Extension Study of Efficacy and Safety of LTP001 in Pulmonary Arterial Hypertension Participants

NCT05764265 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: CLTP001A12201E12022-002007-38
• Study Type: interventional
• Target: 31
• Locations: 7 countries
• Sites: 12

Brief Summary

The purpose of this study was to measure the long-term safety and efficacy profile of LTP001 in participants with pulmonary arterial hypertension (PAH). The study offered participants who had completed the CLTP001A12201 double-blind parent study in PAH an opportunity to receive LTP001 (whether they were on LTP001 or not). Unblinding of the treatment received in CLTP001A12201 was generally not needed but could occur on request by the investigator.

Conditions

Pulmonary Arterial Hypertension

Keywords

Pulmonary Hypertension

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Incidence and severity of adverse events (AEs) by treatment group, including changes in the vital signs, electrocardiogram and laboratory results qualifying and reported as AEs. Due to the study termination, no patient reached Week 52. At the end of treatment visit, final safety assessments were performed.

  Up to approximately 45 weeks

Secondary Outcomes (15)

• Change From Baseline in Average Cardiac Output (CO) at Week 26

  Baseline, Week 26

• Change From Baseline in Mean Pulmonary Artery (PA) Pressure at Week 26

  Baseline, Week 26

• Change From Baseline in Pulmonary Capillary Wedge Pressure (PCWP) at Week 26

  Baseline, Week 26

• Change From Baseline in Right Heart Catheterization Pulmonary Vascular Resistance (PVR) at Week 26

  Baseline, Week 26

• Change From Baseline in Right Atrium (RA) Pressures at Week 26

  Baseline, Week 26

Study Arms (1)

LTP001

EXPERIMENTAL

Participants received LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks

Drug: LTP001

Interventions

LTP001 DRUG

LTP001, 6 mg, was administered orally once daily in the morning

LTP001

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Written informed consent must have been obtained before any assessment was performed.
• Participant was currently completing the Novartis-sponsored study CLTP001A12201 in PAH and completed key efficacy and safety procedures up to the end of treatment of the core study, without meeting discontinuation criteria in the core study.
• Willingness and ability to comply with scheduled visits, treatment plans and any other study procedures.
• In the opinion of the Investigator would benefit from LTP001 treatment.

You may not qualify if:

• History of hypersensitivity to the study treatment.
• Sexually active males not committing to condom use precautions: sexually active males must have used a condom during intercourse while taking drug and for 24 hours after stopping study medication and should not father a child in this period nor donate sperm. A condom was required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid.
• Required or planned transplant or heart/lung surgery.
• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they were using highly effective methods of contraception while taking study treatment and until EOT visit (2 weeks post-last treatment). Highly effective contraception methods included:
• Total abstinence (when this was in line with the preferred and usual lifestyle of the participant. Periodic abstinence (e.g. calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal were not acceptable methods of contraception.
• Female sterilization (had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or bilateral tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman had been confirmed by follow up hormone level assessment.
• Male sterilization (at least 6 months prior to screening). For female participants on the study, the vasectomized male partner should have been the sole partner for that participant
• Use of oral, estrogen and progesterone, injected, or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate \< 1%), for example hormone vaginal ring or transdermal hormone contraception.
• In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.
• Women were considered post-menopausal if they had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate history of vasomotor symptoms). Women were considered not of child-bearing potential if they were post-menopausal or had surgical bilateral oophorectomy (with or without hysterectomy) or total hysterectomy at least six weeks prior. In the case of oophorectomy alone, only when the reproductive status of the woman had been confirmed by follow up hormone level assessment was she considered not of child bearing potential.
• Pregnant or nursing (lactating) women, where pregnancy was defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
• Acute or chronic impairment (other than dyspnea), which would limit the ability to comply with study requirements, including interference with physical activity or execution of study procedures such as 6MWT (e.g., angina pectoris, claudication, musculoskeletal disorder, need for walking aids).
• Permanent discontinuation of Novartis drug in the core efficacy study due to toxicity or disease progression despite active treatment, non-compliance to study procedures, withdrawal of consent or any other reason.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (12)

Pulmonary Associates PA

Mesa, Arizona, 85206, United States

Location

Novartis Investigative Site

Caba, Buenos Aires, C1025ABI, Argentina

Location

Novartis Investigative Site

Dresden, Saxony, 01307, Germany

Location

Novartis Investigative Site

Heidelberg, 69120, Germany

Location

Novartis Investigative Site

Amsterdam, North Holland, 1081 HV, Netherlands

Location

Novartis Investigative Site

Krakow, 31 202, Poland

Location

Novartis Investigative Site

Lodz, 91-347, Poland

Location

Novartis Investigative Site

Wroclaw, 50-556, Poland

Location

Novartis Investigative Site

Barcelona, 08036, Spain

Location

Novartis Investigative Site

Madrid, 28041, Spain

Location

Novartis Investigative Site

Málaga, 29010, Spain

Location

Novartis Investigative Site

Sheffield, South Yorkshire, S10 2JF, United Kingdom

Location

Related Links

• A Plain Language Trial Summary is available on www.novctrd.com

MeSH Terms

Conditions

Pulmonary Arterial Hypertension; Hypertension, Pulmonary

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@Novartis.com

+ 1 862 778 8300

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 10, 2023
• First Posted: March 10, 2023
• Study Start: March 27, 2023
• Primary Completion: April 26, 2024
• Study Completion: May 14, 2024
• Last Updated: January 27, 2026
• Results First Posted: December 26, 2025

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

US (1); AR (1); PL (3); ES (3); DE (2); NL (1); GB (1)