Extension Study to PTR-01-002

Related Clinical Trial
Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over) Growth Hormone in EB Dose-ranging Study of Dentoxol® Mouthrinse for Managing Oral Symptoms in People With Epidermolysis Bullosa. Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa Extension Study to PTR-01-002 Long-Term Follow-up Protocol A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa Improve Adherence to Weak or Strong Opioid Analgesics at the Time of Care in Children With Hereditary Epidermolysis Bullosa Gynecological Follow-up of Patients With Dystrophic Epidermolysis Bullosa (EBD) Topical Gentamicin Nonsense Suppression Therapy of EB The Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III Trial A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB The Objective of This Study is to Compare the Efficacy and Safety of Beremagene Geperpavec (B-VEC) Topical Gel With That of Placebo for the Treatment of Dystrophic Epidermolysis Bullosa (DEB). Trial To Assess Efficacy Of A Chimeric Skin In Patients With Epidermolysys Bullosa Randomised Double Blind Placebo Controlled Cross Over Design of the Efficacy of Topical Morphine for Inflammatory Pain in Children With Epidermolysis Bullosa Study of Cellutome System for Treatment of Individual Lesions in EB Pts Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa Biochemical Correction of Severe EB by Allo HSCT and “Off-the-shelf” MSCs Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa Study of the Nutritional, Metabolic, and Body Composition Profile in Children and Adolescents With Epidermolysis Bullosa MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs Randomised Double Blind Crossover Placebo Controlled Study to Evaluate the Efficacy of Tetracycline in Epidermolysis Bullosa Observational Study of a Cohort of Patients With Hereditary Epidermolysis Bullosa Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa A Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa Evaluation of the Efficacy of ROPIVACAINE in Children and Young Adults With Hereditary Epidermolysis Bullosa Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10) The Efficacy of Trimethoprim in Wound Healing of Patients With Epidermolysis Bullosa A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa A Follow-up Study to Evaluate the Efficacy and Safety of ALLO-ASC-DFU in ALLO-ASC-EB-101 Clinical Trial Proof of Concept Study for a Dressing Glove Pregabalin Treatment for RDEB Pain and Itch Study of Alwextin® Cream in Treating Epidermolysis Bullosa A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing Uses of Irradiated Human Amniotic Membrane in the Treatment of Dystrophic Epidermolysis Bullosa Patients Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa A Comparative Study of the Healing of Chronic Ulcers of Recessive Epidermolysis Bullosa : Dressing vs Amniotic Membrane Topical QR-313 in Dominant Dystrophic Epidermolysis Bullosa (DDEB) or Recessive Dystrophic Epidermolysis Bullosa (RDEB) Due to Mutation(s) in Exon 73 of the COL7A1gene Rigosertib for RDEB-SCC Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa Short Term Observational Study in DEB Patients Gentamicin for RDEB Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB. Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients Self-Assembled Skin Substitute for the Treatment of Epidermolysis Bullosa Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa Topical Beremagene Geperpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB) A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Molecular Signatures of Cutaneous Squamous Cell Carcinoma During Recessive Dystrophic Epidermolysis Bullosa Treatment of Chronic and Non-Chronic Wounds in Patients With Recessive Dystrophic Epidermolysis Bullosa Using Helicoll Collagen Dressings Versus Standard of Care Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) MSC EVs in Dystrophic Epidermolysis Bullosa Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer Study of Immune Tolerance and Capacity for Wound Healing of Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate) Characteristics of Adult Patients With Recessive Dystrophic Epidermolysis Bullosa Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa Characteristics of Patients With Recessive Dystrophic Epidermolysis Bullosa Study to Evaluate the Safety of ALLO-ASC-DFU in the Subjects With Dystrophic Epidermolysis Bullosa The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB) A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa

Brief Title

Extension Study to PTR-01-002

Official Title

A Phase 2b Open-Label Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01 in Study PTR-01-002

Brief Summary

      A sub-set of patients who participated in PTR-01-002 will be enrolled in an open-label study,
      if they meet the study eligibility criteria.
    

Detailed Description

      Protocol PTR-01-003 is a 4-part Phase 2, open-label study of PTR-01 in patients who
      satisfactorily completed study PTR-01-002 and meet current enrollment criteria.

      In Part 1, patients will be monitored monthly for up to 2 months with patient-reported and
      Investigator assessments. In Part 2, patients will receive a dose of 3.0 mg/kg every week for
      a total of 4 doses. This will be followed by Part 3 in which patients will receive a dose of
      3.0 mg/kg monthly for a total of 5 additional doses. At the end of each dosing period,
      efficacy assessments will be performed. During Part 4, patients will be evaluated at Months 1
      and 3 after completion of dosing to assess the durability of wound healing and other efficacy
      parameters. Safety will be assessed continuously throughout the study.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Sustained wound healing

Secondary Outcome

 Delivery of recombinant collagen 7 (PTR-01) to skin

Condition

Recessive Dystrophic Epidermolysis Bullosa

Intervention

PTR-01

Study Arms / Comparison Groups

 PTR-01
Description:  All patients will receive a PTR-01 dose of 3.0 mg/kg once weekly every week for a total of 4 doses, followed by a dose of 3.0 mg/kg once monthly for a total of 5 doses.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

2

Start Date

December 2, 2021

Completion Date

November 30, 2022

Primary Completion Date

November 30, 2022

Eligibility Criteria

        Inclusion Criteria:

          1. Willing to provide informed consent form, or if 12 to <18 years of age, legal guardian
             has provided informed consent form and the minor has signed an assent form
             acknowledging that they understand and agree to study procedures.

          2. Has satisfactorily completed participation in PTR-01-002.

          3. Agrees to use contraception as follows:

               -  For women of childbearing potential (WOCBP) agrees to use highly effective
                  contraceptive (including abstinence) methods from Screening, through the study,
                  and for at least 10 weeks after the last dose of study drug. Non-childbearing
                  potential is defined as a female who meets either of the following criteria: age
                  ≥50 years and no menses for at least 1 year or documented hysterectomy, bilateral
                  tubal ligation, or bilateral oophorectomy.

               -  For males, agrees to use a condom with any WOCBP sexual partner from Day 1 of
                  study treatment, through the study, and at least 10 weeks after the last dose of
                  study drug.

          4. Be willing and able to comply with this protocol.

        Exclusion Criteria:

          1. Has known systemic hypersensitivity to any of the inactive ingredients in PTR-01.

          2. Has previously had an anaphylactic reaction to PTR-01.

          3. Is pregnant or nursing.

          4. Has received in the last six months any investigational gene therapy product or in the
             last three months any non-gene therapy investigational products (other than PTR-01).

          5. Is anticipated to receive new regimens of antibiotics or other anti-infectives during
             the trial.

          6. Has any other medical or personal condition that, in the opinion of the Investigator,
             may potentially compromise the safety or compliance of the
      

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Study Director, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT05143190

Organization ID

PTR-01-003


Responsible Party

Sponsor

Study Sponsor

Phoenix Tissue Repair, Inc.

Collaborators

 Phoenix Tissue Repair, a BridgeBio company

Study Sponsor

Study Director, Study Director, Phoenix Tissue Repair


Verification Date

March 2022