Clinical Trials Register

Summary
EudraCT Number:	2022-000299-20
Sponsor's Protocol Code Number:	SPI-62-CL-2003
National Competent Authority:	Germany - BfArM
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
Date on which this record was first entered in the EudraCT database:	2022-04-22
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Germany - BfArM

A.2

EudraCT number

2022-000299-20

A.3

Full title of the trial

A trial of prednisolone in combination with SPI-62 or placebo in subjects with polymyalgia rheumatica (PMR)

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A study to investigate whether application of SPI-62 necessitates an increase of the prednisolone dose to maintain the same efficacy of treatment of polymyalgia rheumatica

A.4.1

Sponsor's protocol code number

SPI-62-CL-2003

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Sparrow Pharmaceuticals, Inc.
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Sparrow Pharmaceuticals, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Sparrow Pharmaceuticals, Inc.
B.5.2	Functional name of contact point	David A. Katz
B.5.3	Address:
B.5.3.1	Street Address	1050 SW 6th Avenue, Suite 1100
B.5.3.2	Town/ city	Portland
B.5.3.3	Post code	Oregon 97204
B.5.3.4	Country	United States
B.5.4	Telephone number	+13127528446
B.5.6	E-mail	info@sparrowpharma.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Clofutriben
D.3.2	Product code	SPI-62
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Does not exist
D.3.9.1	CAS number	1204178-50-6
D.3.9.2	Current sponsor code	SPI-62
D.3.9.3	Other descriptive name	ASP3662, AS2473662
D.3.9.4	EV Substance Code	SUB263389
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Prednisolon 5 mg JENAPHARM® Tabletten
D.2.1.1.2	Name of the Marketing Authorisation holder	mibe GmbH Arzneimittel
D.2.1.2	Country which granted the Marketing Authorisation	Germany
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Prednisolone
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Prednisolone
D.3.9.1	CAS number	50-24-8
D.3.9.2	Current sponsor code	Prednisolone
D.3.9.4	EV Substance Code	SUB10018MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Clofutriben
D.3.2	Product code	SPI-62
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Does not exist
D.3.9.1	CAS number	1204178-50-6
D.3.9.2	Current sponsor code	SPI-62
D.3.9.3	Other descriptive name	ASP3662, AS2473662
D.3.9.4	EV Substance Code	SUB263389
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0.2
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule, hard
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

polymyalgia rheumatica

E.1.1.1

Medical condition in easily understood language

Polymyalgia rheumatica is a condition that causes pain, stiffness and inflammation in the muscles around the shoulders, neck and hips.

E.1.1.2

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	PT
E.1.2	Classification code	10036099
E.1.2	Term	Polymyalgia rheumatica
E.1.2	System Organ Class	10028395 - Musculoskeletal and connective tissue disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

• To identify whether a prednisolone dose adjustment is necessary to provide equivalent efficacy when administered with SPI-62 in subjects with PMR and, if so, estimate the adjustment

E.2.2

Secondary objectives of the trial

• To observe whether SPI-62 mitigates prednisolone toxicity in subjects with PMR and, if so, estimate the appropriate dose to administer in combination withprednisolone
• To characterize the pharmacological activity of SPI-62 in subjects with PMR
• To report the safety of SPI-62 in subjects with PMR treated with prednisolone

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Able to understand the nature and scope of the trial.
2. Written informed consent given prior to any trial-related procedures.
3. At least 18 years old.
4. Diagnosis of polymyalgia rheumatica according to EULAR/ACR classification criteria.
5. Absence of PMR relapse based on symptoms.
6. Absence of PMR relapse based on acute phase markers
7. Daily oral prednisolone 10 mg dose that will have been stable for at least 1 week at the Baseline Visit and is expected to remain stable during the treatment period.

E.4

Principal exclusion criteria

1. Known hypersensitivity to prednisolone, SPI-62 or any of the excipients.
2. Systemic fungal infection.
3. Administration of any vaccine within 4 weeks prior to the Baseline Visit or during the trial.
4. Any relative contraindication for prednisolone of sufficient severity that the investigator considers that the subject should receive immediate additional treatment or not continue prednisolone.
5. Fulfills all criteria and clearly qualifies to use methotrexate according to national or EULAR/ACR guideline for treatment of PMR, unless the subject refuses to take methotrexate or has a contraindication to methotrexate.
6. A diagnosis or any clinical features of giant cell arteritis.
7. Any known autoimmune disease (e.g., late-onset rheumatoid arthritis) other than PMR.
8. Use of medications other than oral prednisolone for treatment of PMR.
9. Use of other medications likely to interfere with trial assessments.
See Section 7 for lists of medications that are and are not allowed during the trial. Use of any other medication (prescription, OTC, or traditional) or supplement within 4 weeks of the Baseline Visit is allowed only if the medication and dose (or PRN use) are approved by the Medical Monitor or Sponsor, the dose has been stable for at least 4 weeks, and the dose is expected to remain stable for the duration of the trial.
10. History of clinically significant labile diabetes or hypertension during glucocorticoid therapy.
11. History or diagnosis of endogenous hypercortisolism.
12. Moderate or severe renal impairment.
Defined by an estimated glomerular filtration rate (2021 CKD-EPI creatinine equation) repeatedly < 60 mL/min/1.73m2.
13. Medically significant liver disease.
Including cirrhosis, chronic active hepatitis, chronic persistent hepatitis, or subjects with serum total bilirubin > 1.5 × ULN (unless previously diagnosed with benign Gilbert’s disease) or serum ALT or AST >3 × ULN.
14. Medically significant cardiovascular or ECG abnormalities.
This includes subjects with recent (< 1 year) myocardial infarction or stroke, orthostatic or vasovagal syncope, QT interval corrected (QTc) interval > 500 ms, uncorrected QT interval > 600 ms, or evidence of significant, life-threatening arrhythmia or bradycardia (heart rate < 45 bpm).
15. History of idiopathic thrombocytopenic purpura.
16. Recent severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection, which is defined as:
Positive lab-based test (polymerase chain reaction or antigen test) for active infection within the past 4 weeks or hospitalization for coronavirus disease 2019 (COVID-19) within the past 6 months.
17. History of cancer within 3 years other than non-melanoma skin cancer or early-stage prostate cancer (not dependent on hormone-suppression therapy).
18. Any major surgery, or significant post-operative sequelae, within 1 month prior to informed consent or planned during the trial.
19. Pregnant, lactating, or woman of child-bearing potential unwilling to adhere to highly effective contraceptive use or abstinence as defined in Appendix 5.
20. Man unwilling to adhere to highly effective contraceptive use or abstinence as defined in Appendix 5.
21. Participation in any clinical trial of an experimental drug within 5 half-lives or 1 month prior to informed consent (or 3 months for biologic and long-lasting experimental therapies), whichever is longer.
22. Receipt of blood products within 2 months prior to Screening.
23. Donation of blood from 3 months prior to Screening, plasma from 2 weeks prior to Screening, or platelets from 6 weeks prior to Screening. Participants are not to donate blood, plasma, or platelets during the trial.
24. Any current or prior medical condition expected to interfere with the conduct of the trial or the evaluation of its results (e.g., asthma that requires regular or intermittent glucocorticoid treatment), or that requires immediate medical attention (e.g., uncontrolled diabetes or hypertension).
25. Employees of the Sponsor or employees or relatives of the Investigator.
26. Persons committed to an institution by virtue of a judicial order or an order issued by an administrative authority.

E.5 End points

E.5.1

Primary end point(s)

Fibrinogen, erythrocyte sedimentation ratio, C-reactive protein

E.5.1.1

Timepoint(s) of evaluation of this end point

2 weeks of treatment

E.5.2

Secondary end point(s)

oral glucose tolerance test glucose area under the concentration‑time curve, osteocalcin, urinary 11β-hydroxysteroid dehydrogenase type 1 ratio

E.5.2.1

Timepoint(s) of evaluation of this end point

2 weeks of treatment

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

Yes

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

sequential

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

6 sequential cohorts

sequential group

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Study patients will return to their previous standard-of-care treatment.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2022-06-23

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-06-20

P. End of Trial
P.	End of Trial Status	Trial now transitioned

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