A Phase 2 Study to Evaluate Efficacy, Safety, and Pharmacokinetics of PBF-999 in the Treatment of Patients with Prader-Willi Syndrome.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Palo Biofarma S.L.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Phenomena and Processes [G] - Genetic Phenomena [G05]

Trial duration

22 Feb 2023 → 20 Feb 2026

Decision date (initial)

2022-12-01

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Palobiofarma, S.L

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic, Dose response, Efficacy

Evaluate the safety and tolerability of PBF-999 in patients with Prader-Willi Syndrome over 28 days and 90 days.

Secondary objectives 1

1. Explore pharmacokinetics (PK) profile and preliminary therapeutic efficacy associated with PBF-999 through biomarker analysis and clinical assessments.

Conditions and MedDRA coding

Prader-Willi Syndrome

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

IPD plan description

Data will not be shared by now.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Male and female outpatients between 12 and 65 years of age inclusive, before signing informed consent.
2. Confirmed diagnosis of PWS based on genetic confirmation using DNA methylation test.
3. Body mass index (BMI) up to 65 kg/m2, inclusive, at Visit 1. This criterion applies only for adult patients.
4. No evidence of weight excursion beyond 10% of baseline weight within 3 months prior to Visit 1 (self- or caregiver-reported). This criterion applies only for adult patients.
5. Patients must provide assent and have a reliable caregiver (must have been caring for the patient for at least 6 months) who provides a separate written informed consent to participate. In the case of paediatric population, parent’s consent shall be obtained too.
6. Women of child-bearing potential (WCBP) must have a negative pregnancy test. All WCBP, sexually active male patients, and all opposite sex partners of patients should agree to use medically approved effective methods of birth control.

Exclusion criteria 13

1. Are currently enrolled in any other clinical trial involving a study drug.
2. Participated in a clinical trial within 30 days (defined as last dose of study drug), prior to the PBF-999 first dose.
3. Are currently living in a group home for more than 50% of the time, except when in the group home, there is a primary caregiver throughout the study in frequent contact with the patient (defined as at least 4 awake hours per day).
4. Have clinical laboratory test results outside normal reference range, or any clinically significant laboratory abnormality, that in the judgment of the investigator, indicates a medical problem that would preclude study participation.
5. Are hypertensive (defined as sitting systolic BP ≥140 mmHg and diastolic BP ≥90 mmHg) on or off medications for the treatment of hypertension. Blood pressure may be re-tested up to 2 additional times, under well-rested conditions.
6. In addition to conditions described below, have a history or presence of any other medical illness including but not limited to any autoimmune disorder, cardiovascular, hepatic, respiratory, hematological, or uncontrolled neurological disease.
7. Have evidence of significant active or unstable/uncontrolled psychiatric disease by medical history, such as bipolar disorder, schizophrenia, personality disorders, or other serious mood or anxiety disorders.
8. Have an abnormality in the 12-lead electrocardiogram (ECG) or an abnormality that, in the opinion of the investigator, increases the risks associated with participating in the study.
9. Have a family history of Long QT Syndrome.
10. Patients on weight loss medications within 30 days of dosing (GLP1 agonists at doses for Diabetes treatment are allowed).
11. Regular user of known drugs of abuse.
12. Any major surgery within 60 days prior to the first dose or has planned elective surgeries to occur during the study.
13. Unsuitable for inclusion in the study in the opinion of the investigator.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Treatment-emergent adverse events.

Secondary endpoints 4

1. Post-treatment HQ-CT questionnaire total score
2. CGIC questionnaire score.
3. PBF-999 PK parameters: AUC0-3h; Cmax.
4. Physical and biological markers.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Palo Biofarma S.L.

Sponsor organisation

Palo Biofarma S.L.

Address

Calle D'ernest Lluch 32

City

Mataro

Postcode

08302

Country

Spain

Scientific contact point

Organisation

Palo Biofarma S.L.

Contact name

Chief of Clinical Operations

Public contact point

Organisation

Palo Biofarma S.L.

Contact name

Clinical Project Manager

Third parties 3

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 12 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

9 submissions — initial application plus substantial / non-substantial modifications