
8. Subject does not have major cognitive impairment which would impair his ability to
take part in the study as determined by the investigator at screening.
9. Subject has normal adrenal function or appropriate steroid replacement if adrenal
insufficiency is present. |
|
| ExclusionCriteria |
| Details |
1. Subject who had previous bone marrow transplantation (HSCT) or treatment with
ex-vivo gene therapy (eli-Cel).
2. Subject has known type 1 or type 2 diabetes.
3. Subject has known hypersensitivity or intolerance to pioglitazone or any other
thiazolidinedione.
4. Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones
within 3 months prior to Screening.
5. Subject has a requirement for treatment with a prohibited concomitant medication.
6. Subject has a previous or current history of congestive heart failure.
7. Subject has reduced left-ventricular ejection fraction or other clinically significant
cardiac abnormalities on echocardiogram that in the opinion of the investigator
could predispose the subject to volume overload or its associated consequences.
9. Subject has moderate or severe hepatic impairment (Child-Pugh classification
groups B or C).2
10. Subject with chronic kidney disease (CKD) of stage 3 or higher (according to the
Renal Association CKD staging).3
11. Subject has previous or current history of cancer, unless surgically resected and
without evidence of recurrence for a minimum of 5 years.
12. Subjects who are not surgically sterilized. If not surgically sterilized, subjects
should be willing to use adequate contraception and not donate sperm from the first
dose of the study drug until 90 days after the EOT visit.
13. Subject has contraindications for MRI such as having paramagnetic material in the
body (e.g., aneurysm clips, pacemakers, intraocular metal, or cochlear implants).
14. Subject with conditions that could modify absorption of the study drug.
15. Subject with current participation in another interventional clinical study or within
1 month prior to Screening.
16. Subject with other medical, neuropsychiatric or social conditions that, in the
opinion of the investigator, are likely to adversely affect the risk-benefit of study
participation, interfere with study compliance, or confound the study results.
|
|
|
Method of Generating Random Sequence
|
Not Applicable |
|
Method of Concealment
|
An Open list of random numbers |
|
Blinding/Masking
|
Open Label |
|
Primary Outcome
|
| Outcome |
TimePoints |
| To evaluate the efficacy of leriglitazone compared to placebo at increasing survival. |
To evaluate the efficacy of leriglitazone compared to placebo in 6 weeks. |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
To evaluate the efficacy of leriglitazone compared to placebo at slowing radiological
progression. |
Change from Baseline in Loes score at 18 months |
|
|
Target Sample Size
|
Total Sample Size="40"
Sample Size from India="20"
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" |
|
Phase of Trial
|
Phase 3 |
|
Date of First Enrollment (India)
|
10/02/2025 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
10/02/2025 |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
|
Estimated Duration of Trial
|
Years="3"
Months="0"
Days="0" |
|
Recruitment Status of Trial (Global)
|
Open to Recruitment |
| Recruitment Status of Trial (India) |
Open to Recruitment |
|
Publication Details
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
This phase 3, multicenter, randomized, double-blind, placebo-controlled, global clinical
study is designed to compare the efficacy and safety of leriglitazone with that of placebo in
the treatment of cALD. Approximately 40 male subjects will be enrolled. Subjects must be
at least ≥18 years of age with a confirmed genetic diagnosis of X-ALD and diagnosis of
cALD, defined as gadolinium-enhancing positive (GdE+) brain lesions, for whom
hematopoietic stem cell transplantation is not recommended by the investigator, or the
subject is not willing to undergo HSCT.
Subjects with adrenomyeloneuropathy (AMN) whose cALD status is unknown could attend
a prescreening visit (V-2) for a magnetic resonance imaging (MRI). This MRI should be
performed under the specified study conditions and would serve as screening MRI in case
the baseline visit is done within 15 days. If the subject does not have a genetic test already
available, a blood sample for this purpose will be collected at this prescreening visit. If
cALD is confirmed after review of the MRI, then the genetic testing will be performed to
confirm eligibility (Inclusion Criteria #3). A separate prescreening informed consent form
(ICF) will be provided to and signed by those subjects prior to any assessments being
performed.
Subjects must have a diagnosis of cALD, genetic confirmation of X-ALD, an MRI severity
score (referred to as Loes score) of ≥0.5 and ≤12, and GdE+ brain lesions at Screening.
Subjects will not be eligible if they had previous bone marrow transplantation (HSCT) or
treatment with ex-vivo gene therapy (elivaldogene autotemcel [eli-Cel]).
As depicted in Figure 1-1, this will be a double-blind placebo-controlled study with a
1:1 randomization, with a primary objective of assessing the efficacy of leriglitazone
compared to placebo at increasing survival. The individual subject follow-up is divided into
2 parts: a double-blind part and an open-label extension (OLE). The double-blind part will
last for a maximum of 36 months, after which the subject will be allowed to transition to
the OLE. Subjects and investigator site personnel will be kept blinded to the initial treatment
assignment until the end of the study. |