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Disease

pulmonary fibrosis and/or bone marrow failure, Telomere-related, 1

Late-stage therapeutic development

Also known as TERT pulmonary fibrosis and/or bone marrow failure, Telomere-related, pulmonary fibrosis and/or bone marrow failure, Telomere-related caused by mutation in TERT, pulmonary fibrosis and/or bone marrow failure, Telomere-related, type 1, PFBMFT1.

23
Clinical trials
2
Associated genes
1
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 5 clinical trials expected to report results, the earliest in Q4 2027.
Upcoming Milestones
Clinical Milestones11View all 11
Results expected 5
Recently completed 4
Other updates 2
+3 more in the activity timeline below
Activity timeline11

Clinical trials

15 sponsors · 4 new · 3 completed in the last 12 months (net +3)

The current development programme across all trial phases.

Clinical programme
23
All trials
6
Active
18
Late-stage
6
Completed
Late-stage studies

A Multicenter, Parallel, Randomized, Placebo (Double-blind) and Pirfenidone (Open-label) Controlled Phase III Clinical Trial Evaluating the Efficacy and Safety of HEC585 Tablets in Patients With Idiopathic Pulmonary Fibrosis (IPF)

Phase 3 · Recruiting · Sunshine Lake Pharma Co., Ltd.

A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation With Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

Phase 3 · Active, not recruiting · Boston Children's Hospital

A Double Blind, Randomized, Placebo-controlled Exploratory Trial to Investigate the Efficacy and Safety of Nerandomilast Over 24 Months When Administered in Individuals With Interstitial Lung Abnormalities and a Family History of Pulmonary Fibrosis to Reduce the Risk of Worsening (DROP-FPF)

Phase 3 · Recruiting · Boehringer Ingelheim

A Multicentre, Randomised, Double-blind, Placebo-controlled, Adaptive Design Clinical Trial to Evaluate the Efficacy and Safety of TDI01 Suspension in the Treatment of Idiopathic Pulmonary Fibrosis (IPF)

Phase 3 · Recruiting · Beijing Tide Pharmaceutical Co., Ltd

A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy, Safety, and Tolerability of BMS-986278 in Participants With Progressive Pulmonary Fibrosis

Phase 3 · Active, not recruiting · Bristol-Myers Squibb

Glucocorticoids Versus Placebo for the Treatment of Acute Exacerbation of Idiopathic Pulmonary Fibrosis: a Randomized Controlled Trial

Phase 3 · Recruiting · Fondation Hôpital Saint-Joseph
Recruiting

A Multicenter, Parallel, Randomized, Placebo (Double-blind) and Pirfenidone (Open-label) Controlled Phase III Clinical Trial Evaluating the Efficacy and Safety of HEC585 Tablets in Patients With Idiopathic Pulmonary Fibrosis (IPF)

Phase 3 · Recruiting · Sunshine Lake Pharma Co., Ltd.

A Double Blind, Randomized, Placebo-controlled Exploratory Trial to Investigate the Efficacy and Safety of Nerandomilast Over 24 Months When Administered in Individuals With Interstitial Lung Abnormalities and a Family History of Pulmonary Fibrosis to Reduce the Risk of Worsening (DROP-FPF)

Phase 3 · Recruiting · Boehringer Ingelheim

A Multicentre, Randomised, Double-blind, Placebo-controlled, Adaptive Design Clinical Trial to Evaluate the Efficacy and Safety of TDI01 Suspension in the Treatment of Idiopathic Pulmonary Fibrosis (IPF)

Phase 3 · Recruiting · Beijing Tide Pharmaceutical Co., Ltd

Glucocorticoids Versus Placebo for the Treatment of Acute Exacerbation of Idiopathic Pulmonary Fibrosis: a Randomized Controlled Trial

Phase 3 · Recruiting · Fondation Hôpital Saint-Joseph
Recently completed

Prospective Treatment Efficacy in IPF Using Genotype for Nac Selection (PRECISIONS) Trial

Phase 3 · Completed · Fernando J Martinez

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis (TETON-1)

Phase 3 · Completed · United Therapeutics

Pragmatic Management of Progressive Disease in Idiopathic Pulmonary Fibrosis: a Randomized Trial

Phase 4 · Completed · Hospices Civils de Lyon

Ciclosporin + Antithymocyte Immunoglobulin + Avatrombpag Versus Ciclosporin + Avatrombpag for the Treatment of Severe Aplastic Anemia in the Elderly

Phase 4 · Completed · Peking Union Medical College Hospital

A Phase III Multicenter Randomized Study of Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia

Phase 3 · Completed · Jiangsu HengRui Medicine Co., Ltd.

A Trial of Intensive Multi-Modality Therapy for Extra-Ocular Retinoblastoma

Phase 3 · Completed · Children's Oncology Group

Associated genes

2 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

TERT0.81TINF20.12

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Telomerase reverse transcriptasevia TERT0.81

Reference

Authoritative identity, definition & identifiers.

Synonyms

TERT pulmonary fibrosis and/or bone marrow failure, Telomere-related, pulmonary fibrosis and/or bone marrow failure, Telomere-related caused by mutation in TERT, pulmonary fibrosis and/or bone marrow failure, Telomere-related, type 1, PFBMFT1

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.